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Market Research Report

Breakthrough Therapies: Market Dynamics and Investment Opportunities

Published by BCC Research Product code 424439
Published Content info 250 Pages
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Breakthrough Therapies: Market Dynamics and Investment Opportunities
Published: January 25, 2017 Content info: 250 Pages
Description

Report Highlights

The global market for breakthrough therapy designation drugs should reach $99.2 billion by 2022 from $48.8 billion in 2017 at a compound annual growth rate (CAGR) of 15.2%, from 2017 to 2022.

Report Includes:

  • An overview of the global markets for breakthrough therapies, their dynamics, and investment opportunities
  • Analyses of global market trends, with data from 2015, 2016, and projections of compound annual growth rates (CAGRs) through 2022
  • A look at the materials used in the industry ranging from inorganic clays and concrete to iron and steel, and to commodity and specialty polymers
  • The challenges and opportunities of developing breakthrough therapies
  • A compare and contrast of different fast track drug development approaches (logistics, criteria and implications) and the potential risk and rewards of gaining break through designation
  • Analysis of the regulatory environment
  • Profiles of major players in the industry

Report Scope

This report highlights the challenges and opportunities of developing breakthrough therapies, it compares and contrasts difference fast track drug development approaches (logistics, criteria, and implications) and the potential risk and rewards of gaining BTD. It analyzes BTD in different therapy areas (cardiovascular, central nervous systems and neurology, rare diseases, oncology and other diseases), leading companies, approved and pipeline products, pricing market access and product revenues. It reviews BTD successes and failures and discusses the different business strategies that companies have adopted in order to maximize the competitive advantage of BTD. It summarizes the current regulatory framework and the potential application of BTD in other regions of the world, and the potential to combine BTD with early access to medicine schemes to improve patient access to medicine to treat rare diseases and address areas of high unmet clinical need.

Analyst Credentials

The practical pharmaceutical research experience of Dr. Cheryl L. Barton with a leading pharmaceutical company spans over ten years; as well, she has served as a Pan-European pharmaceutical analyst with a European bank. With her business, Dr. C.L. Barton Ltd., Dr. Barton provides independent, customized pharmaceutical thematic research to investment houses. Research reports combine independent scientific analysis with patient- and prescription-based models to forecast the potential sales growth of key developmental drugs and to isolate the key drivers within the pharmaceutical sector.

Dr. Bianca Piachaud-Moustakis has more than 10 years' experience as a freelance writer and research analyst specializing in pharmaceutical and healthcare intelligence. She has worked for a number of business intelligence providers in the U.K. She has a broad range of writing experience ranging from international academic and practitioner journals to market intelligence reports and short articles. Dr. Piachaud-Moustakis is also a reviewer for international peer-reviewed journals and works as a tutor on advanced-level business studies courses.

Table of Contents
Product Code: PHM176A

Table of Contents

CHAPTER 1 - INTRODUCTION

  • STUDY GOALS AND OBJECTIVES
  • REASONS FOR DOING THE REPORT
  • SCOPE OF REPORT
  • INTENDED AUDIENCE
  • METHODOLOGY AND INFORMATION SOURCES
  • ANALYSTS' CREDENTIALS
  • RELATED BCC RESEARCH REPORTS
  • BCC RESEARCH WEBSITE
  • DISCLAIMER

CHAPTER 2 - SUMMARY

  • SUMMARY TABLE: GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, THROUGH 2022 ($ MILLIONS)
  • SUMMARY FIGURE: GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, 2015-2022 ($ MILLIONS)

CHAPTER 3 - OVERVIEW

  • DEFINING BREAKTHROUGH THERAPY DESIGNATION
    • QUALIFYING CRITERIA FOR BREAKTHROUGH THERAPY DESIGNATION
      • Serious Condition
      • Existing (or Available) Therapies
      • Preliminary Clinical Evidence
      • May Demonstrate Substantial Improvement on Clinically Significant Endpoint(s)
    • THE BENEFITS OF BREAKTHROUGH THERAPY DESIGNATION
    • FDA'S APPROACHES TO EXPEDITED DRUG DEVELOPMENT
    • DRUGS GRANTED BREAKTHROUGH THERAPY DESIGNATION
      • TABLE 1: CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-SEPTEMBER 30, 2015
      • TABLE 2: CBER BREAKTHROUGH THERAPY REQUESTS FOR FINAL YEAR, OCTOBER 1, 2014--SEPTEMBER 30, 2015
      • FIGURE 1: BREAKTHROUGH THERAPY REQUESTS GRANTED BY DIVISION, 2015* (%)
    • PRIME (PRIORITY MEDICINE) SCHEME
      • FIGURE 2: PRIME SCHEME APPLICATIONS AND ELIGIBILITY
      • FIGURE 3: PRIME DRUG BY TYPE OF APPLICATION AND THERAPEUTIC AREA
      • FIGURE 4: PRIME DRUG APPLICATIONS, THERAPEUTIC AREA
      • TABLE 3: DRUGS GRANTED PRIME DESIGNATIONS, MARCH 7, 2016-AUGUST 24, 2016

CHAPTER 4 - COMMERCIAL APPLICATION OF BREAKTHROUGH THERAPY DESIGNATION

  • INTRODUCTION
    • FIGURE 5: FDA APPROVALS, BREAKTHROUGH THERAPY DESIGNATION AND DESIGNATION RESCINDED, BY STATUS, 2012-2016 (NUMBER)
    • FIGURE 6: FDA APPROVALS, BREAKTHROUGH DESIGNATION AND DESIGNATION RESCINDED, BY THERAPY AREA, 2012-2016 (NUMBER)
  • CARDIOVASCULAR
    • FIGURE 7: CARDIOVASCULAR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE (NUMBER)
    • TABLE 4: GLOBAL MARKET FOR CV BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
  • CV - BTD DRUGS APPROVED
    • TABLE 5: FDA APPROVED CV BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • Promacta/Revolade (Eltrombopag Olamine: Novartis/GlaxoSmithKline)
      • BTD: February 6, 2014
      • Drug Class: Thrombopoietin Receptor Agonist (Small Molecule)
      • Indication: Severe Aplastic Anemia (SAA; CV)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 6: GLOBAL MARKET FOR PROMACTA, THROUGH 2022 ($ MILLIONS/%)
    • Praxbind (Idarucizumab; Boehringer Ingelheim)
      • BTD: June 30, 2014
      • Drug Class: Humanized Antibody Fragment (Biologic)
      • Indication: Reversal of Bleeding and Clotting Associated with Pradaxa (CV)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 7: GLOBAL MARKET FOR PRAXBIND, THROUGH 2022 ($ MILLIONS/%)
  • CV - BTD IN DEVELOPMENT
    • TABLE 8: UNDER DEVELOPMENT CV BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • Reasanz (Serelaxin; Novartis)
      • BTD: June 21, 2013
      • Drug Class: Human Relaxin 2 Hormone (Biologic)
      • Indication: Acute Heart Failure (AHF; CV)
      • Clinical Stage
      • Forecast
        • TABLE 9: GLOBAL MARKET FOR REASANZ, THROUGH 2022 ($ MILLIONS/%)
    • Actemra/RoActemra (Tocilizumab; Roche)
      • BTD: June 10, 2015, and August 5, 2016
      • Drug Class: Humanized Anti Interleukin L-6 Receptor mAb (Biologic)
      • Indication: Systemic Sclerosis and Giant Cell Arteritis (CNS/Rare Disease)
      • Clinical Stage: Approved/Phase 3 BTD
        • SSC Trial
        • CGA Trials
      • Forecast
        • TABLE 10: GLOBAL MARKET FOR ACTEMRA/ROACTEMRA, THROUGH 2022 ($ MILLIONS/%)
  • CENTRAL NERVOUS SYSTEM AND NEUROLOGICAL DISORDERS
    • FIGURE 8: CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
    • TABLE 11: GLOBAL MARKET FOR CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
  • CNS - BTD DRUGS APPROVED
    • TABLE 12: FDA APPROVED CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • Nuplazid (Primavanserin: Acadia Pharmaceuticals)
      • BTD: February 9, 2014
      • Drug Class: Selective Serotonin Inverse Agonist (Small Molecule)
      • Indication: Major Depressive Disorder
      • Clinical Stage: Approved
      • Forecast
        • TABLE 13: GLOBAL MARKET FOR NUPLAZID, THROUGH 2022 ($ MILLIONS/%)
  • CNS - BTD IN DEVELOPMENT
    • TABLE 14: UNDER DEVELOPMENT CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • Deutetrabenazine ER (Teva Pharmaceuticals)
      • BTD: November 9, 2015
      • Drug Class: Vesicular Monoamine 2 Transporter Inhibitor (Small Molecule)
      • Indication: Tardive Dyskinesia (CNS)
      • Clinical Stage: Phase 2/3
      • Forecast
        • TABLE 15: GLOBAL MARKET FOR DEUTETRABENAZINE ER, THROUGH 2022 ($ MILLIONS/%)
    • Ingrezza (Valbenazine; Neuroscience Biosceinces)
      • BTD: October 30, 2014
      • Drug Class: Vesicular Monoamine 2 Transporter Inhibitor (Small Molecule)
      • Indication: Tardive Dyskinesia (CNS)
      • Clinical Stage: Phase 3
        • Kinect 2 - Phase 2b
        • Kinect 3 - Phase 3
      • Forecast
        • TABLE 16: GLOBAL MARKET FOR INGREZZA, THROUGH 2022 ($ MILLIONS/%)
    • Arzerra (Ofatumumab; Novartis/Genmab)
      • BTD: October 11, 2013
      • Drug Class: Human Monoclonal Antibody Targeting CD20-Positive B Cells (Biologic)
      • Indication: Relapse Remitting Multiple Sclerosis (CNS/Autoimmune)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 17: GLOBAL MARKET FOR OF OFATUMUMAB, THROUGH 2022 ($ MILLIONS/%)
    • Oliceridine (TRV130; Trevena)
      • BTD: February 22, 2016
      • Drug Class: M Opioid Receptor Agonist (Small Molecule)
      • Indication
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 18: GLOBAL MARKET FOR OLICERIDINE, THROUGH 2022 ($ MILLIONS/%)
    • Rapastinel (GLYX-13; Allergan)
      • BTD: January 31, 2016
      • Drug Class: NMDA Partial Receptor Antagonist (Small Molecule)
      • Indication: Major Depressive Disorder (CNS)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 19: GLOBAL MARKET FOR RAPASTINEL, THROUGH 2022 ($ MILLIONS/%)
    • Sepranolone (UC1010; Asarina Pharma)
      • TD: February 22, 2016
      • Drug Class: GABA-A Modulating Steroid Antagonist (Small Molecule)
      • Indication: Premenstrual Dysphoric Disorder (PMDD)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 20: GLOBAL MARKET FOR SEPRANOLONE, THROUGH 2022 ($ MILLIONS/%)
    • NaBen (SND-13; SyneuRx International Corporation)
      • BTD: December 10, 2014
      • Drug class: D-Amino Acid Oxidase (DAAO) Inhibitor (Small Molecule)
      • Indication: Schizophrenia (CNS)
      • Clinical Stage: Phase 2/3
      • Forecast
        • TABLE 21: GLOBAL MARKET FOR SND-13, THROUGH 2022 ($ MILLIONS/%)
    • AVXS-101 (AveXis)
      • BTD: July 20, 2016
      • Drug Class: Gene Therapy (Biologic)
      • Indication: Spinal Muscular Atrophy (SMA; Rare Disease)
      • Clinical Stage: Phase 1
      • Forecast
        • TABLE 22: GLOBAL MARKET FOR AVXS-101, THROUGH 2022 ($ MILLIONS/%)
    • Esketamine (Johnson & Johnson)
      • BTD: November 2013 and August 16, 2016
      • Drug Class: N-methyl-D-aspartate (NMDA) Receptor Antagonist (Small Molecule)
      • Indication: Major Depressive Disorder (MDD)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 23: GLOBAL MARKET FOR ESKETAMINE, THROUGH 2022 ($ MILLIONS/%)
    • RBP-8000 (Indivior)
      • BTD: November 18, 2014
      • Drug Class: Cocaine Esterase Rescue Blocker (Small Molecule)
      • Indication: Cocaine Abuse
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 24: GLOBAL MARKET FOR RBP-8000, THROUGH 2022 ($ MILLIONS/%)
  • INFECTIOUS DISEASES
    • FIGURE 9: INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
    • TABLE 25: GLOBAL MARKET FOR INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
  • INFECTIOUS DISEASES - BTD APPROVED
    • TABLE 26: FDA APPROVED INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
    • Sovaldi (Gilead Sciences)
      • BTD: December 6, 2013
      • Drug Class: Nucleotide Analog Inhibitor (Small Molecule)
      • Indication: Hepatitis C Virus (Infectious Diseases)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 27: GLOBAL MARKET FOR SOLVADI, THROUGH 2022 ($ MILLIONS/%)
    • Epclusa (Gilead Sciences)
      • BTD: April 22, 2014
      • Drug Class: Nucleotide Analog Inhibitor + Pan-Genotypic NS5A Inhibitor (Small Molecule)
      • Indication: Hepatitis C Virus (Infectious Disease)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 28: GLOBAL MARKET FOR EPCLUSA, THROUGH 2022 ($ MILLIONS/%)
    • Technivie/Viekirax (AbbVie)
      • BTD: June 30, 2014
      • Drug Class: Fixed Dose Triple Antiviral (Small Molecule)
      • Indication: Hepatitis C Virus (Infectious Disease)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 29: GLOBAL MARKET FOR VIEKERA/TECHNIVIE, THROUGH 2022 ($ MILLIONS/%)
    • Zepatier (Elbasvir/Grazoprevir; Merck)
      • BTD: October 1, 2013
      • Drug Class: NS5A Inhibitor, NS3/4A Protease Inhibitor (Small Molecule)
      • Indication: Hepatitis C Virus (Infectious Disease)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 30: GLOBAL MARKET FOR ZEPATIER, THROUGH 2022 ($ MILLIONS/%)
    • Harvoni (Gilead Sciences)
      • BTD: October 10, 2014
      • Drug Class: NS5A Inhibitor NS5B Inhibitor (Small Molecule)
      • Indication: Hepatitis C Virus (Infectious Disease)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 31: GLOBAL MARKET FOR HARVONI, THROUGH 2022 ($ MILLIONS/%)
  • INFECTIOUS DISEASES - BTD IN DEVELOPMENT
    • TABLE 32: UNDER DEVELOPMENT INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
    • Arikayce (Amikacin Sulfate: Insmed)
      • BTD: June 20, 2014
      • Drug Class: Aminoglycoside Antibiotic. (Small Molecule)
      • Indication: Nontuberculous Mycobacterial (NTM; Infectious Disease)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 33: GLOBAL MARKET FOR ARIKAYCE, THROUGH 2022 ($ MILLIONS/%)
    • Fostemsavir (ViiV Healthcare)
      • BTD: July 22, 2015
      • Drug Class: Fusion Inhibitor (Small Molecule)
      • Indication: Human Immunodeficiency Virus (HIV)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 34: GLOBAL MARKET FOR FOSTEMSAVIR, THROUGH 2022 ($ MILLIONS/%)
    • Glecaprevir/Pibrentasvir (ABT-493/ABT-530; AbbVie/Enanta Pharmaceuticals)
      • BTD: September 30, 2016
      • Drug Class: NS3 Protein Inhibitor (Small Molecule)
      • Indication: Hepatitis C Virus (HCV; Infectious Disease)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 35: GLOBAL MARKET FOR GLECAPREVIR/PIBRENTASVIR, THROUGH 2022 ($ MILLIONS/%)
    • Ibalizumab (TaiMed Biologics/Theratechnologies)
      • BTD: February 2015
      • Drug Class: CD4 mAb (Biologic)
      • Indication: Human Immunodeficiency Virus (HIV)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 36: GLOBAL MARKET FOR IBALIZUMAB, THROUGH 2022 ($ MILLIONS/%)
    • Tafenoquine (GlaxoSmithKline/MMV)
      • BTD: 20 December, 2013
      • Drug Class: 8-Aminoquinoline Derivative (Small Molecule)
      • Indication: Hepatitis C Virus (HCV; Infectious Disease)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 37: GLOBAL MARKET FOR TAFENOQUINE, THROUGH 2022 ($ MILLIONS/%)
    • V920 (Merck & Co./NewLink Genetics Corporation)
      • BTD: 25, July 2016
      • Drug Class: Ebola Vaccine (Biologic)
      • Indication: Ebola Zane (Infectious Disease)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 38: GLOBAL MARKET FOR V920, THROUGH 2022 ($ MILLIONS/%)
  • ONCOLOGY
    • FIGURE 10: CANCER DRUGS WITH BREAKTHROUGH THERAPY DESIGNATION, BY CANCER TYPE, 2016 (NUMBER OF APPROVED BTD DRUGS)
    • FIGURE 11: CANCER BREAKTHROUGH THERAPY DESIGNATION, BY COMPANY, 2016 (NO. OF BTD IN ONCOLOGY)
    • TABLE 39: GLOBAL MARKET FOR ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
  • ONCOLOGY - BTD DRUGS APPROVED
    • TABLE 40: FDA APPROVED ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2015
    • Alecensa (Alectinib: Genentech/Roche)
      • BTD: June 2013 and October 2016
      • Drug Class: Oral Inhibitor of Anaplastic Lymphoma Kinase (ALK) Inhibitor (Small Molecule)
      • Indication: ALK+ NSCLC (Oncology)
      • Clinical Stage: Approved
        • Second Line Therapy - NP28761 (Study 1)
        • Second Line Therapy - NP28673 (Study 2)
        • First Line Therapy - J-ALEX Study
      • Forecast
        • TABLE 41: GLOBAL MARKET FOR ALENCENSA, THROUGH 2022 ($ MILLIONS/%)
    • Arzerra (Ofatumumab; GlaxoSmithKline/Genmab)
      • BTD: September 13, 2013
      • Drug Class: mAb Target CD20 (Biologic)
      • Indication: CLL
      • Clinical Stage: Approved
      • Forecast
        • TABLE 42: GLOBAL MARKET FOR ARZERRA, THROUGH 2022 ($ MILLIONS/%)
    • Blincyto (Blinatumomab; Amgen)
      • BTD: December 3, 2014
      • Drug Class: CD3/CD19 BiTE Antibody (Biologic)
      • Indication: B-Cell ALL (Oncology)
        • FIGURE 12: TREATMENT ALGORITHM FOR ALL PATIENTS
      • Clinical Stage: Approved
      • Forecast
        • TABLE 43: GLOBAL MARKET FOR BLINCYTO, THROUGH 2022 ($ MILLIONS/%)
    • Darzalex (Daratumumab; Genmab/Johnson & Johnson)
      • BTD: May 1, 2013, May 27, 2013, and July 2016
      • Drug Class: mAb CD38 (Biologic)
      • Indication: Multiple Myeloma (Oncology)
      • Clinical Stage: Phase 3
        • CASTOR Trial-Second Line
        • POLLUX Trial - Second Line
      • Forecast
        • TABLE 44: GLOBAL MARKET FOR DARZALEX, THROUGH 2022 ($ MILLIONS/%)
    • Gazyva/Gazyvaro (Obinutuzumab; Genentech/Roche)
      • BTD: May 2013
      • Drug Class: mAb TargetingCD20 (Biologic)
      • Indication: CLL
      • Clinical Stage: Approved
      • Forecast
        • TABLE 45: GLOBAL MARKET FOR GAZYVA, THROUGH 2022 ($ MILLIONS/%)
    • Ibrance (Palbociclib; Pfizer)
      • BTD: February 3, 2015
      • Indication: Her2 Metastatic Breast Cancer (Oncology)
      • Drug Class: CDK4/6 Inhibitor (Small Molecule)
      • Clinical Stage: Phase 3
        • PALOMA-2 Trial - Combination Therapy First-Line
        • PALOMA-3 - Combination Therapy
      • Forecast
        • TABLE 46: GLOBAL MARKET FOR IBRANCE, THROUGH 2022 ($ MILLIONS/%)
    • Imbruvica (Ibrutinib; Johnson & Johnson/ Pharmacyclic)
      • BTD: January 2013
      • Drug Class: BTK Inhibitor (Small Molecule)
      • Indication: CLL (Oncology)
      • Clinical Phase: Approved
      • Forecast
        • TABLE 47: GLOBAL MARKET FOR IMBRUVICA, THROUGH 2022 ($ MILLIONS/%)
    • Keytruda (Pembrolizumab; Merck)
      • BTD: October 27, 2014, November 2, 2015, April 18, 2016
      • Drug Class: Anti-PD-1 Monoclonal Antibody (Biologic)
      • Indication: Solid Tumors (Oncology)
      • Clinical Stage: Approved
        • Melanoma
          • KEYNOTE-001: Phase 1b
          • KEYNOTE-002: Phase 2
          • KEYNOTE-028 Study: Phase 1b
          • KEYNOTE-006 Study: Phase 3
          • NSCLC - First-Line KEYNOTE-010 Phase 2/3
      • Classical Hodgkin Lymphoma
      • Colorectal Cancer
      • Forecast
        • TABLE 48: GLOBAL MARKET FOR KEYTRUDA, THROUGH 2022 ($ MILLIONS/%)
    • Lartruvo (Olaratumab; Eli Lilly)
      • BTD: August 6, 2015
      • Drug Class: Platelet Derived Growth Factor Receptor Alpha Antagonist (Small Molecule)
      • Indication: Soft Tissue Sarcoma
      • Clinical Stage: Approved
      • Forecast
        • TABLE 49: GLOBAL MARKET FOR LARTRUVO, THROUGH 2022 ($ MILLIONS/%)
    • Lenvima/Kisplyx (Lenvatinib; Eisai)
      • BTD: August 6, 2015
      • Drug Class: Tyrosine Kinase Inhibitor (Small Molecule)
      • Indication: Thyroid Cancer (Oncology)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 50: GLOBAL MARKET FOR LENVIMA/KISPLYX, THROUGH 2022 ($ MILLIONS/%)
    • Lynparza (Olaparib; AstraZeneca)
      • BTD: January 27, 2016
      • Drug Class: Poly ADP-Ribose Polymerase Inhibitor (PARP Oncology)
      • Indication: Metastatic Castration-Resistant Prostate Cancer (mCRPC; Oncology)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 51: GLOBAL MARKET FOR LYNPARZA, THROUGH 2022 ($ MILLIONS/%)
    • Midostaurin (PKC412; Novartis)
      • BTD: February 19, 2016
      • Drug Class: Oral, Multi-Targeted Kinase Inhibitor (Small Molecule)
      • Indication: Acute Myeloid Leukemia (AML; Oncology)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 52: GLOBAL MARKET FOR MIDOSTAURIN, THROUGH 2022 ($ MILLIONS/%)
    • Opdivo (Nivolumab; BMS)
      • BTD: April 25, 2016, and June 27, 2016
      • Drug Class: PD-1 Inhibitor (Small Molecule)
      • Indication: Solid Tumors (Oncology)
      • Clinical Stage: Advanced Melanoma Checkmate 067: Phase 3
      • Checkmate 069: Phase 2
      • Bladder Cancer CheckMate-032:
      • Advanced Squamous Non-Small Cell Lung Cancer: CheckMate -017: Phase 3
      • CheckMate -063: Phase 2
      • Second-Line Treatment of Metastatic Non-Squamous NSCLC
      • Metastatic Renal Cell Carcinoma CheckMate 025 Trial: Phase 3
      • CheckMate-057: Phase 3
      • Forecast
        • TABLE 53: GLOBAL MARKET FOR OPDIVO, THROUGH 2022 ($ MILLIONS/%)
    • Tagrisso (Osimertinib; AstraZeneca)
      • BTD Date; April 24, 2104
      • Drug Class: EGFR- Tyrosine Kinase Inhibitor (Small Molecule)
      • Indication: Non-Small-Cell Lung Cancer (Oncology)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 54: GLOBAL MARKET FOR TAGRISSO, SALES FORECASTS, THROUGH 2022 ($ MILLIONS/%)
    • Tafinlar/Mekinist (Dabrafenib/Trametinib; GlaxoSmithKline)
      • BTD: January 2014 (Monotherapy) and July 23, 2015 (Combination Therapy)
      • Drug Class: BRAF Inhibitor plus MEK Inhibitor (Biologic)
      • Indication: NSCLC (Oncology).
      • Clinical Stage: Approved
        • Monotherapy Trial
      • Combination Therapy Trial
      • Forecast
        • TABLE 55: GLOBAL MARKET FOR TAFINLAR/MEKINIST, THROUGH 2022 ($ MILLIONS/%)
    • Tecentriq (Atezolizumab; Genentech/Roche)
      • BTD: January 23, 2014
      • Drug Class: Programmed Cell Death--1 Ligand 1 Monoclonal Antibody (Biologic)
      • Indication: Metastatic Urothelial Carcinoma (Oncology)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 56: GLOBAL MARKET FOR TECENTRIQ, THROUGH 2022 ($ MILLIONS/%)
    • Venclexta (Venetoclax; AbbVie/Roche/Genentech)
      • BTD: April 2015 and January 2016
      • Drug Class: Oral B-cell Lymphoma-2 Inhibitor (Small Molecule)
      • Indication: CLL (Oncology)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 57: GLOBAL MARKET FOR VENCLEXTA, THROUGH 2022 ($ MILLIONS/%)
    • Xalkori (Crizotinib; Pfizer)
      • BTD: August 26, 2011
      • Drug Class: Anaplastic Lymphoma Kinase (ALK) Tyrosine Kinase Receptor Inhibitor (Small Molecule)
      • Indication: ALK+ NSCLC (Oncology)
      • Clinical Stage: Approved
        • First-Line Therapy - Study 1
        • Second-Line Therapy - Study 2
      • Forecast
        • TABLE 58: GLOBAL MARKET FOR XALKORI, THROUGH 2022 ($ MILLIONS/%)
    • Zydelig (Idelalisib, Gilead Sciences)
      • BTD: November 18, 2013
      • Drug Class: Oral Phosphoinositide 3-Kinase (PI3K) Delta Inhibitor (Small Molecule)
      • Indication: CLL
      • Clinical Stage: Approved
      • Forecast
        • TABLE 59: GLOBAL MARKET FOR ZYDELIG, THROUGH 2022 ($ MILLIONS/%)
    • Zykadia (Ceritinib; Novartis)
      • BTD: March 15, 2013
      • Drug Class: Anaplastic Lymphoma Kinase (ALK) Tyrosine Kinase Receptor Inhibitor (Small Molecule)
      • Indication: ALK+ NSCLC (Oncology)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 60: GLOBAL MARKET FOR ZYKADIA, THROUGH 2022 ($ MILLIONS/%)
  • ONCOLOGY - BTD IN DEVELOPMENT
    • TABLE 61: UNDER DEVELOPMENT ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • Abemaciclib (Eli Lilly)
      • BTD: August 8, 2015
      • Indication: Her2 Metastatic Breast Cancer (Oncology)
      • Drug Class: CDK4-6 Inhibitor (Small Molecule)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 62: GLOBAL MARKET FOR ABEMACICLIB, THROUGH 2022 ($ MILLIONS/%)
    • Avelumab (Merck KGaA/PFizer)
      • BTD: November 18, 2015
      • Drug Class: Fully Human Monoclonal Antibody Targeting Programmed Death Ligand-1 (Biologic)
      • Indication: Metastatic Merkel Cell Carcinoma (Oncology)
      • Clinical Stage: Phase 3 NSCL/ Phase 2 MCC/ Phase 1 Solid Tumors
      • Forecast
        • TABLE 63: GLOBAL MARKET FOR AVELUMAB, THROUGH 2022 ($ MILLIONS/%)
    • Brigatinib (AP26113; ARIAD Pharmaceuticals)
      • BTD: October 1, 2014
      • Drug Class: Oral Inhibitor of Anaplastic Lymphoma Kinase (ALK) Inhibitor (Small Molecule)
      • Indication: ALK+ NSCLC (Oncology)
      • Clinical Stage: Filed
      • Forecast
        • TABLE 64: GLOBAL MARKET FOR BRIGATINIB, THROUGH 2022 ($ MILLIONS/%)
    • CTL019 (Novartis)
      • BTD: July 2014
      • Drug Class: Autologous Chimeric Antigen Receptor (CAR)- T Tell Therapy targeting CD19 (Biologic)
      • Indication: CLL
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 65: GLOBAL MARKET FOR CTL019, THROUGH 2022 ($ MILLIONS/%)
    • CRS-207 and GVAX (Aduro Biotech)
      • BTD: 17 February, 2016
      • Drug Class: Tumor-Associated Antigen Mesothelin (Biologic)
      • Indication: Metastatic Pancreatic Cancer (Oncology)
      • Clinical Stage: Phase 2b
      • Forecast
        • TABLE 66: GLOBAL MARKET FOR CRS-207 AND GVAX PANCREAS, THROUGH 2022 ($ MILLIONS/%)
    • Durvalumab (MEDI4736; AstraZeneca-MedImmune)
      • BTD: February 17, 2016
      • Drug Class: Humanized Monoclonal Antibody Targeting Programmed Death Ligand-1 (Biologic)
      • Indication: Metastatic Urothelial Bladder (Oncology)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 67: GLOBAL MARKET FOR DURVALUMAB, THROUGH 2022 ($ MILLIONS/%)
    • EBV-CTL (Atara and MSKCC)
      • BTD: February 3, 2015
      • Drug Class: Allogeneic Hematopoietic Cell Transplant (Biologic)
      • Indication: EBV-Associated Lymphoproliferative Disease (Oncology)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 68: GLOBAL MARKET FOR EBV-CTL, THROUGH 2022 ($ MILLIONS/%)
    • Entinostat (Syndax Pharmaceuticals)
      • BTD: September 11, 2013
      • Drug Class: Antibody Histone Deacetylase Inhibitor (Small Molecule)
        • FIGURE 13: ENTINOSTAT MECHANISM OF ACTION
      • Indication: Advanced HER2 Breast Cancer
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 69: GLOBAL MARKET FOR ENTINOSTAT, THROUGH 2022 ($ MILLIONS/%)
    • Inotuzumab Ozogamicin (Pfizer, Celltech)
      • BTD: October 19, 2015
      • Drug Class: Antibody Drug Conjugate (ADC) mAb Targeting CD22 plusC Agent Inotuzumab (Biologic)
      • Indication: B-cell ALL
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 70: GLOBAL MARKET FOR INOTUZUMAB OZOGAMICIN, THROUGH 2022 ($ MILLIONS/%)
    • JCAR015 (Juno Therapeutics)
      • BTD: November 24, 2014
      • Drug Class: Autologous Chimeric Antigen Receptor (CAR) - T Cell Therapy Targeting CD19 (Biologic)
      • Indication: Relapse-Refractory B-Cell ALL
      • Clinical Stage: Phase 1-2
      • Forecast
        • TABLE 71: GLOBAL MARKET FOR JCAR015, THROUGH 2022 ($ MILLIONS/%)
    • KTE-C19 (Kite Pharma)
      • BTD: December 7, 2015
      • Drug Class: Allogenic Chimeric Antigen Receptor (CAR) - T Cell Therapy Targeting CD19 (Biologic)
      • Indication: Refractory, Diffuse Large B-Bell Lymphoma (DLBCL; Oncology)
        • Clinical Stage: Phase 2
      • Forecast
        • TABLE 72: GLOBAL MARKET FOR KTE-C19, THROUGH 2022 ($ MILLIONS/%)
    • LOXO-101 (Loxo Oncology, Inc)
      • BTD: July 13, 2016
      • Drug Class: Pan Tropomyosin Receptor Kinase Inhibitor (Small Molecules)
      • Indication: Solid Tumors (Oncology)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 73: GLOBAL MARKET FOR LOXO-101, THROUGH 2022 ($ MILLIONS/%)
    • Nicord (Elbit Imaging/Gamida Cell)
      • BTD: October 11, 2016
      • Drug Class: Allogeneic Hematopoietic Cell Transplant (Biologic)
      • Indication: haematological malignancies (Oncology)
      • Clinical Stage: Phases 1 and 2
      • Forecast
        • TABLE 74: GLOBAL MARKET FOR NICORD, THROUGH 2022 ($ MILLIONS/%)
    • Olmutinib (Boehringer Ingelheim)
      • BTD: December 21, 2015
      • Drug Class: Fully Human Monoclonal Antibody Targeting Programmed Death Ligand-1 (Biologic)
      • Indication: T790M Mutation NSCLC (Oncology)
      • Clinical Stage: Phases 1 and 2 (Approved in South Korea)
      • Forecast
        • TABLE 75: GLOBAL MARKET FOR OLMUTINIB, THROUGH 2022 ($ MILLIONS/%)
    • NY-ESO Peptide (Affinity Enhanced T-Cell Therapy; Adaptimmune)
      • BTD: February 9, 2016
      • Drug class: T-cell Therapy (TCR; Biologic)
      • Indication: Synovial Sarcoma (Oncology)
      • Clinical Stage: Phases 1 and 2
      • Forecast
        • TABLE 76: GLOBAL MARKET FOR NY-ESO, THROUGH 2022 ($ MILLIONS/%)
    • Pexidartinib (Daiichi Sankyo/Plexxikon)
      • BTD: October 30, 2015
      • Drug Class: Multitargeted Receptor Tyrosine Kinase (RTK) Inhibitor (Small Molecule)
      • Indication: Tenosynovial Giant Cell Tumor (TGCT; Oncology)
      • Clinical Stage: Phase 1
      • Forecast
        • TABLE 77: GLOBAL MARKET FOR PEXIDARTINIB, THROUGH 2022 ($ MILLIONS/%)
    • Pracinostat (MEI Pharma)
      • BTD: August 1, 2016
      • Drug Class: HDAC Inhibitor (Small Molecule)
      • Indication: AML (Oncology)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 78: GLOBAL MARKET FOR PRACINOSTAT, THROUGH 2022 ($ MILLIONS/%)
    • PVS-RIPO (Duke Medicine)
      • BTD: May 16, 2016
      • Drug Class: Genetically Modified Poliovirus (Biologic)
      • Indication: Glioblastoma (Oncology)
      • Clinical Stage: Phase 1
      • Forecast
        • TABLE 79: GLOBAL MARKET FOR PVS-RIPO, THROUGH 2022 ($ MILLIONS/%)
    • Ribociclib (LEE011; Novartis)
      • BTD: August 3, 2016
      • Indication: Her2 Metastatic Breast Cancer (Oncology)
      • Drug Class: CDK4-6 Inhibitor (Small Molecule)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 80: GLOBAL MARKET FOR RIBOCICLIB, THROUGH 2022 ($ MILLIONS/%)
    • Rintega (Celldex)
      • BTD: February 23, 2015
      • Indication: Glioblastoma (Oncology)
      • Drug class: Cancer Vaccine (Biologic)
      • Clinical Stage: Discontinued
      • Forecast
        • TABLE 81: GLOBAL MARKET FOR RINTEGA, THROUGH 2022 ($ MILLIONS/%)
    • Rociletinib (Clovis Oncology)
      • BTD: August 3, 2016
      • Indication: Her2 Metastatic Breast Cancer (Oncology)
      • Drug Class: Epidermal Growth Factor Receptor Inhibitor (Small Molecule)
      • Indication: Non-small Cell Lung Cancer (Oncology)
      • Clinical Stage: Discontinued
      • Forecast
        • TABLE 82: GLOBAL MARKET FOR ROCILETINIB, THROUGH 2022 ($ MILLIONS/%)
    • Rucaparib (Clovis Pharmaceuticals)
      • BTD: April 2015
      • Drug Class: Oral PARP Inhibitor (PARP; Small Molecule)
      • Indication: Ovarian Cancer (Oncology)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 83: GLOBAL MARKET FOR RUCAPARIB, THROUGH 2022 ($ MILLIONS/%)
    • Sacituzumab Govitecan (IMMU-132; Immunomedics)
      • BTD: February 8, 2016
      • Drug Class: Antibody-Drug Conjugate (ADC; Biologic)
      • Indication: Triple-Negative Breast Cancer (TNBC; Oncology)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 84: GLOBAL MARKET FOR SACITUZUMAB GOVITECAN, THROUGH 2022 ($ Millions/%)
    • Volasertib (Boehringer Ingelheim)
      • BTD: September 17, 2013
      • Drug Class: Polo-Like Kinase (Plk) Inhibitor (Biologic)
      • Indication: Acute Myeloid Leukemia (AML)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 85: GLOBAL MARKET FOR VOLASERTIB, THROUGH 2022 ($ MILLIONS/%)
    • Vyxeos (Cpx-351; Celator Pharmaceuticals-Jazz Pharmaceuticals)
      • BTD: May 19, 2016
      • Drug Class: Nano-Scale Liposomal Formulation of Cytarabine: Daunorubicin (Small Molecule)
      • Indication: AML
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 86: GLOBAL MARKET FOR VYXEOS, THROUGH 2022 ($ MILLIONS/%)
  • RARE DISEASES
    • FIGURE 14 RARE DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
  • RARE DISEASES - BTD APPROVED
    • TABLE 87: FDA APPROVED RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015-2016
    • TABLE 88: GLOBAL MARKET FOR RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • Ilaris (Canak Inumab, Novartis)
      • BTD: April 27, 2016
      • Drug Class: Human Monoclonal Antibody Targeting Interleukin-
      • Beta (Biologic)
      • Indication: PFS, TRAPS and FMF (Rare Disease)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 89: GLOBAL MARKET FOR ILARIS, THROUGH 2022 ($ MILLIONS/%)
    • Kanuma (Sebelipase Alfa; Alexion Pharmaceuticals)
      • BTD May 20, 2013
      • Drug Class: Enzyme Replacement Therapy (Biologic)
      • Indication: Lysosomal Acid Lipase Deficiency (Rare Disease)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 90: GLOBAL MARKET FOR KANUMA, THROUGH 2022 ($ MILLIONS/%)
    • Strensiq (Asfotase Alfa; Alexion Pharmaceuticals)
      • BTD: May 28, 2013
      • Drug Class: Enzyme Replacement Therapy (Biologic)
      • Indication: Hypophosphatasia (Rare Disease)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 91: GLOBAL MARKET FOR STRENSIQ, THROUGH 2022 ($ MILLIONS/%)
  • RARE DISEASES - BTD IN DEVELOPMENT
    • TABLE 92: UNDER DEVELOPMENT RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2016
    • ALXN1101 (Alexion Pharmaceuticals)
      • BTD: October 24, 2013
      • Drug Class: Enzyme Co-Factor Replacement Therapy (Biologic)
      • Indication: Molybdenum Cofactor Deficiency (MoCD; Rare Diseases)
      • Clinical Stage: Phase 2 and Phase 3
      • Forecast
        • TABLE 93: GLOBAL MARKET FOR ALXN-1101, THROUGH 2022 ($ MILLIONS/%)
    • Firdapse (Amifampridine; Catalyst Pharmaceuticals)
      • BTD: February 22, 2016
      • Drug Class: Potassium channel blocker (Small Molecule)
      • Indication: Lambert-Eaton Myasthenic Syndrome (LEMS; Rare Disease)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 94: GLOBAL MARKET FOR FIRDAPSE, THROUGH 2022 ($ MILLIONS/%)
    • Olipudase Alfa (Sanofi Genzyme)
      • BTD: June 4, 2014
      • Drug Class: Enzyme Replacement Therapy (Biologic)
      • Indication: Acid Sphingomyelinase Deficiency-Niemann-Pick Disease Type B
      • Clinical Stage: Phase 2 and Phase 3
      • Forecast
        • TABLE 95: GLOBAL MARKET FOR OLIPUDASE ALFA, THROUGH 2022 ($ MILLIONS/%)
    • Zorblisa (SD-101; -Amicus Therapeutics /Scioderm
      • BTD: April 23, 2013
      • Drug Class: Topical Cream (Small Molecule)
      • Indication: Epidermolysis Bullosa (Rare Disease)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 96: GLOBAL MARKET FOR ZORBLISA, THROUGH 2022 ($ MILLIONS/%)
    • Setmelanotide (RM-493; Rhythm Pharmaceuticals)
      • BTD: January 7, 2016
      • Drug Class: Melanocortin-4 Receptor (MC4R) Agonist (Small Molecule)
      • Indication: Pro-Opiomelanocortin (POMC) Deficiency Obesity (Rare Diseases)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 97: GLOBAL MARKET FOR SETMELANOTIDE, THROUGH 2022 ($ MILLIONS)
    • VTS-270 (Vtesse Pharmaceuticals)
      • BTD: January 6, 2016
      • Drug Class: 2-Hydroxypropyl-B-Cyclodextrin (Small Molecule)
      • Indication: Niemann-Pick Type C1 (NPC) Disease (Rare Disease)
      • Clinical Stage: Phase 2 and Phase 3
      • Forecast
        • TABLE 98: GLOBAL MARKET FOR VTS-270, THROUGH 2022 ($ MILLIONS/%)
  • OTHER DISEASES
    • FIGURE 15 OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
  • OTHER - BTD APPROVED
    • TABLE 99: FDA APPROVED OTHER BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015
    • TABLE 100: GLOBAL MARKET FOR OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • Rapamune (Sirolimus, Pfizer)
      • BTD Date: May 28, 2015
      • Drug Class: Macrolide Lactam Immunosuppressant (Small Molecule)
      • Indication: Lymphangioleiomyomatosis (LAM)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 101: GLOBAL MARKET FOR RAPAMUNE BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • Lucentis (Ranibizumab Genentech-Roche)
      • BTD Date: December 15, 2014
      • Drug Class: (EGF Inhibitor (Biologic)
      • Indication Diabetic Retinopathy (DR)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 102: GLOBAL MARKET FOR LUCENTIS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • Eylea (Aflibercept; Bayer)
      • BTD Date: September 16, 2014
      • Drug Class: VEGF Fusion Protein (Biologic)
      • Indication Diabetic Retinopathy (DR)
      • Clinical Stage: Approved
      • Forecast
        • TABLE 103: GLOBAL MARKET FOR EYLEA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
  • OTHER DISEASES - BTD IN DEVELOPMENT
    • TABLE 104: OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS AWAITING APPROVAL, 2015-2022
    • Dupilumab (Sanofi-Regeneron)
      • BTD Date: November 20, 2014
      • Drug Class: IL-4 mAb (Biologic)
      • Indication: Severe Atopic Dermatitis
      • Clinical Stage: Filed
      • Forecast
        • TABLE 105: GLOBAL MARKET FOR DUPILUMAB BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • AR101 (Aimmune Therapeutics)
      • BTD Date: June 18, 2015
      • Indication: Peanut Allergen (Small Molecule)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 106: GLOBAL MARKET FOR AR101 BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • Ocaliva (Obeticholic acid; Intercept Pharmaceuticals)
      • BTD Date: January 1, 2015
      • Drug Class: Farnesoid X Receptor Agonist (Small Molecule)
      • Indication: Nonalcoholic Steatohepatitis (NASH) with Liver Fibrosis (Rare Disease)
      • Clinical Stage: Phase 3
      • Forecast
        • TABLE 107: GLOBAL MARKET FOR OCALIVA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • Viaskin Peanut (DBV Technologies)
      • BTD: April 9, 2015
      • Drug Class: Peanut Allergen (Small Molecule)
      • Indication: Peanut Allergen (Small Molecule)
      • Clinical Stage: Phase 2
      • Forecast
        • TABLE 108: GLOBAL MARKET FOR VIASKIN PEANUT BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)

CHAPTER 5 - IMPLICATIONS OF BREAKTHROUGH THERAPY DESIGNATION ON BUSINESS STRATEGY

  • INTRODUCTION
    • FIGURE 16: COMPARISON OF APPROVAL TIMES STANDARD VERSUS BREAKTHROUGH DESIGNATION (MONTHS)
  • IMPLICATIONS FOR CLINICAL DEVELOPMENT
  • STRATEGIES FOR EXPEDITING CLINICAL DEVELOPMENT
    • Process and Formulation Development
    • Process Validation
    • Analytical Development
    • Availability of Stability Data
  • IMPLICATIONS FOR MANUFACTURING
    • Companion Diagnostics
  • STRATEGIES FOR EXPEDITING MANUFACTURING CAPABILITIES
  • MANAGING TECHNICAL RISKS VERSUS PATIENT BENEFIT
  • LOOKING AHEAD

CHAPTER 6 - REGULATORY IMPLICATIONS AND BREAKTHROUGH THERAPY DESIGNATION

  • EXPEDITED PROGRAMS
    • TABLE 109: AN OVERVIEW OF THE KEY FEATURES OF THE FDA'S EXPEDITED PROGRAMS
  • REGULATORY PATHWAYS
    • TABLE 110: AN OVERVIEW OF THE FDA'S EXPEDITED DEVELOPMENT AND REVIEW PROGRAMS
  • FAST TRACK DESIGNATION
    • Qualifying Criteria
      • Serious Condition
      • Demonstrates the Potential to Address Unmet Medical Need
    • Key Features of Fast Track Designation
  • BREAKTHROUGH THERAPY DESIGNATION
    • Qualifying Criteria
    • Key Features
      • Intensive Guidance on an Efficient Drug Development Program, Beginning as early as Phase 1
        • TABLE 111: BREAKTHROUGH THERAPIES CONSIDERATIONS: DRUG DEVELOPMENT
      • Organizational Commitment Involving Senior Managers
      • Submission of Portions of an Application (Rolling Review)
        • TABLE 112: BREAKTHROUGH THERAPY CONSIDERATIONS: MARKETING APPLICATION REVIEW
      • Other Actions to Expedite Review
    • When to Make a BTD Request Submission
    • The BTD Review Process
      • FIGURE 17: COMPARISON OF STANDARD AND BREAKTHROUGH THERAPY APPROVAL TIMES (MONTHS)
    • Rescinding BTD Status
  • ACCELERATED APPROVAL
    • Qualifying Criteria
  • ACCELERATED APPROVAL ENDPOINTS
    • Conditions of Accelerated Approval
  • PRIORITY REVIEW DESIGNATION
    • Qualifying Criteria
    • Key Features
  • DRUGS GRANTED BREAKTHROUGH THERAPY DESIGNATION
    • TABLE 113: CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-SEPTEMBER 30, 2015
    • TABLE 114: CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: OCTOBER 1, 2015-SEPTEMBER 30, 2016
    • TABLE 115: CDER BREAKTHROUGH THERAPY DESIGNATION APPROVALS DATA AS OF DECEMBER 31, 2015
    • TABLE 116: CUMULATIVE CBER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-DECEMBER 31, 2015
    • TABLE 117: CBER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: OCTOBER 1, 2015-SEPTEMBER 30, 2016
    • TABLE 118: CUMULATIVE CBER BREAKTHROUGH THERAPY APPROVAL DATA AS OF SEPTEMBER 30, 2016
    • TABLE 119: MOST RECENTLY ANNOUNCED BREAKTHROUGH THERAPY DESIGNATIONS DATA AS OF FEBRUARY 29, 2016
  • SIGNIFICANCE OF BTD FOR THE PHARMACEUTICAL AND BIOTECHNOLOGY INDUSTRIES

CHAPTER 7 - COMPANY PROFILES

  • ABBVIE INC.
  • ACADIA PHARMACEUTICALS INC.
  • ADAPTIMMUNE THERAPEUTICS PLC
  • ADURO BIOTECH INC.
  • AIMMUNE THERAPEUTICS INC.
  • ALEXION PHARMACEUTICALS INC.
  • ALLERGAN PLC
  • AMGEN INC.
  • ANATARA LIFESCIENCES LIMITED
  • ARIAD PHARMACEUTICALS INC.
  • ASTRAZENECA PLC
  • AVEXIS INC.
  • BOEHRINGER INGELHEIM GMBH
  • CELLDEX THERAPEUTICS INC.
  • CLOVIS ONCOLOGY INC.
  • DAIICHI SANKYO COMPANY, LIMITED
  • DBV TECHNOLOGIES
  • EISAI CO. LTD.
  • ELI LILLY AND COMPANY
  • EXELIXIS INC.
  • F. HOFFMANN-LA ROCHE LTD.
  • GILEAD SCIENCES INC.
  • GLAXOSMITHKLINE PLC
  • IMMUNOMEDICS INC.
  • INDIVIDOR PLC
  • JAZZ PHARMACEUTICALS
  • JOHNSON & JOHNSON
  • JUNO THERAPEUTICS INC.
  • KITE PHARMA INC.
  • MERCK & CO. INC.
  • MERCK KGAA
  • NOVARTIS AG
  • PFIZER INC.
  • PHARMACYCLICS INC.
  • PROGENICS PHARMACEUTICALS INC.
  • REGENERON PHARMACEUTICALS INC.
  • RHYTHM PHARMACEUTICALS INC.
  • SYNDAX PHARMACEUTICALS INC.
  • SYNEURX INTERNATIONAL CORPORATION
  • TEVA PHARMACEUTICALS INC.
  • TREVENA INC.
  • VERTEX PHARMACEUTICALS INC.
  • VTESSE INC.
  • WELLSTAT THERAPEUTICS CORPORATION

CHAPTER 8 - REFERENCES

  • CHAPTER 3
  • CHAPTER 4
  • CHAPTER 5
  • CHAPTER 6

CHAPTER 9 - ACRONYMS

CHAPTER 10 - ENDNOTES

LIST OF TABLES

  • SUMMARY TABLE: GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, THROUGH 2022 ($ MILLIONS)
    • TABLE 1: CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-SEPTEMBER 30, 2015
    • TABLE 2: CBER BREAKTHROUGH THERAPY REQUESTS FOR FINAL YEAR, OCTOBER 1, 2014--SEPTEMBER 30, 2015
    • TABLE 3: DRUGS GRANTED PRIME DESIGNATIONS, MARCH 7, 2016-AUGUST 24, 2016
    • TABLE 4: GLOBAL MARKET FOR CV BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 5: FDA APPROVED CV BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • TABLE 6: GLOBAL MARKET FOR PROMACTA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 7: GLOBAL MARKET FOR PRAXBIND, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 8: UNDER DEVELOPMENT CV BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • TABLE 9: GLOBAL MARKET FOR REASANZ, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 10: GLOBAL MARKET FOR ACTEMRA/ROACTEMRA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 11: GLOBAL MARKET FOR CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 12: FDA APPROVED CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • TABLE 13: GLOBAL MARKET FOR NUPLAZID, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 14: UNDER DEVELOPMENT CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • TABLE 15: GLOBAL MARKET FOR DEUTETRABENAZINE ER, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 16: GLOBAL MARKET FOR INGREZZA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 17: GLOBAL MARKET FOR OF OFATUMUMAB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 18: GLOBAL MARKET FOR OLICERIDINE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 19: GLOBAL MARKET FOR RAPASTINEL, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 20: GLOBAL MARKET FOR SEPRANOLONE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 21: GLOBAL MARKET FOR SND-13, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 22: GLOBAL MARKET FOR AVXS-101, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 23: GLOBAL MARKET FOR ESKETAMINE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 24: GLOBAL MARKET FOR RBP-8000, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 25: GLOBAL MARKET FOR INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 26: FDA APPROVED INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
    • TABLE 27: GLOBAL MARKET FOR SOLVADI, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 28: GLOBAL MARKET FOR EPCLUSA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 29: GLOBAL MARKET FOR VIEKERA/TECHNIVIE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 30: GLOBAL MARKET FOR ZEPATIER, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 31: GLOBAL MARKET FOR HARVONI, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 32: UNDER DEVELOPMENT INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
    • TABLE 33: GLOBAL MARKET FOR ARIKAYCE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 34: GLOBAL MARKET FOR FOSTEMSAVIR, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 35: GLOBAL MARKET FOR GLECAPREVIR/PIBRENTASVIR, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 36: GLOBAL MARKET FOR IBALIZUMAB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 37: GLOBAL MARKET FOR TAFENOQUINE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 38: GLOBAL MARKET FOR V920, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 39: GLOBAL MARKET FOR ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 40: FDA APPROVED ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2015
    • TABLE 41: GLOBAL MARKET FOR ALENCENSA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 42: GLOBAL MARKET FOR ARZERRA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 43: GLOBAL MARKET FOR BLINCYTO, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 44: GLOBAL MARKET FOR DARZALEX, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 45: GLOBAL MARKET FOR GAZYVA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 46: GLOBAL MARKET FOR IBRANCE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 47: GLOBAL MARKET FOR IMBRUVICA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 48: GLOBAL MARKET FOR KEYTRUDA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 49: GLOBAL MARKET FOR LARTRUVO, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 50: GLOBAL MARKET FOR LENVIMA/KISPLYX, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 51: GLOBAL MARKET FOR LYNPARZA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 52: GLOBAL MARKET FOR MIDOSTAURIN, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 53: GLOBAL MARKET FOR OPDIVO, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 54: GLOBAL MARKET FOR TAGRISSO, SALES FORECASTS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 55: GLOBAL MARKET FOR TAFINLAR/MEKINIST, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 56: GLOBAL MARKET FOR TECENTRIQ, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 57: GLOBAL MARKET FOR VENCLEXTA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 58: GLOBAL MARKET FOR XALKORI, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 59: GLOBAL MARKET FOR ZYDELIG, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 60: GLOBAL MARKET FOR ZYKADIA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 61: UNDER DEVELOPMENT ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS
    • TABLE 62: GLOBAL MARKET FOR ABEMACICLIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 63: GLOBAL MARKET FOR AVELUMAB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 64: GLOBAL MARKET FOR BRIGATINIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 65: GLOBAL MARKET FOR CTL019, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 66: GLOBAL MARKET FOR CRS-207 AND GVAX PANCREAS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 67: GLOBAL MARKET FOR DURVALUMAB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 68: GLOBAL MARKET FOR EBV-CTL, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 69: GLOBAL MARKET FOR ENTINOSTAT, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 70: GLOBAL MARKET FOR INOTUZUMAB OZOGAMICIN, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 71: GLOBAL MARKET FOR JCAR015, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 72: GLOBAL MARKET FOR KTE-C19, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 73: GLOBAL MARKET FOR LOXO-101, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 74: GLOBAL MARKET FOR NICORD, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 75: GLOBAL MARKET FOR OLMUTINIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 76: GLOBAL MARKET FOR NY-ESO, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 77: GLOBAL MARKET FOR PEXIDARTINIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 78: GLOBAL MARKET FOR PRACINOSTAT, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 79: GLOBAL MARKET FOR PVS-RIPO, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 80: GLOBAL MARKET FOR RIBOCICLIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 81: GLOBAL MARKET FOR RINTEGA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 82: GLOBAL MARKET FOR ROCILETINIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 83: GLOBAL MARKET FOR RUCAPARIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 84: GLOBAL MARKET FOR SACITUZUMAB GOVITECAN, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 85: GLOBAL MARKET FOR VOLASERTIB, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 86: GLOBAL MARKET FOR VYXEOS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 87: FDA APPROVED RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015-2016
    • TABLE 88: GLOBAL MARKET FOR RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 89: GLOBAL MARKET FOR ILARIS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 90: GLOBAL MARKET FOR KANUMA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 91: GLOBAL MARKET FOR STRENSIQ, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 92: UNDER DEVELOPMENT RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2016
    • TABLE 93: GLOBAL MARKET FOR ALXN-1101, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 94: GLOBAL MARKET FOR FIRDAPSE, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 95: GLOBAL MARKET FOR OLIPUDASE ALFA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 96: GLOBAL MARKET FOR ZORBLISA, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 97: GLOBAL MARKET FOR SETMELANOTIDE, THROUGH 2022 ($ MILLIONS)
    • TABLE 98: GLOBAL MARKET FOR VTS-270, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 99: FDA APPROVED OTHER BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015
    • TABLE 100: GLOBAL MARKET FOR OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 101: GLOBAL MARKET FOR RAPAMUNE BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 102: GLOBAL MARKET FOR LUCENTIS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 103: GLOBAL MARKET FOR EYLEA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 104: OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS AWAITING APPROVAL, 2015-2022
    • TABLE 105: GLOBAL MARKET FOR DUPILUMAB BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 106: GLOBAL MARKET FOR AR101 BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 107: GLOBAL MARKET FOR OCALIVA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 108: GLOBAL MARKET FOR VIASKIN PEANUT BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
    • TABLE 109: AN OVERVIEW OF THE KEY FEATURES OF THE FDA'S EXPEDITED PROGRAMS
    • TABLE 110: AN OVERVIEW OF THE FDA'S EXPEDITED DEVELOPMENT AND REVIEW PROGRAMS
    • TABLE 111: BREAKTHROUGH THERAPIES CONSIDERATIONS: DRUG DEVELOPMENT
    • TABLE 112: BREAKTHROUGH THERAPY CONSIDERATIONS: MARKETING APPLICATION REVIEW
    • TABLE 113: CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-SEPTEMBER 30, 2015
    • TABLE 114: CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: OCTOBER 1, 2015-SEPTEMBER 30, 2016
    • TABLE 115: CDER BREAKTHROUGH THERAPY DESIGNATION APPROVALS DATA AS OF DECEMBER 31, 2015
    • TABLE 116: CUMULATIVE CBER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-DECEMBER 31, 2015
    • TABLE 117: CBER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: OCTOBER 1, 2015-SEPTEMBER 30, 2016
    • TABLE 118: CUMULATIVE CBER BREAKTHROUGH THERAPY APPROVAL DATA AS OF SEPTEMBER 30, 2016
    • TABLE 119: MOST RECENTLY ANNOUNCED BREAKTHROUGH THERAPY DESIGNATIONS DATA AS OF FEBRUARY 29, 2016

LIST OF FIGURES

  • SUMMARY FIGURE: GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, 2015-2022 ($ MILLIONS)
    • FIGURE 1: BREAKTHROUGH THERAPY REQUESTS GRANTED BY DIVISION, 2015* (%)
    • FIGURE 2: PRIME SCHEME APPLICATIONS AND ELIGIBILITY
    • FIGURE 3: PRIME DRUG BY TYPE OF APPLICATION AND THERAPEUTIC AREA
    • FIGURE 4: PRIME DRUG APPLICATIONS, THERAPEUTIC AREA
    • FIGURE 5: FDA APPROVALS, BREAKTHROUGH THERAPY DESIGNATION AND DESIGNATION RESCINDED, BY STATUS, 2012-2016 (NUMBER)
    • FIGURE 6: FDA APPROVALS, BREAKTHROUGH DESIGNATION AND DESIGNATION RESCINDED, BY THERAPY AREA, 2012-2016 (NUMBER)
    • FIGURE 7: CARDIOVASCULAR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE (NUMBER)
    • FIGURE 8: CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
    • FIGURE 9: INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
    • FIGURE 10: CANCER DRUGS WITH BREAKTHROUGH THERAPY DESIGNATION, BY CANCER TYPE, 2016 (NUMBER OF APPROVED BTD DRUGS)
    • FIGURE 11: CANCER BREAKTHROUGH THERAPY DESIGNATION, BY COMPANY, 2016 (NO. OF BTD IN ONCOLOGY)
    • FIGURE 12: TREATMENT ALGORITHM FOR ALL PATIENTS
    • FIGURE 13: ENTINOSTAT MECHANISM OF ACTION
    • FIGURE 14: RARE DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
    • FIGURE 15: OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
    • FIGURE 16: COMPARISON OF APPROVAL TIMES STANDARD VERSUS BREAKTHROUGH DESIGNATION (MONTHS)
    • FIGURE 17: COMPARISON OF STANDARD AND BREAKTHROUGH THERAPY APPROVAL TIMES (MONTHS)
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