US Orphan Cancer Drug Market Size, Drug Pricing, Sales, Market Forecast, Competitive Landscape & Clinical Trials Insight 2031

List of Tables

US Orphan Cancer Drug Market Size, Drug Pricing, Sales, Market Forecast, Competitive Landscape & Clinical Trials Insight 2031 Report Findings & Highlights:

US Orphan Cancer Drugs Market Opportunity: > USD 100 Billion
Insight On Orphan Cancer Drugs In Pipeline: > 400 Drugs Profiles
Clinical Trials Insight By Company, Indication, Priority Status & Phase
Clinical Insight On Marketed Orphan Cancer Drugs: > 200 Drugs Profiles
Pricing, Dosage & Treatment Cost Insight: > 140 Marketed Orphan Drugs
FDA Designated Orphan Drugs Sales Insight (2021 - 2026): > 60 Orphan Drugs
Orphan Designated Drugs Reimbursement Scenario
Competitive Landscape: 40 Companies Profiles

Need For Orphan Designated Cancer Drugs & Why This Report?

The US market for FDA orphan designated cancer drugs is gaining importance as pharmaceutical and biotechnology companies increasingly invest in treatments for rare malignancies and small, molecularly defined patient populations. Many rare cancers, including acute myeloid leukemia (AML), malignant brain tumors, hepatocellular carcinoma (HCC), and certain pediatric cancers, have limited treatment options and present challenges for conventional drug development because of their relatively small patient populations. The FDA's Orphan Drug Designation (ODD) program addresses some of these challenges by providing incentives for developing therapies targeting diseases affecting fewer than 200,000 people in the US. These incentives include potential tax credits for eligible clinical testing, exemptions from certain application fees, and seven years of market exclusivity following approval for the designated indication.

The increasing number of orphan designated cancer drug candidates reflects the growing focus on rare cancer indications, innovative treatment mechanisms and unmet clinical needs. Developers are exploring targeted therapies, bispecific antibodies, gene therapies, radiopharmaceuticals, and immunotherapies to address cancers that may not attract sufficient investment through conventional commercial development models.

This report examines the regulatory landscape, clinical development activity, competitive environment, and emerging commercial opportunities in the US FDA orphan designated cancer drug market.

Clinical Trials Insight Included In Report

Clinical development in the US orphan designated cancer drug market spans multiple therapeutic areas, including hematological malignancies and solid tumors, with investigational therapies targeting acute myeloid leukemia (AML), pediatric brain tumors, hepatocellular carcinoma, and other rare cancers. Development approaches include bispecific antibodies, radiopharmaceuticals, RNA based gene therapies, targeted inhibitors, and immunotherapies. Recent examples include Cullinan Therapeutics' orphan designated AML candidate and Plus Therapeutics' investigational radiotherapeutic for pediatric malignant gliomas.

The report covers the orphan designated cancer drug pipeline across preclinical, Phase I, Phase II, and Phase III development stages, highlighting key companies, investigational candidates, mechanisms of action, target indications, and clinical progress. It also examines the involvement of technology providers, research institutions, and strategic collaborators, alongside clinical trial geographies, regulatory designations, licensing agreements, and partnerships. Particular attention is given to emerging therapeutic platforms, company-specific development strategies, and trends shaping the future of orphan-designated cancer drug development in the US.

Major Companies Driving The US FDA Orphan Designated Cancer Drug Market

The US orphan designated cancer drug market includes emerging biotechnology companies, established pharmaceutical organizations, and academic research institutions. Smaller biotechnology developers are contributing to innovation through early-stage research and the development of therapies for rare malignancies, while larger companies provide resources for clinical development and commercialization.

Recent FDA designation announcements highlight the growing participation of smaller developers. In May 2026, Roswell Park spin-off Canget BioTekpharma received Orphan Drug and Rare Pediatric Disease designations for an osteosarcoma treatment candidate. Similarly, RenovoRx received FDA Orphan Drug Designation for a pancreatic cancer indication in May 2026. These developments illustrate how biotechnology companies are expanding their oncology pipelines by pursuing regulatory incentives for rare cancer indications.

Future Outlook For The US FDA Orphan Designated Cancer Drug Market

The future of the US orphan designated cancer drug market is expected to be shaped by advances in precision oncology, immunotherapy, gene therapy, and radiopharmaceuticals. Increasing research into rare molecular subtypes of common cancers is likely to create additional opportunities for orphan drug designation, particularly for therapies targeting specific genetic alterations and cancers with limited treatment options. Recent FDA approvals, such as AbbVie's pivekimab sunirine for blastic plasmacytoid dendritic cell neoplasm (BPDCN) in May 2026, demonstrate the potential transition of orphan-designated candidates into commercially available treatments.

Furthermore, strategic acquisitions, licensing agreements, and research collaborations are expected to support pipeline expansion and accelerate clinical development. Advances in biomarker discovery, molecular diagnostics, and personalized medicine may also improve patient selection and clinical trial outcomes. Future market growth will depend on clinical success, regulatory approvals, reimbursement opportunities, and the ability of companies to translate innovative research into effective treatments for rare cancer populations.

1. Research Methodology

2. FDA Orphan Designation Criteria & Market Exclusivity

3. US Orphan Designated Cancer Drugs Market Insight

3.1 Recent Research & Development
3.2 Current R&D and Market Overview
3.3 Future Market Opportunity Assessment

4. US Orphan Designated Cancer Market Trends & Clinical Innovation By Indications

4.1 Leukemia
4.2 Lymphoma
4.3 Multiple Myeloma
4.4 Brain & CNS Cancers
4.5 Sarcomas & Gastrointestinal Stromal Tumors
4.6 Lung & Thoracic Cancers
4.7 Skin Cancer
4.8 Gastrointestinal, Hepatobiliary & Pancreatic Cancers
4.9 Genitourinary & Gynecological Cancers
4.10 Head & Neck, Endocrine & Other Rare Solid Tumors

5. US Orphan Designated Drugs Reimbursement Scenario

5.1 Medicare
5.2 Medicaid
5.3 Private Insurers
5.4 Pharmaceutical Companies

6. US Orphan Designated Cancer Drugs Clinical Trials By Company, Indication & Phase

6.1 Research
6.2 Preclinical
6.3 Phase-I
6.4 Phase-I/II
6.5 Phase-II
6.6 Phase-II/III
6.7 Phase-III
6.8 Preregistration
6.9 Registered

7. Marketed Orphan Designated Cancer Drugs Clinical Insight By Company & Indication

8. US Orphan Designated Cancer Drugs Dosage, Price & Treatment Cost Insight

This Chapter Gives Insight On 144 Orphan Cancer Designated Drugs Dosage, Price & Treatment Cost Insight

8.1 Lynparza
8.2 Tafinlar
8.3 Halaven
8.4 Xpovio
8.5 Gavreto
8.6 Lumakras
8.7 Monjuvi

8.141 Xolremdi
8.142 Ojemda
8.143 Ojjaara
8.144 Ogsiveo

9. US Orphan Designated Cancer Drugs Sales Insight (2021-2026)

This Chapter Gives Insight On 67 Orphan Cancer Designated Drugs Sales Insight

9.1 Krazati
9.2 Tecvayli
9.3 Imjudo
9.4 Elrexfio
9.5 Jaypirca

9.62 Poteligeo
9.63 Xospata
9.64 Yescarta
9.65 Lunsumio
9.66 Keytruda
9.67 Pemazyre

10. Competitive Landscape

10.1 AbbVie
10.2 Amgen
10.3 Astellas Pharma
10.4 AstraZeneca
10.5 Autolus Therapeutics
10.6 Bayer HealthCare
10.7 Boehringer Ingelheim
10.8 Bristol-Myers Squibb
10.9 Daiichi Sankyo Company
10.10 Eisai Co Ltd
10.11 Eli Lilly & Company
10.12 Flavocure Biotech
10.13 Genentech
10.14 Genmab
10.15 Gilead Sciences
10.16 GSK
10.17 Incyte Corporation
10.18 Ipsen
10.19 Jazz Pharmaceuticals
10.20 Johnson & Johnson
10.21 Kamada
10.22 Krystal Biotech
10.23 Kyowa Kirin
10.24 Leadiant Biosciences
10.25 Ligand Pharmaceuticals
10.26 Merck
10.27 Novartis
10.28 Otsuka Pharmaceutical
10.29 Pfizer
10.30 PTC Therapeutics
10.31 Regeneron Pharmaceuticals
10.32 Roche
10.33 Sanofi
10.34 Servier
10.35 Sumitomo Pharma
10.36 Sutro Biopharma
10.37 Swedish Orphan Biovitrum
10.38 Takeda
10.39 Teva Pharmaceutical Industries
10.40 TG Therapeutics

List of Figures

Figure 3-1: US - Orphan Designated Drugs Market Size (US$ Billion), 2024 - 2031
Figure 3-2: US - Orphan Designated Cancer Drugs Market Size (US$ Billion), 2024 - 2031
Figure 4-1: Leukemia - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-2: CLN-049-001 Phase I Study (NCT05143996) - Initiation & Completion Year
Figure 4-3: CLN-049-AML-103 Phase I Study (NCT07722767) - Initiation & Completion Year
Figure 4-4: HORIZEN-1 Phase I/II Study (NCT04988555) - Initiation & Completion Year
Figure 4-5: HORIZEN-1 Phase I/II Study (NCT04988555) - Initiation & Completion Year
Figure 4-6: BRUIN-CLL-313 Phase III Study (NCT05023980) - Initiation & Completion Year
Figure 4-7: Lymphoma - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-8: TRANSCEND FL Phase II Study (NCT04245839) - Initiation & Completion Year
Figure 4-9: SWOG 1826 Phase III Study (NCT03907488) - Initiation & Completion Year
Figure 4-10: BGB-11417-201 Phase I/II Study (NCT05471843) - Initiation & Completion Year
Figure 4-11: CELESTIAL-RRMCL Phase III Study (NCT06742996) - Initiation & Completion Year
Figure 4-12: TENACITY-01 Phase I/II Study (NCT03907488) - Initiation & Completion Year
Figure 4-13: VIPER101 Phase I Study (NCT06420089) - Initiation & Completion Year
Figure 4-14: SOUNDTRACK-F1 Phase III Study (NCT06549595) - Initiation & Completion Year
Figure 4-15: SOUNDTRACK-B Phase II Study (NCT06526793) - Initiation & Completion Year
Figure 4-16: Multiple Myeloma - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-17: EXCALIBER-RRMM Phase III Study (NCT04975997) - Initiation & Completion Year
Figure 4-18: MajesTEC-3 Phase III Study (NCT05083169) - Initiation & Completion Year
Figure 4-19: CIBI3003A101 Phase I/II Study (NCT06083207) - Initiation & Completion Year
Figure 4-20: CIBI3003A101EX Phase I/II Study (NCT07336472) - Initiation & Completion Year
Figure 4-21: TriadicMM-1 Phase III Study (NCT07623798) - Initiation & Completion Year
Figure 4-22: IBI3003A201 Phase II Study (NCT07764861) - Initiation & Completion Year
Figure 4-23: ARC-101-ARM1 Phase I Study (NCT04155749) - Initiation & Completion Year
Figure 4-24: iMMagine-1 Phase II Study (NCT05396885) - Initiation & Completion Year
Figure 4-25: Brain & CNS Cancers - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-26: OPTICAL Phase II Study (NCT07356973) - Initiation & Completion Year
Figure 4-27: GLIOMAX-101 Phase II Study (NCT07386002) - Initiation & Completion Year
Figure 4-28: ReSPECT-PBC Phase I/II Study (NCT07061626) - Initiation & Completion Year
Figure 4-29: Sarcomas & Gastrointestinal Stromal Tumors - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-30: ARTEMIS-011 Phase III Study (NCT06935409) - Initiation & Completion Year
Figure 4-31: EMBOLD Sarcoma-202 Phase I/II Study (NCT07602777) - Initiation & Completion Year
Figure 4-32: LP-284-001 Phase I Study (NCT06132503) - Initiation & Completion Year
Figure 4-33: EFTISARC-NEO Phase II Study (NCT06128863) - Initiation & Completion Year
Figure 4-34: ChonDRAgon Phase II Study (NCT04950075) - Initiation & Completion Year
Figure 4-35: Lung & thoracic cancers - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-36: PRESERVE-003 Phase III Study (NCT05671510) - Initiation & Completion Year
Figure 4-37: TEADES Phase I/II Study (NCT06526793) - Initiation & Completion Year
Figure 4-38: TEADCO Phase I/II Study (NCT07563738) - Initiation & Completion Year
Figure 4-39: ARTEMIS-001 Phase I Study (NCT05276609) - Initiation & Completion Year
Figure 4-40: EMBOLD SCLC-301Phase III Study (NCT07099898) - Initiation & Completion Year
Figure 4-41: SOHO-01 Phase I/II Study (NCT05099172) - Initiation & Completion Year
Figure 4-42: ARROS-1 Phase I/II Study (NCT05118789) - Initiation & Completion Year
Figure 4-43: Skin Cancer - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-44: DOC-RM Phase I/II Study (NCT07288112) - Initiation & Completion Year
Figure 4-45: TILVANCE-301 Phase III Study (NCT05727904) - Initiation & Completion Year
Figure 4-46: AMXT1501-103 Phase I/II Study (NCT07287917) - Initiation & Completion Year
Figure 4-47: Gastrointestinal, hepatobiliary & pancreatic cancers - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-48: RZ-001-102 Phase I/II Study (NCT06695026) - Initiation & Completion Year
Figure 4-49: RMC-6236-001 Phase I Study (NCT05379985) - Initiation & Completion Year
Figure 4-50: RASolute-302 Phase III Study (NCT06625320) - Initiation & Completion Year
Figure 4-51: RASolute-303 Phase III Study (NCT07491445) - Initiation & Completion Year
Figure 4-52: RASolute-303 Phase III Study (NCT07252232) - Initiation & Completion Year
Figure 4-53: NP-G2-044-P2-01 Phase I/II Study (NCT05023486) - Initiation & Completion Year
Figure 4-54: TIGeR-PaC Phase III Study (NCT03257033) - Initiation & Completion Year
Figure 4-55: Genitourinary & gynecological cancers - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-56: PYNNACLE Phase II Study (NCT04585750) - Initiation & Completion Year
Figure 4-57: MYTHIC Phase I Study (NCT04855656) - Initiation & Completion Year
Figure 4-58: Head & neck, endocrine & other rare solid tumors - FDA Orphan Drug Designation Status & Approval Outcomes
Figure 4-59: NCI-2022-04100 Phase I Study (NCT05372640) - Initiation & Completion Year
Figure 4-60: NCI-2021-08926 Phase I/II Study (NCT05019716) - Initiation & Completion Year
Figure 8-1: US - Lynparza Approvals by Indication
Figure 8-2: US - Lynparza Orphan Drug Designation Years by Indication
Figure 8-3: US - Cost Of 60 & 120 Tablets supply of 100 mg & 150 mg Lynparza (US$), November'2026
Figure 8-4: US - Monthly Cost Of Treatment with 100 mg & 150 mg Lynparza Tablets (US$), November'2026
Figure 8-5: US - Tafinlar Approvals by Indication
Figure 8-6: US - Tafinlar Orphan Drug Designation Years
Figure 8-7: US - Cost Per Unit & Supply Of 120 Capsules of 50mg & 75mg Tafinlar (US$), November'2026
Figure 8-8: US - Cost Per Unit & Supply Of 210 Tablet 10mg Tafinlar (US$), November'2026
Figure 8-9: US - Monthly Cost Of Tafinlar Treatment By Weight (US$), November'2026
Figure 8-10: US - Halaven Approvals by Indication
Figure 8-11: US - Monthly Cost Of Treatment with Havalen Injection (US$), November'2026
Figure 8-12: US - Xpovio Orphan Drug Designation & Approval Year By Indication
Figure 8-13: US - Cost Per Unit & Supply Of 10mg, 20mg, 50mg, 60mg, 80mg Xpovio Tablets (US$), November'2026
Figure 8-14: US - Cost Per Unit & Supply Of 40mg Xpovio Tablets (US$), November'2026
Figure 8-15: US - Gavreto Orphan Drug Designation & Approval Year By Indication
Figure 8-16: US - Cost Per Unit & Supply Of 100mg Gavreto Tablets (US$), November'2026
Figure 8-17: US - Lumakras Orphan Drug Designation & Approval Year By Indication
Figure 8-18: US - Cost Per Unit & Supply Of 120mg & 320mg Lumakras Doses (US$), November'2026
Figure 8-19: US - Monjuvi Orphan Drug Designation & Approval Year By Indication
Figure 8-20: US - Cost Per Unit & Supply Of 10 mg/mL Opdivo (US$), November'2026
Figure 8-21: US - Opdualag Orphan Drug Designation & Approval Year
Figure 8-22: US - Cost Per Unit & Supply Of Opdualag Solution (US$), November'2026
Figure 8-23: US - Orladeyo Orphan Drug Designation & Approval Year
Figure 8-24: US - Cost Per Unit & Supply Of Rubraca Tablet (US$), November'2026
Figure 8-25: US - US - Monthly Cost Of Treatment with Rubraca Tablet (US$), November'2026
Figure 8-26: US - Bendeka Orphan Drug Designation & Approval Year By Indication
Figure 8-27: US - Cost Per Unit & Supply Of Bendeka Solution (US$), November'2026
Figure 8-28: US - Braftovi Orphan Drug Designation & Approval Year
Figure 8-29: US - Cost Per Unit & Supply Of 75mg Braftovi Capsule (US$), November'2026
Figure 8-30: US - Monthly Cost Of Melanoma & NSCLC Treatment with Braftovi (US$), November'2026
Figure 8-31: US - Cost Per Unit & Supply Of 15mg Mektovi Tablet (US$), November'2026
Figure 8-32: US - Monthly Cost Of Melanoma Treatment with Mektovi Tablet (US$), November'2026
Figure 8-33: Yervoy - First Approval by Indication
Figure 8-34: US - Cost Per Unit & Supply Of Yervoy Solution (US$), November'2026
Figure 8-35: US - Lorbrena Orphan Drug Designation & Approval Year
Figure 8-36: US - Cost Per Unit & Supply Of Lorbrena Tablet (US$), November'2026
Figure 8-37: US - Brukinsa Orphan Drug Designation & Approval Year By Indication
Figure 8-38: US - Cost Per Unit & Supply Of Brukinsa 160 mg Tablet & 80 mg Capsule (US$), November'2026
Figure 8-39: US - Monthly & Yearly Cost of Treatment with Brukinsa (US$), November'2026
Figure 8-40: US - Tecentriq Orphan Drug Designation & Approval Year
Figure 8-41: US - Cost Per Unit & Supply Of Tecentriq Intravenous Solution (US$), November'2026
Figure 8-42: US - Qinlock Orphan Drug Designation & Approval Year
Figure 8-43: US - Cost Per Unit & Supply Of Qinlock Tablets (US$), November'2026
Figure 8-44: US - Unit, Monthly & Annual Cost of Treatment with Qinlock (US$), November'2026
Figure 8-45: US - Kimmtrak Orphan Drug Designation & Approval Year
Figure 8-46: US - Cost Per Unit & Supply Of Kimmtrak (US$), November'2026
Figure 8-47: US - Elahere Orphan Drug Designation & Approval Year
Figure 8-48: US - Cost Per Unit & Supply Of 100 mg/20 mL Elahere Solution (US$), November'2026
Figure 8-49: US - Price for Single Unit of Polivy Intravenous Powder For Injection (US$), November'2026
Figure 8-50: US - Enhertu Orphan Drug Designation & Approval Year
Figure 8-51: Enhertu - Cost Of Single Treatment Cycle (US$), November'2026
Figure 8-52: US - Ruxolitinib Orphan Drug Designation & Approval Year By Indication
Figure 8-53: US - Cost Per Unit & Supply Of Different Jakafi Doses (US$), November'2026
Figure 8-54: US - Luspatercept Orphan Drug Designation & Approval Year
Figure 8-55: US - Cost Per Unit of Reblozyl powder (US$), November'2026
Figure 8-56: US - Monthly & Annual Cost of Treatment with Reblozyl (US$), November'2026
Figure 8-57: US - Luspatercept Orphan Drug Designation & Approval Year
Figure 8-58: US - Tazemetostat Orphan Drug Designation & Approval Year
Figure 8-59: US - Cost Per Unit & Supply Of Tazverik Tablet (US$), November'2026
Figure 8-60: US - Ninlaro Orphan Drug Designation & Approval Year
Figure 8-61: US - Cost Per Unit & Supply Of Ninlaro Capsule (US$), November'2026
Figure 8-62: US - Cost Of Single Dose, Monthly & Yearly Treatment with Ninlaro (US$), November'2026
Figure 8-63: US - Tagrisso Orphan Drug Designation & Approval Year
Figure 8-64: US - Cost Per Unit & Supply Of Tagrisso Tablets (US$), November'2026
Figure 8-65: US - Tagrisso Monthly, Yearly & Full Course Treatment Cost (US$), November'2026
Figure 8-66: US - Prevymis Orphan Drug Designation & Approval Year
Figure 8-67: US - Copiktra Orphan Drug Designation & Approval Year
Figure 8-68: US - Cost Per Unit & Supply Of 15 mg Copiktra Capsules (US$), November'2026
Figure 8-69: US - Jelmyto Orphan Drug Designation & Approval Year
Figure 8-70: US - Empliciti Orphan Drug Designation & Approval Year
Figure 8-71: US - Cost Per Unit & Supply Of Empliciti Powders (US$), November'2026
Figure 8-72: US - Kyprolis Orphan Drug Designation & Approval Year By Indication
Figure 8-73: US - Cost Per supply of 10mg, 30mg & 60mg Kyprolis (US$), November'2026
Figure 8-74: US - Gleolan Orphan Drug Designation & Approval Year By Indications
Figure 8-75: US - Darzalex Orphan Drug Designation & Approval Year
Figure 8-76: US - Cost Per Unit & Supply Of Darzalex Solution (US$), November'2026
Figure 8-77: US - Darzalex Orphan Drug Designation & Approval Year
Figure 8-78: US - Cost Per Unit & Supply Of Darzalex Faspro Solution (US$), November'2026
Figure 8-79: US - Lymphoseek Orphan Drug Designation & Approval Year
Figure 8-80: US - Detectnet Orphan Drug Designation & Approval Year
Figure 8-81: US - Folotyn Orphan Drug Designation & Approval Year
Figure 8-82: US - Cost Per Unit & Supply Of Folotyn (US$), November'2026
Figure 8-83: US - Xatmep Orphan Drug Designation & Approval Year
Figure 8-84: US - Cost Per Unit & Supply Of Xatmep (US$), November'2026
Figure 8-85: US - Purixan Orphan Drug Designation & Approval Year
Figure 8-86: US - Cost Per Unit & Supply Of Purixan (US$), November'2026
Figure 8-87: US - Busulfex Orphan Drug Designation & Approval Year
Figure 8-88: US - Cost Per Unit & Supply Of Busulfex (US$), November'2026
Figure 8-89: US - Doxil Orphan Drug Designation & Approval Year
Figure 8-90: US - Cost Per Unit & Supply Of Doxil (US$), November'2026
Figure 8-91: US - Targretin Orphan Drug Designation & Approval Year
Figure 8-92: US - Cost Per Unit & Supply Of Priftin (US$), November'2026
Figure 8-93: US - Azedra Orphan Drug Designation & Approval Year
Figure 8-94: US - Cost Of Treatment With Elzonris (US$), November'2026
Figure 8-95: US - Cost Per Unit & Supply Of Rituxan (US$), November'2026
Figure 8-96: US - Rezlidhia Orphan Drug Designation & Approval Year
Figure 8-97: US - Cost Per Unit & Supply Of Rezlidhia (US$), November'2026
Figure 8-98: US - Cost Of Treatment With Rezlidhia (US$), November'2026
Figure 8-99: US - Tecartus Orphan Drug Designation & Approval Year
Figure 8-100: US - Cresemba Orphan Drug Designation & Approval Year
Figure 8-101: US - Cost Per Unit & Supply Of Cresemba (US$), November'2026
Figure 8-102: US - Velcade Orphan Drug Designation & Approval Year
Figure 8-103: US - Besponsa Orphan Drug Designation & Approval Year
Figure 8-104: US - Vyxeos Orphan Drug Designation & Approval Year
Figure 8-105: US - Cost Per Unit & Supply Of Vyxeos (US$), November'2026
Figure 8-106: US - Unituxin Orphan Drug Designation & Approval Year
Figure 8-107: US - Cost Per Unit & Supply Of Unituxin (US$), November'2026
Figure 8-108: US - Alimta Orphan Drug Designation & Approval Year
Figure 8-109: US - Cost Of supply of Alimta (US$), November'2026
Figure 8-110: US - Aliqopa Orphan Drug Designation & Approval Year
Figure 8-111: US - Cost Of Treatment With Aliqopa (US$), November'2026
Figure 8-112: US - Aliqopa Orphan Drug Designation & Approval Year
Figure 8-113: US - Cost Per Unit & Supply Of Oncaspar (US$), November'2026
Figure 8-114: US - Rylaze Orphan Drug Designation & Approval Year
Figure 8-115: US - Cost Per Unit & of supply of Rylaze (US$), November'2026
Figure 8-116: US - Vonjo Orphan Drug Designation & Approval Year
Figure 8-117: US - Cost Per Unit & of supply of Vonjo (US$), November'2026
Figure 8-118: US - Xermelo Orphan Drug Designation & Approval Year
Figure 8-119: US - Cost Per Unit & Supply Of Xermelo (US$), November'2026
Figure 8-120: US - Gilotrif Orphan Drug Designation & Approval Year
Figure 8-121: US - Cost Per Unit & Supply Of Gilotrif Oral Tablets (US$), November'2026
Figure 8-122: US - Wilate Orphan Drug Designation & Approval Year
Figure 8-123: US - Stivarga Orphan Drug Designation & Approval Year
Figure 8-124: US - Cost Per Unit & Supply Of Stivarga Oral Tablets (US$), November'2026
Figure 8-125: US - Turalio Orphan Drug Designation & Approval Year
Figure 8-126: US - Cost Per Unit & Supply Of Turalio (US$), November'2026
Figure 8-127: US - Yondelis Orphan Drug Designation & Approval Year
Figure 8-128: US - Cost per Yondelis Injection (US$), November'2026
Figure 8-129: US - Onureg Orphan Drug Designation & Approval Year
Figure 8-130: US - Cost Per Unit & Supply Of Onureg (US$), November'2026
Figure 8-131: US - Cost per Onureg Cycle (US$), November'2026
Figure 8-132: US - Cabometyx Orphan Drug Designation & Approval Year
Figure 8-133: US - Cost Per Unit & Supply Of Cabometyx (US$), November'2026
Figure 8-134: US - Cabometyx Orphan Drug Designation & Approval Year
Figure 8-135: US - Cost Per Unit & Supply Of Cometriq (US$), November'2026
Figure 8-136: US - Carvykti Orphan Drug Designation & Approval Year
Figure 8-137: US - Hemlibra Orphan Drug Designation & Approval Year
Figure 8-138: US - Cost Of supply of Hemlibra (US$), November'2026
Figure 8-139: US - Exkivity Orphan Drug Designation & Approval Year
Figure 8-140: US - Cost per Unit & supply of Exkivity (US$), November'2026
Figure 8-141: US - Kymriah Orphan Drug Designation & Approval Year
Figure 8-142: US - Istodax Orphan Drug Designation & Approval Year
Figure 8-143: US - Bosulif Orphan Drug Designation & Approval Year
Figure 8-144: US - Cost per Unit & supply of Bosulif Oral Tablets (US$), November'2026
Figure 8-145: US - Cost per Unit & supply of Bosulif Oral Capsules (US$), November'2026
Figure 8-146: US - Idhifa Orphan Drug Designation & Approval Year
Figure 8-147: US - Monthly & Yearly Cost Of Treatment With Idhifa (US$), November'2026
Figure 8-148: US - Dojolvi Orphan Drug Designation & Approval Year
Figure 8-149: US - Cost Per Unit & Supply Of Pomalyst (US$), November'2026
Figure 8-150: US - Cost Per Unit & Supply Of Gleevec (US$), November'2026
Figure 8-151: US - Nexavar Orphan Drug Designation & Approval Year
Figure 8-152: US - Cost Per Unit & Supply Of Nexavar (US$), November'2026
Figure 8-153: US - Nexavar Orphan Drug Designation & Approval Year
Figure 8-154: US - Cost Of Supply of Treanda (US$), November'2026
Figure 8-155: US - Nexavar Orphan Drug Designation & Approval Year
Figure 8-156: US - Cost Per Unit & Supply Of Treanda (US$), November'2026
Figure 8-157: US - Xospata Orphan Drug Designation & Approval Year
Figure 8-158: US - Cost Per Unit & Supply Of Xospata (US$), November'2026
Figure 8-159: US - Thalomid Orphan Drug Designation & Approval Year
Figure 8-160: US - Cost Per Unit & Supply Of 50mg Thalomid Capsule (US$), November'2026
Figure 8-161: US - Cost Per Unit & Supply Of 100mg & 150mg Thalomid Capsule (US$), November'2026
Figure 8-162: US - Valchlor Orphan Drug Designation & Approval Year
Figure 8-163: US - Cost Per Unit & Supply Of Valchlor (US$), November'2026
Figure 8-164: US - Rozlytrek Orphan Drug Designation & Approval Year
Figure 8-165: US - Cost Per Unit & Supply Of Rozlytrek Oral Capsule (US$), November'2026
Figure 8-166: US - Cost Per Unit & Supply Of Rozlytrek Oral Pellet (US$), November'2026
Figure 8-167: US - Poteligeo Orphan Drug Designation & Approval Year
Figure 8-168: US - Cost Per Unit & Supply Of Poteligeo (US$), November'2026
Figure 8-169: US - Caprelsa Orphan Drug Designation & Approval Year
Figure 8-170: US - Cost Per Unit & Supply Of Caprelsa (US$), November'2026
Figure 8-171: US - Zolinza Orphan Drug Designation & Approval Year
Figure 8-172: US - Cost Per Unit & Supply Of Zolinza (US$), November'2026
Figure 8-173: US - Vitrakvi Orphan Drug Designation & Approval Year
Figure 8-174: US - Cost Per Unit & Supply Of Vitrakvi Capsule (US$), November'2026
Figure 8-175: US - Cost Per Unit & Supply Of Vitrakvi Solution (US$), November'2026
Figure 8-176: US - Obizur Orphan Drug Designation & Approval Year
Figure 8-177: US - Torisel Orphan Drug Designation & Approval Year
Figure 8-178: US - Unit & Monthly Cost Of Treatment With Torisel
Figure 8-179: US - Ayvakit Orphan Drug Designation & Approval Year
Figure 8-180: US - Cost Per Unit & Supply Of Ayvakit (US$), November'2026
Figure 8-181: US - Mekinist Orphan Designations & Approval Year
Figure 8-182: US - Cost Per Unit & Supply Of Mekinist Oral Tablet (US$), November'2026
Figure 8-183: US – Cost Per Unit & Supply Of Mekinist Powder (US$), November’2026
Figure 8-184: US – Yescarta Orphan Drug Designation & Approval Year
Figure 8-185: US – Lunsumio Orphan Drug Designation & Approval Year
Figure 8-186: US – Cost Per Unit & Supply Of Lunsumio (US$), November’2026
Figure 8-187: US – Bavencio Orphan Drug Designation & Approval Year
Figure 8-188: US – Cost Per Unit & Supply Of Bavencio (US$), November’2026
Figure 8-189: US – Pemazyre Orphan Drug Designation & Approval Year
Figure 8-190: US – Cost Per Unit & Supply Of Pemazyre (US$), November’2026
Figure 8-191: US – Inqovi Orphan Drug Designation & Approval Year
Figure 8-192: US – Cost Per Unit & Supply Of Inqovi (US$), November’2026
Figure 8-193: US – Cost Per Unit & Supply Of Keytruda (US$), November’2026
Figure 8-194: US – Cost Per Unit & Supply Of 10 mg-50 mg-100 mg Venclexta (US$), November’2026
Figure 8-195: US – Cost Per Unit & Supply Of 10 mg Venclexta (US$), November’2026
Figure 8-196: US – Cost Per Unit & Supply Of 50 mg Venclexta (US$), November’2026
Figure 8-197: US – Cost Per Unit & Supply Of 100 mg Venclexta (US$), November’2026
Figure 8-198: US – Beleodaq Orphan Drug Designation & Approval Year
Figure 8-199: US – Jaypirca Orphan Drug Designation & Approval Year
Figure 8-200: US – Cost Per Unit & Supply Of Jaypirca (US$), November’2026
Figure 8-201: US – Tibsovo Orphan Drug Designation & Approval Year
Figure 8-202: US – Cost Per Unit & Supply Of Filspari (US$), November’2026
Figure 8-203: US – Idecabtagene Vicleucel Orphan Drug Designation & Approval Year
Figure 8-204: US – Abraxane Orphan Drug Designation & Approval Year
Figure 8-205: US – Arranon Orphan Drug Designation & Approval Year
Figure 8-206: US – Cost Per Unit & Supply Of Arranon (US$), November’2026
Figure 8-207: US – Brigatinib Orphan Drug Designation & Approval Year
Figure 8-208: US – Cost Per Unit & Supply Of Alunbrig (US$), November’2026
Figure 8-209: US – Dacomitinib Orphan Drug Designation & Approval Year
Figure 8-210: US – Cost Per Unit & Supply Of Vizimpro (US$), November’2026
Figure 8-211: US – Netspot Orphan Drug Designation & Approval Year
Figure 8-212: US – Cost Per Unit & Supply Of Trisenox (US$), November’2026
Figure 8-213: US – Dacogen Orphan Drug Designation & Approval Year
Figure 8-214: US – Cost Per Unit & Supply Of 2.5mg, 5mg &10mg Revlimid (US$), November’2026
Figure 8-215: US – Cost Per Unit & Supply Of 15m, 20mg & 25mg Revlimid (US$), November’2026
Figure 8-216: US – Asparlas Orphan Drug Designation & Approval Year
Figure 8-217: US – Cost Per Unit & Supply Of Asparlas (US$), November’2026
Figure 8-218: US – Cost Per Unit & Supply Of Sarclisa (US$), November’2026
Figure 8-219: US – Vorasidenib Orphan Drug Designation & Approval Year
Figure 8-220: US – Cost Per Unit & Supply Of Voranigo (US$), November’2026
Figure 8-221: US – Tepotinib Orphan Drug Designation & Approval Year
Figure 8-222: US – Cost Per Unit & Supply Of Tepmetko (US$), November’2026
Figure 8-223: US – Pafolacianine Orphan Drug Designation & Approval Year
Figure 8-224: US – Treosulfan Orphan Drug Designation & Approval Year
Figure 8-225: US – Cost Of Supply Of Grafapex (US$), November’2026
Figure 8-226: US – Zolbetuximab Orphan Drug Designation & Approval Year
Figure 8-227: US – Cost Of Supply Of Vyloy (US$), November’2026
Figure 8-228: US – Talquetamab Orphan Drug Designation & Approval Year
Figure 8-229: US – Cost Per Unit & Supply Of Talvey (US$), November’2026
Figure 8-230: US – Imetelstat Orphan Drug Designation & Approval Year
Figure 8-231: US – Cost Of Supply Of Rytelo (US$), November’2026
Figure 8-232: US – Repotrectinib Orphan Drug Designation & Approval Year
Figure 8-233: US – Cost Per Unit & Supply Of 40mg Augtyro (US$), November’2026
Figure 8-234: US – Cost Per Unit & Supply Of 60mg Augtyro (US$), November’2026
Figure 8-235: US – Penpulimab Orphan Drug Designation & Approval Year
Figure 8-236: US – Tislelizumab Orphan Drug Designation & Approval Year
Figure 8-237: US – Cost Per Unit & Supply Of Tevimbra (US$), November’2026
Figure 8-238: US – Adagrasib Orphan Drug Designation & Approval Year
Figure 8-239: US – Cost Per Unit & Supply Of Krazati (US$), November’2026
Figure 8-240: US – Futibatinib Orphan Drug Designation & Approval Year
Figure 8-241: US – Cost Per Unit & Supply Of Lytgobi (US$), November’2026
Figure 8-242: US – Zanidatamab Orphan Drug Designation & Approval Year
Figure 8-243: US – Cost Per Unit & Supply Of Ziihera (US$), November’2026
Figure 8-244: US – Cost Per Unit & Supply Of Loqtorzi (US$), November’2026
Figure 8-245: US – Retifanlimab Orphan Drug Designation & Approval Year
Figure 8-246: US – Cost Per Unit & Supply Of Zynyz (US$), November’2026
Figure 8-247: US – Zenocutuzumab Orphan Drug Designation & Approval Year
Figure 8-248: US – Cost Per Unit & Supply Of Bizengri (US$), November’2026
Figure 8-249: US – Elranatamab Orphan Drug Designation & Approval Year
Figure 8-250: US – Cost Per Unit & Supply Of Elrexfio (US$), November’2026
Figure 8-251: US – Revumenib Orphan Drug Designation & Approval Year
Figure 8-252: US – Cost Per Unit & Supply Of Revuforj (US$), November’2026
Figure 8-253: US – Asciminib Orphan Drug Designation & Approval Year
Figure 8-254: US – Cost Per Unit & Supply Of Scemblix (US$), November’2026
Figure 8-255: US – Tarlatamab Orphan Drug Designation & Approval Year
Figure 8-256: US – Cost Of Supply Of Imdelltra (US$), November’2026
Figure 8-257: US – Teclistamab Orphan Drug Designation & Approval Year
Figure 8-258: US – Cost Per Unit & Supply Of Tecvayli (US$), November’2026
Figure 8-259: US – Quizartinib Orphan Drug Designation & Approval Year
Figure 8-260: US – Cost Per Unit & Supply Of Vanflyta (US$), November’2026
Figure 8-261: US – Tremelimumab Orphan Drug Designation & Approval Year
Figure 8-262: US – Cost Per Unit & Supply Of Imjudo (US$), November’2026
Figure 8-263: US – Mavorixafor Orphan Drug Designation & Approval Year
Figure 8-264: US – Cost Per Unit & Supply Of Xolremdi (US$), November’2026
Figure 8-265: US – Tovorafenib Orphan Drug Designation & Approval Year
Figure 8-266: US – Cost Per Unit & Supply Of Ojemda Oral Suspension (US$), November’2026
Figure 8-267: US – Cost Per Unit & Supply Of Ojemda Oral Tablets (US$), November’2026
Figure 8-268: US – Momelotinib Orphan Drug Designation & Approval Year
Figure 8-269: US – Cost Per Unit & Supply Of Ojjaara (US$), November’2026
Figure 8-270: US – Nirogacestat Orphan Drug Designation & Approval Year
Figure 8-271: US – Cost Per Unit & Supply Of Ogsiveo (US$), November’2026
Figure 9-1: Krazati - Global Sales (US$ Million), 2023-2026
Figure 9-2: Krazati - Global Sales By Region (US$ Million), 2023-2026
Figure 9-3: Tecvayli - Global Sales (US$ Million), 2022-2026
Figure 9-4: Tecvayli - Global Sales By Region (US$ Million), 2022-2026
Figure 9-5: Imjudo - Global Sales (US$ Million), 2024-2026
Figure 9-6: Imjudo - Global Sales By Region (US$ Million), 2024-2026
Figure 9-7: Elrexfio - Global Sales (US$ Million), 2024-2026
Figure 9-8: Elrexfio - Global Sales By Region (US$ Million), 2024-2026
Figure 9-9: Jaypirca - Global Sales (US$ Million), 2024-2026
Figure 9-10: Jaypirca - Global Sales By Region (US$ Million), 2024-2026
Figure 9-11: Imdelltra - Global Sales (US$ Million), 2024-2026
Figure 9-12: Imdelltra - Global Sales By Region (US$ Million), 2024-2026
Figure 9-13: Talvey - Global Sales (US$ Million), 2023-2026
Figure 9-14: Talvey - Global Sales By Region (US$ Million), 2023-2026
Figure 9-15: Zynyz - Global Sales (US$ Million), 2023-2026
Figure 9-16: Ojjaara - Global Sales (US$ Million), 2023-2026
Figure 9-17: Ojjaara - Global Sales By Region (US$ Million), 2023-2026
Figure 9-18: Welireg - Global Sales (US$ Million), 2022-2026
Figure 9-19: Welireg - Global Sales By Region (US$ Million), 2022-2026
Figure 9-20: Scemblix - Global Sales (US$ Million), 2021-2026
Figure 9-21: Scemblix - Global Sales By Region (US$ Million), 2024-2026
Figure 9-22: Zynlonta - Global Sales (US$ Million), 2021-2026
Figure 9-23: Zynlonta - Global Sales (US$ Million), Q1 & Q2’2026
Figure 9-24: Zynlonta - Global Sales (US$ Million), Q1-Q4’2025
Figure 9-25: Lytgobi - Global Sales (US$ Million), 2023-2026
Figure 9-26: Lytgobi - Global Sales (US$ Million), Q1 & Q2’2026
Figure 9-27: Lytgobi - Global Sales (US$ Million), Q1-Q4’2025
Figure 9-28: Vanflyta - Global Sales (US$ Million), 2023-2026
Figure 9-29: Vanflyta - Global Sales (US$ Million), Q1 & Q2’2026
Figure 9-30: Vanflyta - Global Sales (US$ Million), Q1-Q4’2025
Figure 9-31: Tafinlar + Mekinist - Global Sales (US$ Million), 2021-2026
Figure 9-32: Tafinlar + Mekinist - Global Sales (US$ Million), Q1 & Q2’2026
Figure 9-33: Tafinlar + Mekinist - Global Sales (US$ Million), Q1-Q4’2025
Figure 9-34: Kimmtrak- Global Sales (US$ Million), 2022-2026
Figure 9-35: Kimmtrak- Global Sales By Region (US$ Million), 2023-2026
Figure 9-36: Rezlidhia- Global Sales (US$ Million), 2022-2026
Figure 9-37: Ogsiveo - Global Sales (US$ Million), 2023-2026
Figure 9-38: Revuforj - Global Sales (US$ Million), 2024-2026
Figure 9-39: Ojemda - Global Sales (US$ Million), 2024-2025
Figure 9-40: Rytelo - Global Sales (US$ Million), 2024-2026
Figure 9-41: Ziihera – US Sales (US$ Million), 2024-2026
Figure 9-42: Augtyro - Global Sales (US$ Million), 2023-2024
Figure 9-43: Augtyro - Global Sales By Region (US$ Million), 2024
Figure 9-44: Vonjo - Global Sales (US$ Million), 2023-2026
Figure 9-45: Vyloy - Global Sales (US$ Million), 2024-2026
Figure 9-46: Vyloy - Global Sales By Region (US$ Million), 2023-2026
Figure 9-47: Loqtorzi - US Sales (US$ Million), 2022-2026
Figure 9-48: Tevimbra - Global Sales (US$ Million), 2024-2026
Figure 9-49: Tevimbra - Global Sales (US$ Million), Q1 & Q2’2026
Figure 9-50: Tevimbra - Global Sales (US$ Million), Q1-Q4’2025
Figure 9-51: Lynparza - Global Sales (US$ Million), 2021-2026
Figure 9-52: Lynparza - Global Sales By Region (US$ Million), 2026
Figure 9-53: Lynparza - US Sales (US$ Million), 2021-2026
Figure 9-54: Lynparza - Europe Sales (US$ Million), 2021-2026
Figure 9-55: Lynparza – Emerging Markets Sales (US$ Million), 2021-2026
Figure 9-56: Lynparza – ROW Sales (US$ Million), 2021-2026
Figure 9-57: Lumakras - Global Sales (US$ Million), 2021-2026
Figure 9-58: Lumakras - US Sales (US$ Million), 2021-2026
Figure 9-59: Lumakras - ROW Sales (US$ Million), 2021-2026
Figure 9-60: Kyprolis - Global Sales (US$ Million), 2021-2026
Figure 9-61: Kyprolis – Global Sales By Region (US$ Million), 2021-2026
Figure 9-62: Opdivo - Global Sales (US$ Million), 2021-2026
Figure 9-63: Opdivo – Global Sales By Region (US$ Million), 2025
Figure 9-64: Opdualag - Global Sales (US$ Million), 2022-2026
Figure 9-65: Opdualag – Global Sales By Region (US$ Million), 2022-2026
Figure 9-66: Braftovi + Mektovi - Global Sales (US$ Million), 2021-2026
Figure 9-67: Braftovi + Mektovi – Global Sales By Region (US$ Million), 2022-2026
Figure 9-68: Lorbrena - Global Sales (US$ Million), 2021-2026
Figure 9-69: Lorbrena - Global Sales By Region (US$ Million), 2021-2026
Figure 9-70: Besponsa - Global Sales (US$ Million), 2021-2023
Figure 9-71: Besponsa - Global Sales (US$ Million), Q1-Q4’2022
Figure 9-72: Besponsa - US Sales (US$ Million), 2021-2023
Figure 9-73: Besponsa – US Sales (US$ Million), Q1-Q4’2022
Figure 9-74: Besponsa – Global Sales By Region (US$ Million), 2022
Figure 9-75: Besponsa - ROW Sales (US$ Million), 2021-2023
Figure 9-76: Tecentriq - Global Sales (US$ Million), 2021-2026
Figure 9-77: Tecentriq - Global Sales By Region (US$ Million), 2026
Figure 9-78: Tecentriq - US Sales (US$ Million), 2021-2026
Figure 9-79: Tecentriq – Europe Sales (US$ Million), 2021-2026
Figure 9-80: Tecentriq – Japan Sales (US$ Million), 2021-2026
Figure 9-81: Tecentriq – ROW Sales (US$ Million), 2021-2026
Figure 9-82: Polivy - Global Sales (US$ Million), 2021-2026
Figure 9-83: Polivy - Global Sales By Region (US$ Million), 2026
Figure 9-84: Polivy - US Sales (US$ Million), 2021-2026
Figure 9-85: Polivy – Europe Sales (US$ Million), 2021-2026
Figure 9-86: Polivy – Japan Sales (US$ Million), 2021-2026
Figure 9-87: Polivy – ROW Sales (US$ Million), 2021-2026
Figure 9-88: Yervoy - Global Sales (US$ Million), 2021-2026
Figure 9-89: Yervoy - Global Sales By Region (US$ Million), 2021-2026
Figure 9-90: Elahere - Global Sales (US$ Million), 2022-2026
Figure 9-91: Elahere - Global Sales By Region (US$ Million), 2024-2026
Figure 9-92: Jakafi - Global Sales (US$ Million), 2021-2026
Figure 9-93: Jakafi - Global Sales By Region (US$ Million), 2021-2025
Figure 9-94: Ninlaro - Global Sales (US$ Million), 2022-2026
Figure 9-95: Ninlaro - Global Sales By Region (US$ Million), 2022-2026
Figure 9-96: Adcetris - US Sales (US$ Million), 2023-2026
Figure 9-97: Bendeka - Global Sales (US$ Million), 2021-2026
Figure 9-98: Tagrisso - Global Sales (US$ Million), 2021-2026
Figure 9-99: Tagrisso - Global Sales By Region (US$ Million), 2026
Figure 9-100: Tagrisso - US Sales (US$ Million), 2021-2026
Figure 9-101: Tagrisso - Europe Sales (US$ Million), 2021-2026
Figure 9-102: Tagrisso – Emerging Markets Sales (US$ Million), 2021-2026
Figure 9-103: Tagrisso – ROW Sales (US$ Million), 2021-2026
Figure 9-104: Enhertu - Global Sales (US$ Million), 2021-2026
Figure 9-105: Enhertu - Global Sales By Region (US$ Million), Q1 & Q2’2026
Figure 9-106: Enhertu - Global Sales By Region (US$ Million), Q1-Q4’2026
Figure 9-107: Tecartus - Global Sales (US$ Million), 2021-2026
Figure 9-108: Tecartus - US Sales (US$ Million), 2021-2026
Figure 9-109: Tecartus - Europe Sales (US$ Million), 2021-2026
Figure 9-110: Tecartus - ROW Sales (US$ Million), 2021-2026
Figure 9-111: Darzalex - Global Sales (US$ Million), 2021-2026
Figure 9-112: Darzalex - Global Sales By Region (US$ Million), 2021-2026
Figure 9-113: Cyramza - Global Sales (US$ Million), 2021-2026
Figure 9-114: Cyramza - Global Sales By Region (US$ Million), 2021-2026
Figure 9-115: Alimta - Global Sales (US$ Million), 2021-2026
Figure 9-116: Alimta - Global Sales By Region (US$ Million), 2021-2026
Figure 9-117: Vyxeos - Global Sales (US$ Million), 2021-2026
Figure 9-118: Rylaze - Global Sales (US$ Million), 2021-2026
Figure 9-119: Hemlibra - Global Sales (US$ Million), 2021-2026
Figure 9-120: Hemlibra – Global Sales By Region (US$ Million), 2026
Figure 9-121: Hemlibra - US Sales (US$ Million), 2021-2026
Figure 9-122: Hemlibra - Europe Sales (US$ Million), 2021-2026
Figure 9-123: Hemlibra - Japan Sales (US$ Million), 2021-2026
Figure 9-124: Hemlibra - ROW Sales (US$ Million), 2021-2026
Figure 9-125: Venclexta - Global Sales (US$ Million), 2021-2026
Figure 9-126: Venclexta - Global Sales By Region (US$ Million), 2021-2026
Figure 9-127: Cometriq - US Sales (US$ Million), 2021-2026
Figure 9-128: Cabometyx - US Sales (US$ Million), 2021-2026
Figure 9-129: Cabometyx - Global Sales (US$ Million), Q1 & Q2’2026
Figure 9-130: Cabometyx - Global Sales (US$ Million), Q1-Q4’2025
Figure 9-131: Carvykti - Global Sales (US$ Million), 2022-2026
Figure 9-132: Carvykti - Global Sales By Region (US$ Million), 2022-2026
Figure 9-133: Kymriah - Global Sales (US$ Million), 2021-2026
Figure 9-134: Pomalyst - Global Sales (US$ Million), 2021-2026
Figure 9-135: Pomalyst - Global Sales By Region (US$ Million), 2021-2026
Figure 9-136: Gleevec - Global Sales (US$ Million), 2019-2023
Figure 9-137: Gleevec – Global Sales (US$ Million), Q1-Q4’ 2022
Figure 9-138: Turalio - Global Sales (US$ Million), 2023-2026
Figure 9-139: Bosulif - Global Sales (US$ Million), 2021-2026
Figure 9-140: Bosulif - Global Sales By Region (US$ Million), 2021-2026
Figure 9-141: Poteligeo - Global Sales (US$ Million), 2023-2026
Figure 9-142: Xospata - Global Sales (US$ Million), 2023-2026
Figure 9-143: Xospata - Global Sales By Region (US$ Million), 2023-2026
Figure 9-144: Yescarta - Global Sales (US$ Million), 2021-2026
Figure 9-145: Yescarta - Global Sales By Region (US$ Million), 2021-2026
Figure 9-146: Lunsumio - Global Sales (US$ Million), 2022-2026
Figure 9-147: Lunsumio - Global Sales By Region (US$ Million), 2026
Figure 9-148: Lunsumio - US Sales (US$ Million), 2022-2026
Figure 9-149: Lunsumio - Europe Sales (US$ Million), 2022-2026
Figure 9-150: Lunsumio – Japan Sales (US$ Million), 2025 & 2026
Figure 9-151: Lunsumio – ROW Sales (US$ Million), 2024-2026
Figure 9-152: Keytruda - Global Sales (US$ Million), 2021-2026
Figure 9-153: Keytruda – Global Sales By Region (US$ Million), 2021-2026
Figure 9-154: Pemazyre - Global Sales (US$ Million), 2021-2026

List of Tables

Table 2-1: US – FDA Categorization for Cancer
Table 2-2: US – FDA Criteria for Orphan & non-Orphan Drug Approvals
Table 8-1: Tafinlar – Recommended Weight-Adjusted Dose
Table 8-2: Nivolumab – FDA Orphan Drug Designation & Approvals
Table 8-3: Opdivo - Recommended Dosages for Monotherapy
Table 8-4: Opdivo - Recommended Dosages in Combination with Other Therapeutic Agents
Table 8-5: Ipilimumab – FDA Orphan Drug Designation & Approvals
Table 8-6: Yervoy - Recommended Dosages as a Single Agent
Table 8-7: Yervoy - Recommended Dosages in Combination with Other Therapeutic Agents
Table 8-8: Tecentriq - Recommended Dosage in Combination with Other Therapeutic Agents
Table 8-9: Kimmtrak - Recommended Dosage for Treatment of Unresectable or Metastatic Uveal Melanoma
Table 8-10: Jakafi - Starting Doses for Myelofibrosis
Table 8-11: Empliciti - Recommended Dosing Schedule in Combination with Lenalidomide & Dexamethasone
Table 8-12: Empliciti - Recommended Dosing Schedule in Combination with Pomalidomide & Dexamethasone
Table 8-13: Kyprolis - 20/70 mg/m2 Once Weekly (30-Minute Infusion)
Table 8-14: Kyprolis - 20/56 mg/m2 Twice Weekly (30-Minute Infusion)
Table 8-15: Daratumumab – FDA Orphan Drug Designation & Approvals
Table 8-16: Darzalex - Dosing Schedule in Combination With Lenalidomide or Pomalidomide (4-Week Cycle) and Low-Dose Dexamethasone and for Monotherapy
Table 8-17: Darzalex - Dosing Schedule in Combination With Bortezomib, Melphalan and Prednisone ([VMP], 6-Week Cycle)
Table 8-18: Darzalex - Dosing Schedule in Combination With Bortezomib, Thalidomide and Dexamethasone ([VTd]; 4-Week Cycle)
Table 8-19: Darzalex - Dosing Schedule With Bortezomib and Dexamethasone (3-Week Cycle)
Table 8-20: Darzalex - Dosing Schedule With Carfilzomib and Dexamethasone (4-Week Cycle)
Table 8-21: Darzalex Faspro - Dosing Schedule in Combination With Bortezomib, Cyclophosphamide and Dexamethasone (4-week cycle)
Table 8-22: Aldesleukin - FDA Orphan Drug Designation & Approvals
Table 8-23: Rituximab – Orphan Drug Designation & Approvals
Table 8-24: Rituxan – Recommended Dosage
Table 8-25: Cresemba - Recommended Dosage Regimen In Adult Patients
Table 8-26: Cresemba - Recommended Dosage Regimen In Adult Patients
Table 8-27: Velcade - Dosage Regimen for Patients with Previously Untreated Multiple Myeloma
Table 8-28: Vyxeos - Dose & Schedule
Table 8-29: Brentuximab Vedotin – FDA Orphan Drug Designation & Approvals
Table 8-30: Adcetris - Recommended Dosage
Table 8-31: Imatinib – FDA Orphan Drug Designation & Approvals
Table 8-32: Rozlytrek - Recommended Dosage For Adults and Pediatric patients for Treatment of NTRK Gene Fusion-Positive Solid Tumors
Table 8-33: Rozlytrek – Recommended Dosage For Pediatric Patients Older than 6 Months for the Treatment of NTRK Gene Fusion-Positive Solid Tumors
Table 8-34: Mekinist – Recommended Dosage & Treatment Cost per Month (30 days) in Pediatric Patients (Weight-Based) (US$), November’2026
Table 8-35: Lunsumio – Recommended Dose, Schedule & Cost Of Treatment (21-day Treatment Cycle) (US$), November’2026
Table 8-36: US – Keytruda Orphan Drug Designation Year
Table 8-37: US – Keytruda Orphan Drug Designation Year
Table 8-38: Venclexta – Recommended Dosing Schedule for Ramp-up Phase for Patients with CLL
Table 8-39: Venclexta – Recommended Dosing Schedule for RamUp Phase with Cost per Dose for AML (US$), June’2023
Table 8-40: Trisenox - Recommended Dosage in Combination With Tretinoin
Table 8-41: US – lenalidomide Orphan Drug Designations
Table 8-42: Sarclisa – Dosing Schedule In Combination With Pomalidomide & Dexamethasone Or In Combination With Carfilzomib & Dexamethasone
Table 8-43: Sarclisa – Dosing Schedule In Combination With Bortezomib, Lenalidomide & Dexamethasone
Table 8-44: Grafapex - Dosage Regimen for Therapy-Based Allogeneic HSCT
Table 8-45: Talvey - Weekly Dosing Schedule
Table 8-46: Talvey - Biweekly Dosing Schedule
Table 8-47: Penpulimab - Order of Administration & Regimen in Combination with Either Cisplatin or Carboplatin & Gemcitabine
Table 8-48: Tevimbra - Recommended Dosages as a Single Agent Or In Combination with Other Therapeutic Agent
Table 8-49: Toripalimab - FDA Orphan Drug Designation Dates
Table 8-50: Elrexfio - Dosing Schedule
Table 8-51: Revuforj - Recommended Dosage for Patients 1 Year & Older
Table 8-52: Revuforj - Recommended Dosage Using Tablets for Patients Weighing Less Than 40 kg
Table 8-53: Imdelltra - Recommended Dosage and Schedule
Table 8-54: Tecvayli - Dosing Schedule
Table 8-55: Vanflyta - Dosage Regimen
Table 8-56: Imjudo - Recommended Dosage In Combination with Durvalumab
Table 8-57: Ojemda - Recommended Tablets Dosage Based On Body Surface Area
Table 8-58: Ojemda - Recommended Dosage Oral Suspension Based On Body Surface Area

US Orphan Cancer Drug Market Size, Drug Pricing, Sales, Market Forecast, Competitive Landscape & Clinical Trials Insight 2031

Description

Table of Contents

List of Tables

Table of Contents

1. Research Methodology

2. FDA Orphan Designation Criteria & Market Exclusivity

3. US Orphan Designated Cancer Drugs Market Insight

4. US Orphan Designated Cancer Market Trends & Clinical Innovation By Indications

5. US Orphan Designated Drugs Reimbursement Scenario

6. US Orphan Designated Cancer Drugs Clinical Trials By Company, Indication & Phase

7. Marketed Orphan Designated Cancer Drugs Clinical Insight By Company & Indication

8. US Orphan Designated Cancer Drugs Dosage, Price & Treatment Cost Insight

9. US Orphan Designated Cancer Drugs Sales Insight (2021-2026)

10. Competitive Landscape

List of Figures

List of Tables