PUBLISHER: DelveInsight | PRODUCT CODE: 1376840
PUBLISHER: DelveInsight | PRODUCT CODE: 1376840
Report Summary
The table given below further depicts the key segments provided in the report:
Various key players currently leading the treatment landscape of SCD include Novartis Pharmaceuticals, Global Blood Therapeutics/Pfizer, and Emmaus Life Sciences. Moreover, various key players are investigating their therapeutic candidates in the late phases of clinical development. Vertex Pharmaceuticals/CRISPR Therapeutics, Global Blood Therapeutics/Pfizer, Forma Therapeutics/Novo Nordisk, Bluebird Bio, etc. The details of the country-wise and therapy-wise market size have been provided below.
The section dedicated to drugs in the SCD report provides an in-depth evaluation of marketed therapies and the late-stage pipeline drugs (Phase III and Phase II) related to SCD.
The drug chapters section provides valuable information on various aspects related to clinical trials of SCD, including specific details, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting SCD.
ENDARI (L-glutamine) is an oral-administered pharmaceutical grade L-glutamine (PGLG), an amino acid formulation to relieve pain, swelling, and other complications of sickle cell anemia in adults and children 5 years and older.
ADAKVEO (crizanlizumab) is a humanized anti-P selectin monoclonal antibody that binds a molecule called P-selectin on the surface of endothelial cells and platelets in the blood vessels, causing the blockade of P-selectin; it drives the vaso-occlusive process. The therapeutic blockade of P-selectin can prevent painful vaso-occlusion in small blood vessels and maintain blood flow.
OXBRYTA is an oral, once-a-day tablet developed by Global Blood Therapeutics. It is designed to block sickle hemoglobin polymerization, the root cause of sickle cell disease. This results in poor blood flow that affects the oxygenation of tissues, ultimately leading to painful vaso-occlusive crises. By increasing hemoglobin's affinity to oxygen, OXBRYTA halts this process.
Exa-cel (CTX001) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient's hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. Currently, it is being evaluated in Phase III for treating SCD patients.
Inclacumab is a novel, fully human monoclonal antibody that selectively targets P-selectin. This protein mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab can be a best-in-class option for reducing VOCs in people with SCD. The therapy is estimated to launch by 2025 and has a slow-medium uptake.
Etavopivat is an orally available, small-molecule allosteric activator of the selective red blood cell (RBC) isoform of pyruvate kinase (PK-R), with the potential to improve symptoms in sickle cell disease (SCD) patients. Currently being evaluated in Phase II/III clinical development, the therapy is estimated to launch by 2026 and attain its peak in 7 years.
The treatment goals for SCD aim to relieve pain, prevent infections, and specifically manage complications. The goals are for symptom control and management of disease complications. The treatment starts with the recognition of pain in SCD patients. Recurrent episodes of acute, severe pain are the hallmark of SCD.
Initial management aims at providing rapid pain control. Many drug regimens have effectively treated acute pain in SCD. Pain management should follow the three-step "analgesic ladder" recommended by the World Health Organization for treating cancer-related pain. The choice of analgesic and dosage should be based on the severity of pain in the individual patient.
The treatment pattern currently consists of different approaches classified into pharmacologic and nonpharmacological therapies. The pharmacological therapies, including hydroxyurea, ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor), are approved. Pain management agents are segregated into opioids, nonsteroidal anti-inflammatory drugs (NSAIDs), acetaminophen, corticosteroids, etc. Acute vaso-occlusive crisis is generally managed using opioids and nonsteroidal anti-inflammatory drugs (NSAIDs). Further, nonpharmacological therapies include cognitive behavioral therapy, biofeedback, relaxation techniques, acupuncture, hypnosis, etc.
The current SCD treatment patterns allow emerging therapies to fill the voids, such as providing a complete cure, tolerable treatment side effects per se, and improved dosing and dosage forms. The patient burden will increase in the coming times, and approval of the emerging drug will expand the market. Therefore, the overall market size will increase, which could be a better investment therapy area.
Sickle Cell Disease (SCD) is a group of lifelong inherited conditions that affect hemoglobin. It is characterized as a chronic hemolytic disorder marked by the tendency of hemoglobin molecules within red blood cells to polymerize and deform the red cell into sickle (or crescent) shape (Hb S), resulting in characteristic vaso-occlusive events and accelerated hemolysis.
Sickle cell disease is inherited in an autosomal fashion, whether in the homozygous or double heterogeneous state. Sickle cell disease is called sickle cell anemia (SCA) when there is an inheritance in the homozygous state. Other known SCD genotypes include hemoglobin SC disease, sickle beta plus thalassemia, sickle beta zero thalassemia (which has similar severity with sickle cell anemia), hemoglobin SD Punjab disease, hemoglobin SO Arab disease, and others. Hemoglobin S (Hb S) differs from normal hemoglobin (Hb A) because of the substitution of valine for glutamic acid in the sixth position in the B-globin gene.
Sickle cell anemia can lead to many complications, including stroke, acute chest syndrome, pulmonary hypertension, organ damage, splenic sequestration, leg ulcers, gallstones, priapism, deep vein thrombosis, and others.
SCD diagnosis starts with a blood test that is analyzed for defective genes or hemoglobin cells. Various screening programs also help in the early diagnosis of the disease during the prenatal or infancy period.
The primary objective of early intervention is to relieve pain. Numerous medication plans have successfully addressed sudden pain in sickle cell disease (SCD). For effective pain relief, it is advisable to adhere to the three-stage "analgesic ladder" endorsed by the World Health Organization, which is commonly used for managing cancer-related pain. The selection of the appropriate painkiller and its dosage needs to be determined by the intensity of pain experienced by each patient.
Oxygen therapy is often used routinely in managing vaso-occlusive crises, despite a lack of evidence supporting the effectiveness of these measures in all patients. Fluids should be administered in a quantity sufficient to correct existing deficits and replace ongoing losses to maintain a normal state. Moreover, antibiotics should be considered if there is evidence of infection.
Hydroxyurea is often used in patients with severe complications who can reliably follow the regimen. Hydroxyurea reduces the frequency of painful crises and the need for blood transfusions in patients with recurrent painful crises. Lastly, the only chance for a cure for Sickle Cell Disease is bone marrow or stem cell transplantation. The bone marrow nurtures stem cells, which are early cells that mature into red and white blood cells and platelets.
The Sickle Cell Disease (SCD) epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of SCD Trait, Total Prevalent Cases of SCD, Diagnosed Cases of SCD, Age-specific Cases of SCD, and Type-specific Cases of SCD in the 6MM from 2019 to 2032.
To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.
We have reached out to industry experts to gather insights on various aspects of SCD, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the United States.
Our team of analysts at DelveInsight connected with more than 10 KOLs in the United. We contacted institutions such as the Brooklyn Hospital Center, the Department of Pediatrics, Duke University, and others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the SCD market, which will assist our clients in analyzing the overall epidemiology and market scenario.
We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in trials for SCD, one of the most important primary endpoints was the proportion of subjects who have not experienced any severe vaso-occlusive crisis, frequency, and severity of collected adverse events (AEs). Based on these, the overall efficacy is evaluated.
Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.