This report covers the sickle cell anemia market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, patent information, probability of success, a 10-year disease prevalence forecast, and licensing and acquisition deals.
The research estimates that in 2017, there were 3.2 million prevalent cases of sickle cell anemia worldwide, and forecasts that number to increase to 3.5 million prevalent cases by 2026.
Africa is estimated to have had 2.7 million prevalent cases of sickle cell anemia in 2017, accounting for 85% of worldwide cases. The approved drugs in the sickle cell anemia space target selectins, fetal hemoglobin, nicotinamide adenine dinucleotide, and hemoglobin. These drugs are administered via the oral route, except for Adakveo, which is available as an intravenous formulation.
The majority of industry-sponsored drugs in active clinical development for sickle cell anemia are spread evenly across Phase I and Phase II, with two drugs in Phase III.
Therapies in development for sickle cell anemia focus on a wide variety of targets. All the pipeline drugs are administered via the intravenous or oral routes.
High-impact upcoming events for drugs in the sickle cell anemia space comprise an expected CHMP opinion for Adakveo, and topline Phase II trial results for Olinciguat.
The overall likelihood of approval of a Phase I hematology asset is 27.7%, and the average probability a drug advances from Phase III is 74.8%. Drugs, on average, take 9.1 years from Phase I to approval in the overall hematology space.
There have been 16 licensing and asset acquisition deals involving sickle cell anemia drugs during 2015-20. The $3,520m coexclusive license agreement made in 2017 between Vertex Pharmaceuticals and CRISPR Therapeutics for the development and commercialization of potential hemoglobinopathy treatments, including treatments for sickle cell disease, was the largest deal.
The distribution of clinical trials across Phase I-IV indicates that the majority of trials for sickle cell anemia have been in the early and mid-phases of development, with 80% of trials in Phase I-II, and only 20% in Phase III-IV.
The US has a substantial lead in the number of sickle cell anemia clinical trials globally. The UK leads the major EU markets, while Lebanon has the top spot in Asia.
Clinical trial activity in the sickle cell anemia space is dominated by completed trials. Global Blood Therapeutics has the highest number of completed clinical trials for sickle cell anemia, with 10 trials.
Global Blood Therapeutics leads industry sponsors with the highest overall number of clinical trials for sickle cell anemia, followed by Novartis.
Key Topics Covered
2. KEY TAKEAWAYS
3. DISEASE BACKGROUND
- Bone marrow transplantation
- Blood transfusions
- Prophylactic therapy
- Pain management
6. MARKETED DRUGS
7. PIPELINE DRUGS
8. RECENT EVENTS AND ANALYST OPINION
- CTX001 for Sickle Cell Anemia (November 19, 2019)
- Rivipansel for Sickle Cell Anemia (August 2, 2019)
- Olinciguat for Sickle Cell Anemia (April 1, 2019)
- Oxbryta for Sickle Cell Anemia (December 3, 2018)
- RVT-1801 for Sickle Cell Anemia (December 3, 2018)
- Zynteglo for Sickle Cell Anemia (December 3, 2018)
- BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
9. KEY UPCOMING EVENTS
10. KEY REGULATORY EVENTS
- Global Blood Therapeutics' Oxbryta Approved Broadly For Sickle Cell Disease
- FDA Approval For Novartis's Sickle Cell Treatment Adakveo
- Emmaus: We Might Stand A Better Chance In Europe Without The EMA
- Global Blood Therapeutics' Voxelotor On Track For 2020 Sickle Cell Shakeup
11. PROBABILITY OF SUCCESS
12. LICENSING AND ASSET ACQUISITION DEALS
- Global Blood Partners With Syros For Sickle Cell, Beta Thalassemia Therapies
- Vertex Partners With Molecular Templates To Enhance Stem Cell Candidates
- Emmaus Grants Taiba Rights To Endari In MENA Nations
13. PARENT PATENTS
14. CLINICAL TRIAL LANDSCAPE
- Sponsors by status
- Sponsors by phase
- Recent events
- CRISPR Therapeutics
- Global Blood Therapeutics
- Vertex Pharmaceuticals
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