This Market Spotlight 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.

Key Takeaways

• Datamonitor Healthcare 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 predominantly target fetal hemoglobin, with the exception of Endari, which targets nicotinamide adenine dinucleotide. These drugs are administered via the oral route.
• The largest proportion of industry-sponsored drugs in active clinical development for sickle cell anemia are in Phase I, with only one drug in the NDA/BLA phase.
• Therapies in development for sickle cell anemia focus on a wide variety of targets. The majority of pipeline drugs are administered via the intravenous or oral routes, with one product also being tested in a subcutaneous formulation.
• High-impact upcoming events in the sickle cell anemia space comprise an expected PDUFA date for crizanlizumab, topline Phase III trial results for rivipansel, and topline Phase II trial results for olinciguat.
• The overall likelihood of approval of a Phase I hematology asset is 30%, and the average probability a drug advances from Phase III is 76.3%. Drugs, on average, take 9.1 years from Phase I to approval in the overall hematology space.
• There have been 15 licensing and asset acquisition deals involving sickle cell anemia drugs during 2014–19. 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 79% of trials in Phase I–II, and only 21% 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 and Pfizer have the highest number of completed clinical trials for sickle cell anemia, with nine trials each.
• Global Blood Therapeutics leads industry sponsors with the highest overall number of clinical trials for sickle cell anemia, followed by Novartis.