- The author estimates that in 2016, 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 2025.
- Africa is estimated to have had 2.7 million prevalent cases of sickle cell anemia in 2016, accounting for 85% of worldwide cases. Endari, a patented drug treatment that involves an oral administration of L-glutamine, is the only US Food and Drug Administration approved drug for sickle cell anemia.
- The majority of industry-sponsored drugs in active clinical development for sickle cell anemia are in Phase II. Therapies in mid- and late-stage development for sickle cell anemia focus on a wide variety of targets. The majority of the pipeline drugs in mid-to-late-stage development for sickle cell anemia are administered via the intravenous or oral routes, with one product being tested in both subcutaneous and intravenous formulation.
- High-impact upcoming events in the sickle cell anemia space include topline Phase III trial results for voxelotor. There were eight licensing and asset acquisition activities involving sickle cell anemia drugs during 2013-18, three of which occurred in 2015. The $2,520m strategic research collaboration deal between Vertex Pharmaceuticals and CRISPR Therapeutics, for the discovery and development of drugs based on CRISPR-Cas9 technology, was the largest deal during the 2013-18 period.
- The clinical trials distribution across Phase I-IV indicates that the majority of the drugs are in 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, while the UK leads the major EU markets Clinical trial activity in the sickle cell anemia space is dominated by completed trials. Pfizer and Eli Lilly have the highest number of completed clinical trials for sickle cell anemia, with seven each.
- Global Blood Therapeutics leads industry sponsors with the highest number of clinical trials for sickle cell anemia, followed by Novartis and Pfizer.
Bone marrow transplantation
Approvals by country
KEY UPCOMING EVENTS
KEY REGULATORY EVENTS
US FDA approves sickle cell therapy, Endari
LICENSING AND ASSET ACQUISITION DEALS
Generex signs LOI to acquire 51% of Emmaus Life Sciences
Novartis buys Selexys as competitors stumble in sickle cell
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Phase III trial of Mast Therapeutics product in sickle cell disease failed
List of Figures
Figure 1: Pipeline drugs for sickle cell anemia in the US
Figure 2: Pipeline drugs for sickle cell anemia, by company
Figure 3: Pipeline drugs for sickle cell anemia, by drug type
Figure 4: Pipeline drugs for sickle cell anemia, by drug classification
Figure 5: Key upcoming events in sickle cell anemia
Figure 6: Licensing and asset acquisition deals in sickle cell anemia
Figure 7: Parent patents in sickle cell anemia
Figure 8: Clinical trials in sickle cell anemia
Figure 9: Top 10 drugs for clinical trials in sickle cell anemia
Figure 10: Top 10 companies for clinical trials in sickle cell anemia
Figure 11: Trial locations in sickle cell anemia
Figure 12: Sickle cell anemia trials status
Figure 13: Sickle cell anemia trial sponsors, by phase
List of Tables
Table 1: Prevalent cases of sickle cell anemia, 2016-25
Table 2: Marketed drugs for sickle cell anemia
Table 3: Approvals by country for sickle cell anemia
Table 4: Pipeline drugs for sickle cell anemia in the US