- The author estimates that in 2016, there were 3.2 million prevalent cases of sickle cell anemia worldwide, and forecasts for 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.
- 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 targets such as hemoglobin and selectins, adenosine diphosphate P2Y12 receptor, nuclear factor-kappa beta, and nuclear erythroid 2-related factor 2. More than half of the pipeline drugs in mid-to-late-stage development for sickle cell anemia are administered via the intravenous/oral route.
- Endari (levoglutamide), 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 high impact upcoming events in the sickle cell anemia space include top-line Phase III trial results for GBT440 and Altemia. Licensing and asset acquisition activity involving sickle cell anemia drugs has been weak during 2012-17, with only eight deals occurring over this time period. 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 2012-17. Brilinta sales have ranked highest in the sickle cell anemia space during 2012-16.
- The clinical trials distribution across Phase I-IV indicates that the majority of the drugs are in early and mid phases of development with 81% of trials in Phase I-II, and only 19% in Phase III-IV.
- While the US has a substantial lead in the number of sickle cell anemia clinical trials globally, the UK leads the major EU markets. Clinical trial activity in the sickle cell anemia space is dominated by completed trials, with only 30% of ongoing trials. Pfizer, Eli Lilly and Novartis have the highest number of completed clinical trials for sickle cell anemia number of clinical trials for cervical cancer, followed by Roche.
2 DISEASE BACKGROUND
- Bone marrow transplantation
- Blood transfusions
- Prophylactic therapy
- Pain management
5 PIPELINE DRUGS
6 MARKETED DRUGS
- Approvals by country
8 KEY REGULATORY EVENTS
- US FDA approves sickle cell therapy, Endari
- Generex signs LOI to acquire 51% of Emmaus Life Sciences
- Novartis buys Selexys as competitors stumble in sickle cell
11 CLINICAL TRIAL LANDSCAPE
- Sponsors by status
- Sponsors by phase
- Phase III trial of Mast Therapeutics product in sickle cell disease failed
- Prescription information
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: Key upcoming events in sickle cell anemia
Figure 5: Licensing and asset acquisition deals in sickle cell anemia
Figure 6: Parent patents in sickle cell anemia
Figure 7: Clinical trials in sickle cell anemia
Figure 8: Top 10 drugs for clinical trials in sickle cell anemia
Figure 9: Top 10 companies for clinical trials in sickle cell anemia
Figure 10: Trial locations in sickle cell anemia
Figure 11: Sickle cell anemia trials status
Figure 12: Sickle cell anemia trial sponsors, by phase
List of Tables
Table 1: Prevalent cases of sickle cell anemia, 2016-25
Table 2: Pipeline drugs for sickle cell anemia in the US
Table 3: Marketed drugs for sickle cell anemia
Table 4: Approvals by country for sickle cell anemia