Drug Pipeline
31 drugs associated with Hemophilia B, severe
| Drug | Phase | Sponsor | |
|---|---|---|---|
BeneFIX Coagulation factor IX exogenous protein | Phase 3 | Wyeth is now a wholly owned subsidiary of Pfizer | ↗ |
BPA prophylaxis | Phase 3 | Genzyme, a Sanofi Company | ↗ |
Bypassing agents Toll-like receptor 2 antagonist | Phase 3 | Genzyme, a Sanofi Company | ↗ |
factor concentrates | Phase 3 | Genzyme, a Sanofi Company | ↗ |
Factor IX (FIX) Coagulation factor IX exogenous protein | Phase 3 | CSL Behring | ↗ |
fitusiran QFITLIA is a double-stranded siRNA that causes degradation of AT messenger RNA (mRNA) through RNA interference, reducing plasma AT levels. | Phase 3 | Genzyme, a Sanofi Company | ↗ |
IB1001 Following oral administration, levacetylleucine is taken up via ubiquitously expressed monocarboxylate transporters and distributed to all body tissues. It enters enzyme-controlled pathways that correct metabolic dysfunction and improves adenosine triphosphate (ATP) energy production. The normalization of energy metabolism ameliorates mitochondrial and lysosomal dysfunction and leads to a reduction in the storage of unesterified cholesterol and sphingolipids. Levacetylleucine has also been shown | Phase 3 | Cangene Corporation | ↗ |
PF-06838435/ fidanacogene elaparvovec Coagulation factor IX exogenous gene | Phase 3 | Pfizer | ↗ |
ReFacto AF | Phase 3 | Wyeth is now a wholly owned subsidiary of Pfizer | ↗ |
rFIX-R | Phase 3 | Wyeth is now a wholly owned subsidiary of Pfizer | ↗ |
rFIXFc Coagulation factor IX exogenous protein | Phase 3 | Bioverativ Therapeutics Inc. | ↗ |