Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Fidanacogene elaparvovec · 1 trial · 1 indication
ABR = number of total bleeding episodes on study during the given time period) \*365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
| Arm | Type | Description |
|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | BIOLOGICAL | Gene Therapy |
Inclusion Criteria * Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study. * Documented moderately severe to severe hemophilia B (Factor IX activity \< =2%) * Previous experience with FIX t...
Top 8 of 9 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 15 | PHASE3 | Concizumab, Mim8, Denecimig, NNC0442-0344 A, turoctocog alfa pegol |
| Sanofi SA Sponsored ADR | SNY | 5 | PHASE3 | Fitusiran, efanesoctocog alfa |
| Pfizer Inc. | PFE | 8 | PHASE3 | Fidanacogene elaparvovec, Marstacimab |
| BioMarin Pharmaceutical Inc. | BMRN | 2 | PHASE3 | Valoctocogene roxaparvovec |
| Hoffmann-La Roche | RHHBY | 8 | PHASE3 | Emicizumab, NXT007, SPK-8011QQ, SPK-8011, SPK-8016 |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | REGV131, LNP1265 |
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 2 | PHASE1 | AskBio009 |
| Bayer | BAYRY | 6 | - | Damoctocog-alfa-pegol |
Fidanacogene elaparvovec is an investigational gene therapy being developed for the treatment of hemophilia B, a bleeding disorder caused by deficient clotting factor IX. It is designed as a one-time treatment approach and is currently in Phase 3 clinical development. It has not been approved by the FDA.
Fidanacogene elaparvovec is being studied for use in adult males with moderately severe to severe hemophilia B. The goal of the therapy is to deliver a functional copy of the factor IX gene so the body can produce factor IX on its own, potentially reducing the need for regular factor replacement infusions.
Fidanacogene elaparvovec targets the F9 gene, which encodes clotting factor IX. It is an exogenous gene therapy, meaning it delivers a working copy of the F9 gene into the patient's cells. The target class is an exogenous gene, and the aim is to restore factor IX production in hemophilia B patients.
Fidanacogene elaparvovec is being developed by Pfizer, Inc., which trades on the New York Stock Exchange under the ticker PFE. Pfizer is the sponsor of the clinical program evaluating this gene therapy in hemophilia B.
Fidanacogene elaparvovec is in Phase 3 clinical development. It is investigational and has not been approved by the FDA. The Phase 3 trial is currently active but not recruiting new participants.
Fidanacogene elaparvovec is being evaluated in the Phase 3 trial NCT03861273, titled a study to evaluate the efficacy and safety of factor IX gene therapy with PF-06838435 in adult males with moderately severe to severe hemophilia B. The trial has enrolled 51 participants across the United States, Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey, and the United Kingdom.
Yes, fidanacogene elaparvovec is also known as PF-06838435. Both names refer to the same investigational gene therapy developed by Pfizer for hemophilia B. The PF-06838435 designation is the company's internal compound code used during development.