Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PF-06838435/ fidanacogene elaparvovec · 2 trials · 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 | - |
| PF-06838435 Dose-Escalation | EXPERIMENTAL | Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken. |
| Name | Type | Description |
|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | BIOLOGICAL | Gene Therapy |
| PF-06838435 (formerly SPK-9001) | BIOLOGICAL | Gene Therapy: A novel, bioengineered adeno-associated viral vector carrying human factor IX variant |
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...
PF-06838435, also known as fidanacogene elaparvovec, is an investigational gene therapy being developed for the treatment of hemophilia B. It is intended for adult males with moderately severe to severe hemophilia B. The drug is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.
PF-06838435 is a gene therapy designed to deliver a functional copy of the factor IX gene to patients with hemophilia B. Hemophilia B is caused by a deficiency in factor IX, a protein essential for blood clotting. By providing the correct gene, the therapy aims to enable the body to produce factor IX and reduce bleeding episodes.
PF-06838435 is being developed by Pfizer, Inc., a multinational pharmaceutical company listed on the New York Stock Exchange under the ticker symbol PFE. Pfizer is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for hemophilia B.
PF-06838435 is currently in Phase 3 clinical development. It has completed a Phase 2 trial and is now being evaluated in an active Phase 3 study. The drug is investigational and has not received FDA approval or any other regulatory approval for commercial use.
PF-06838435 has been studied in two clinical trials. The first, NCT03307980, was a Phase 2 study that has been completed, involving 21 participants. The second, NCT03861273, is an active Phase 3 trial with 51 participants. Both trials are studying the drug in adult males with hemophilia B.
Yes, PF-06838435 is the same as fidanacogene elaparvovec. PF-06838435 is the code name used during development, while fidanacogene elaparvovec is the international nonproprietary name. Both names refer to the same investigational gene therapy for hemophilia B.