May 28, 2024

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Pfizer’s hemophilia gene therapy receives FDA acceptance for market application

Pfizer’s hemophilia gene therapy receives FDA acceptance for market application


Pfizer’s hemophilia gene therapy receives FDA acceptance for market application: Patient annual bleeding rates reduced by 71%.


Recently, Pfizer Inc. (NYSE: PFE) announced that the FDA has accepted its Biologics License Application (BLA) for fidanacogene elaparvovec, a gene therapy for the treatment of adult patients with hemophilia B. The final review date is set for the second quarter of 2024.

Concurrently, the Marketing Authorization Application (MAA) for fidanacogene elaparvovec has also been accepted and is undergoing review by the European Medicines Agency (EMA).


Pfizer's hemophilia gene therapy receives FDA acceptance for market application: Patient annual bleeding rates reduced by 71%.




Hemophilia is a rare disease characterized by the lack of clotting proteins (coagulation factors), which prevents blood from clotting properly.

Hemophilia is classified into types A (accounting for 80-85%), B (accounting for 15-20%), and a very small number of cases as type C, depending on the specific clotting protein deficiency.


According to data from the World Federation of Hemophilia, there were over 38,000 individuals globally living with hemophilia B in 2021.

Patients with hemophilia B lack factor IX (FIX), and the current standard of care requires regular intravenous injections of FIX derived from plasma or recombinant sources to control and prevent bleeding episodes.


Fidanacogene elaparvovec is a novel investigational gene therapy that contains a high-activity variant of the factor IX gene encapsulated in an adeno-associated virus (AAV) protein shell.

The therapy aims to provide long-term FIX production in patients with a single treatment, eliminating the need for regular intravenous FIX injections.


In December 2014, Pfizer acquired a license for fidanacogene elaparvovec from Spark Therapeutics (now owned by Roche).

Under the agreement, Pfizer is responsible for key research, regulatory matters, and potential global commercialization of this investigational gene therapy.


The submission to the FDA and Europe is based on the results of the Phase III study BENEGENE-2 (NCT03861273) conducted by Pfizer last year. In the trial, a single dose of treatment resulted in an average 71% reduction in the annual bleeding rate for participants compared to the baseline.

The therapy also reduced the frequency of bleeding requiring medical intervention and the need for standard prophylactic infusions.


Fidanacogene elaparvovec has generally demonstrated good tolerability, consistent with the results from Phases I and II.

However, to assess its long-term safety and efficacy, clinical trial participants will be followed for up to 15 years, including the 6-year follow-up from the BENEGENE-2 study and an additional 9 years as part of the Phase III study.


If approved, fidanacogene elaparvovec will become the second gene therapy for hemophilia B, following Hemlibra, which provides the FIX gene and is developed by Dutch biotechnology company UniQure and marketed by Australian pharmaceutical company CSL Behring.

CSL priced their treatment at $3.5 million, making it the most expensive one-time therapy in the United States.


For Pfizer, obtaining approval also indicates that its investment in gene therapy is starting to pay off.

Over the past decade, the company has introduced a portfolio of projects through acquisitions and advanced them through clinical trials.

In addition to fidanacogene elaparvovec from Spark Therapeutics, Pfizer entered the field of Duchenne muscular dystrophy after acquiring Bamboo Therapeutics and is jointly developing a gene therapy for hemophilia A with Sangamo Therapeutics.


All of the above are currently in Phase III clinical trials but face competition from rivals. Just last week, Sarepta Therapeutics received FDA approval for a gene therapy for Duchenne, and BioMarin Pharmaceutical’s gene therapy for hemophilia A, Roctavian, has been approved in Europe and may soon receive approval from US regulatory authorities.



(source:internet, reference only)

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Important Note: The information provided is for informational purposes only and should not be considered as medical advice.