Pfizer’s hemophilia B gene therapy inches closer to regulatory approval
27 Jun 2023
Phase 3Gene TherapyLicense out/inClinical Result
Pfizer is nearing an approval of what could be its first marketed gene therapy, now that the Food and Drug Administration has begun reviewing the company’s request to approve an experimental treatment for hemophilia B.
Pfizer on Tuesday said the FDA accepted its application for the treatment, known as idanacogene elaparvovec, and plans to issue a decision in the second quarter of 2024. A regulatory review is also underway in Europe.
Pfizer submitted its application based on Phase 3 study results that were announced last year. In that trial, a single dose of the therapy lowered participants' yearly bleeding rates by an average of 71% compared to the study’s start. Treatment also reduced the frequency of bleeds that required medical intervention, as well as the need for standard preventive infusions.
If cleared, the treatment would follow UniQure and CSL Behring’s Hemgenix to become the second available gene therapy for hemophilia B, the rarer of the two main forms of the chronic blood disease. Both treatments are designed to deliver functional copies of the gene that instructs the body to make the clotting protein Factor IX.
For Pfizer, an approval would show that the money it’s invested in gene therapy is beginning to pay dividends. Over the last decade, the company has used dealmaking to bring in a group of programs and advance them through clinical testing. Pfizer gained the rights to its hemophilia B treatment, for instance, through a 2014 licensing deal with Spark Therapeutics, which is now owned by Roche. It acquired a Duchenne muscular dystrophy prospect when it acquired Bamboo Therapeutics, and is co-developing a gene therapy for hemophilia A with Sangamo Therapeutics.
All are now in Phase 3 testing, but face competitive threats from more advanced rivals. Sarepta Therapeutics won approval of a Duchenne gene therapy last week, while BioMarin Pharmaceutical’s hemophilia A gene therapy, Roctavian, is already cleared in Europe and could be approved by U.S. regulators by Friday.
Pfizer has had setbacks as well. Testing of its Duchenne and hemophilia A therapies were both temporarily halted due to safety concerns.
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