FDA Accepts Pfizer's (PFE) Hemophilia B Gene Therapy Filing
Pfizer PFE announced that its regulatory submissions in the United States and European Union (“EU”) seeking approval for hemophilia B gene therapy treatment fidanacogene elaparvovec have been accepted for review.
While a final decision of the FDA is expected in second-half 2024, a decision from the European Medicines Agency (EMA) is anticipated in mid-2024.
The regulatory filings for fidanacogene elaparvovec in both markets are based on data from the phase III BENGENE-2 study. The study achieved its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (“ABR”) of total bleeds post infusion with fidanacogene elaparvovec compared with those study participants who were administered the current standard-of-care (“SOC”) of treatment, i.e., a prophylaxis regimen with Factor IX (“FIX”).
Year to date, Pfizer’s shares have lost 28.9% against the industry’s 0.7% growth.
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Fidanacogene elaparvovec is a one-time gene therapy product consisting of a bio-engineered AAV capsid and a high-activity human coagulation FIX gene. The treatment aims to allow patients to produce FIX themselves without the need to receiving exogenous FIX.
Pfizer has in-licensed fidanacogene elaparvovec gene therapy from Spark Therapeutics, a subsidiary of Roche RHHBY. The deal was entered in 2014, five years prior to the acquisition of Spark Therapeutics by Roche in 2019. Per the terms of the licensing deal with the Roche-subsidiary, Pfizer is solely responsible for all pivotal studies and the potential commercialization of fidanacogene elaparvovec.
If approved, Pfizer’s gene therapy will face competition from Hemgenix, a one-time gene therapy that was approved by the FDA last November for the treatment of hemophilia B in patients older than 18 years. Hemgenix has been developed by uniQure QURE in partnership with CSL Behring, the latter being a subsidiary of CSL Limited. Following approval, uniQure/CSL’s Hemgenix became the first FDA-approved gene therapy for treating hemophilia B. In February, the treatment also received approval in the EU.
The FDA’s approval of uniQure/CSL’s Hemgenix was based on results from the pivotal HOPE-B study, which demonstrated that following the infusion with the gene-therapy, patients produced mean FIX activity of 39% at six months and 36.7% at 24 months. The mean adjusted ABR for all bleeds was also reduced by 54% after seven to 18 months of administering Hemgenix. The clinical development program for Hemgenix was initially led by uniQure, which was subsequently transferred to CSL after it acquired global rights to commercialize the treatment.
Other than fidanacogene elaparvovec, Pfizer is developing another gene therapy — giroctocogene fitelparvovec — to treat hemophilia A. The therapy is being developed in collaboration with Sangamo Therapeutics SGMO. Sangamo and Pfizer are evaluating giroctocogene fitelparvovec in the phase III AFFINE study. Pfizer/Sangamo expects to report pivotal data from the AFFINE study by mid-2024. Based on this data, Sangamo/Pfizer expect to start regulatory submissions for the gene therapy candidate in the EU and United States during second-half 2024.
Apart from gene therapies, Pfizer is also evaluating marstacimab, an investigational anti-tissue factor pathway inhibitor, to treat hemophilia A and B patients — with and without inhibitors — in the ongoing phase III BASIS study. Last month, Pfizer announced that the BASIS study achieved its primary endpoints. Data from the study showed treatment with marstacimab achieved statistically significant and clinically relevant reduction in ABR compared with the current SOC treatment for hemophilia patients, during a 12-month period.
A rare genetic blood disorder, hemophilia is caused by a missing clotting protein (FVIII in case of hemophilia A and FIX in case of hemophilia B), which prevents normal blood clotting. If the blood does not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.
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