▎WuXiEdited by Kant Content Team

Today, the European Medicines Agency (EMA) recommended granting Pfizer's one-time gene therapy Durveqtix (fidanacogene elaparvovec) conditional marketing authorization for the treatment of adults with severe and moderately severe hemophilia B who have no factor IX (FIX) inhibitors and have not detected antibodies against the adeno-associated virus serotype Rh74 variant (AAVRh74var).

This recommendation is based on the results of an ongoing single-arm, open-label Phase 3 trial involving 45 adult male patients with moderate or severe hemophilia B who tested negative for AAVRh74var neutralizing antibodies and received a single intravenous infusion.Durveqtix。

The results showed that, compared with standard treatment, Durveqtix significantly reduced the frequency of bleeding.The annualized bleeding rate (ABR) was 1.44 for patients in the Durveqtix group and 4.50 for those receiving prophylactic therapy. Additionally, 60% of patients in the Durveqtix group remained free of bleeding events during the individual observation period (range: 2-4 years), compared to only 29% of patients who received routine prophylactic treatment during the lead-in period.After receiving Durveqtix treatment, the patient's consumption of preventive coagulation factor IX decreased by 92.4%.

▲ InIn the trial, patients receivedDurveqtixAfter infusion, FIX activity in the body remained stable (Image source: Reference [2]).

The most common side effect in the trial was elevated liver transaminase levels, but this could be managed with corticosteroid treatment. Patients receiving Durveqtix will undergo 15 years of follow-up, including 6 years in the pivotal clinical trial and an additional 9 years as part of a separate study, to monitor the long-term efficacy and safety of the gene therapy.

Hemophilia B is a life-threatening degenerative disease where patients lack clotting factors due to genetic mutations.IXPatients with this disease are prone to bleeding in the joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments include lifelong...Multiple regular intravenous infusions per week or monthFactor IX may require more than a hundred treatments per year to temporarily replace or supplement low levels of the clotting factor.

Durveqtix is a novel investigational gene therapy that contains a bioengineered adeno-associated virus capsid and a transgene encoding a highly active FIX variant.For patients with Hemophilia B, the goal of this gene therapy is to enable them to produce their own FIX protein through a one-time treatment, rather than requiring regular intravenous infusions of FIX as with the current standard treatment. In December 2014, Pfizer acquired this therapy from Spark Therapeutics for a $20 million upfront payment. Health Canada approved it in January this year.ApprovalThe therapy was launched in the U.S. in April this year, with FDA approval.ApprovalThis gene therapy, trade name Beqvez.

References:

[1] New gene therapy treatment for haemophilia B. Retrieved May 31, 2024 from https://www.ema.europa.eu/en/news/new-gene-therapy-treatment-haemophilia-b

[2] Efficacy and Safety of Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial. Retrieved January 4, 2024 from https://genetherapy.isth.org/efficacy-and-safety-of-fidanacogene-elaparvovec-in-adults-with-moderately-severe-or-severe-hemophilia-b-results-from-the-phase-3-benegene-2-gene-therapy-trial

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