The European Medicines Agency’s human medicines committee recommended 14 new drugs for approval this week, including Pfizer’s gene therapy for hemophilia B.
The approval recommendations include:
→ Pfizer’s therapy, which would be marketed as Durveqtix in the EU, was already approved by the FDA in April and is marketed as Beqvez in the US. The CHMP recommended a conditional authorization for the drug, so Pfizer will need to show more positive data in the future. The one-time therapy will be priced at $3.5 million, the same as its competitor, CSL’s Hemgenix. In clinical trials, the drug reduced yearly bleeding rates by 71% compared to before treatment when they were taking prophylactic factor IX therapy; 60% of patients had their bleeds eliminated completely. The CHMP also noted in its summary that most patients didn’t need factor IX replacement therapy for at least two years after treatment.
→ The CHMP followed in the FDA’s footsteps again with Takeda’s Adzynma, recommending that the EMA authorize the enzyme replacement therapy for both children and adults with a rare blood clotting disorder called congenital thrombotic thrombocytopenic purpura. The US agency approved it last year based on data that showed it reduced instances of low blood platelet counts.
→ A rare eye disease that mostly impacts people who wear contact lenses also scored a recommendation: Akantior, from the Italian company SIFI, would be the first-ever approved treatment for the disease. According to SIFI, the disease called acanthamoeba keratitis begins as an infection in the cornea caused by an amoeba. It can lead to vision or eye loss and often requires corneal transplants.
→ AstraZeneca’s influenza vaccine Fluenz for children and adolescents got a thumbs-up, as did Valneva’s chikungunya vaccine Ixchiq. The FDA approved Ixchiq last year, and if the EMA authorizes it, it will be the first chikungunya vaccine in the EU to protect adults against disease caused by the virus, which is transmitted from infected mosquitoes.
The committee voted in favor of seven label expansions for drugs that are already approved: Dupixent, Eliquis, Kinpeygo, Livmarli, Skyrizi, Tagrisso and Tevimbra, and also recommended authorization for one biosimilar and five generics.