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On January 3, Pfizer stated:Canadian Health Regulatory Agency Approves Gene Therapy for Hemophilia BBeqvez（ fidanacogene elaparvovec）, this is the first time Pfizer has received approval for this type of gene therapy.The U.S. FDA is expected to make a regulatory decision in the second quarter of 2024.。This approval is based on a positive Phase III study, in which patients treated with fidanacogene elaparvovec experienced an average of 1.3 bleeding episodes in the year following the initial three months of treatment.Decreased by 71% compared to the bleeding rate before treatment。Hemophilia B is a hereditary bleeding disorder caused by a deficiency or insufficiency of blood coagulation factor IX. Patients face prolonged or excessive bleeding after injuries, surgeries, or dental procedures, which can lead to severe complications such as bleeding in the joints, muscles, or internal organs, including the brain.Patients need lifelong continuous injection of coagulation factors to suppress the disease.。The therapy wasIn 2014 from Spark Therapeutics(The company was wholly acquired by Roche in 2019) introduced this therapy, which uses adeno-associated virus (AAV) to deliver the human coagulation factor IX gene, thereby helping patients with hemophilia B to clot blood and avoid the frequent infusion of coagulation factors.Currently,Only Two Hemophilia Gene Therapies Approved for Marketing Worldwide。- One was approved by the U.S. FDA on November 22, 2022.CSL's AAV Gene Therapy Hemgenix(Etranacogene Dezaparvovec), for the treatment of adult Hemophilia B,Priced as high as 3.5 million US dollars, leaping to become the most expensive drug globally. Later inOctober 2023Approved by Health Canada.
- Another one was approved by the EU on August 24, 2022.BioMarin Pharmaceutical Hemophilia A Gene TherapyRoctavian(Valoctocogene Roxaparvovec), Pricing$2.9 million。
According to data from the World Federation of Hemophilia, more than 38,000 people worldwide were living with hemophilia B in 2021.In Canada, Hemophilia B affects over 700 people.。BeqvezInApproval in Canada, will compete with Hemgenix in treatment.Canadian Hemophilia Society (CHS) President Wendy Quinn said at a press conference on Wednesday: "The CHS will work closely with health authorities in provinces and territories across Canada to ensure that eligible patients in Canada can access gene therapy for hemophilia B as soon as possible."https://endpts.com/pfizer-gene-therapy-gets-hemophilia-b-approval-in-canada-setting-stage-for-csl-rivalry/
