April 26,PfizerAnnounced that the FDA has approved its gene therapyBEQVEZ(Fidanacogene elaparvovec-dzkt, SPK-9001) has been launched for the treatment of adult patients with moderate to severe hemophilia B. BEQVEZ is priced at $3.5 million, in line with previously approved gene therapies for hemophilia B.HemgenixThe prices are consistent.With the approval and application of BEQVEZ, Pfizer is launching an innovative warranty program based on the durability of patients' response to the treatment. This program aims to provide greater certainty for payers, maximize access to BEQVEZ for eligible patients, and offer financial protection for patients with unsatisfactory efficacy after treatment.BEQVEZ was initially developed bySpark Therapeutics(Has beenRocheAcquisition) development, a one-time gene therapy targeting FIX, containing a bioengineered adeno-associated virus (AAV) capsid (protein shell) and a highly active variant of the human FIX gene. In December 2014, Pfizer collaborated with Spark in a deal worth a total of $280 million to acquire global rights to this product.This approval is mainly based on the positive data from the Phase III BENEGENE-2 study (n=45). The results showed that within 12 months of receiving a single dose of BEQVEZ, the annualized bleeding rate (ABR) in patients significantly decreased compared to the standard prophylactic treatment group (receiving FIX drugs as preventive replacement therapy) (1.3 vs. 4.43, P<0.0001), meeting both non-inferiority and superiority criteria.In addition, Pfizer's TFPI monoclonal antibodymarstacimabIt has also been submitted for marketing approval in Europe and the US in December 2023, intended for the treatment of Hemophilia A and B. The FDA has set the PDUFA date for Q4 2024, and the EMA is expected to make an approval decision in Q1 2025.It is worth noting that,Novo NordiskDeveloped TFPI Monoclonal AntibodyconcizumabAlready Approved for Marketing in Canada and Japan, but Temporarily Rejected by the FDA. In its Q1 2023 financial report, Novo Nordisk disclosed that it had received a Complete Response Letter (CRL) from the FDA regarding the Biologics License Application (BLA) for concizumab for the prophylactic treatment of Hemophilia A and B with inhibitors. The FDA requested additional patient-related monitoring and dosing information to ensure concizumab is administered according to the predetermined regimen.Hemophilia B is a hereditary bleeding disorder caused by a congenital deficiency of FIX. The standard treatment for Hemophilia B is prophylactic infusion of FIX replacement therapy to temporarily substitute or supplement low levels of clotting factors. Despite preventive measures and regular intravenous infusions, many patients with moderate to severe Hemophilia B are still at risk of spontaneous bleeding episodes. According to statistics from the World Federation of Hemophilia, there are more than 38,000 cases of Hemophilia B globally.Copyright © 2023 PHARMCUBE. All Rights Reserved.
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