Pfizer stops commercialization of hemophilia gene therapy Beqvez
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작성자 Chance Spalding 댓글 0건 조회 19회 작성일 25-06-26 07:39본문
By Christy Santhosh
Feb 20 (Reuters) - Pfizer said on Thursday it will stop global development and commercialization of its hemophilia gene therapy, Beqvez, citing soft demand from patients and their doctors.
Beqvez is a one-time therapy that was approved in the U.S. last year, for the treatment of adults with moderate to severe hemophilia B.
The company said the discontinuation was due to several reasons, including limited interest in gene therapies for the bleeding disorder.
However, the company said it will redirect its time and resources towards Hympavzi, an injectable drug approved in October to prevent or reduce bleeding episodes in hemophilia A or B patients aged 12 years and older.
This is Pfizer's second therapy discontinuation for the bleeding disorder in recent months after it terminated its co-development agreement with Sangamo Therapeutics for a hemophilia A gene therapy, citing slow uptake for such therapies in patients.
Doctors have said that high cost, logistical issues and the prospect of potential treatment advances were holding back the adoption of gene therapies for hemophilia.
Beqvez was priced at $3.5 million in the U.S. during its launch - the same price tag as Australian drugmaker CSL Ltd's rival gene therapy Hemgenix and higher than BioMarin Pharmaceutical's hemophilia A gene therapy Roctavian, which was priced at $2.9 million per patient.
Experts have also expressed their concerns about the long-term risks associated with the durability of the replacement genes used in these therapies.
People with hemophilia have a defect in a gene that regulates production of proteins called clotting factors. It primarily affects men and causes spontaneous and potentially severe bleeding after injuries or surgery.
The disorder impacts an estimated 33,000 men in the United States, according to government data.
(Reporting by Christy Santhosh in Bengaluru; Editing by Alan Barona)
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Feb 20 (Reuters) - Pfizer said on Thursday it will stop global development and commercialization of its hemophilia gene therapy, Beqvez, citing soft demand from patients and their doctors.
Beqvez is a one-time therapy that was approved in the U.S. last year, for the treatment of adults with moderate to severe hemophilia B.
The company said the discontinuation was due to several reasons, including limited interest in gene therapies for the bleeding disorder.
However, the company said it will redirect its time and resources towards Hympavzi, an injectable drug approved in October to prevent or reduce bleeding episodes in hemophilia A or B patients aged 12 years and older.
This is Pfizer's second therapy discontinuation for the bleeding disorder in recent months after it terminated its co-development agreement with Sangamo Therapeutics for a hemophilia A gene therapy, citing slow uptake for such therapies in patients.
Doctors have said that high cost, logistical issues and the prospect of potential treatment advances were holding back the adoption of gene therapies for hemophilia.
Beqvez was priced at $3.5 million in the U.S. during its launch - the same price tag as Australian drugmaker CSL Ltd's rival gene therapy Hemgenix and higher than BioMarin Pharmaceutical's hemophilia A gene therapy Roctavian, which was priced at $2.9 million per patient.
Experts have also expressed their concerns about the long-term risks associated with the durability of the replacement genes used in these therapies.
People with hemophilia have a defect in a gene that regulates production of proteins called clotting factors. It primarily affects men and causes spontaneous and potentially severe bleeding after injuries or surgery.
The disorder impacts an estimated 33,000 men in the United States, according to government data.
(Reporting by Christy Santhosh in Bengaluru; Editing by Alan Barona)
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