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If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.
The price is less than the $3.5 million announced last year for a similar gene therapy for hemophilia B, a less common form of the disease. Like most medicines in the U.S., the new treatment will ...
It priced the one-time therapy, Roctavian, at $2.9 million. Pivotal trial results showed that Roctavian reduced bleeding events, but its durability is not known, and the company said it would ...
Giroctocogene fitelparvovec (PF-07055480) is an experimental gene therapy for hemophilia A via a recombinant adeno-associated virus serotype 6-based vector. [1]
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
ASC618 (also known as AAV2/8 HCB-ET3-LCO BDD FVIII) is an experimental gene therapy for Hemophilia A. It was developed by Applied StemCell Therapeutics and is delivered via a hybrid of adeno-associated virus types 2 and 8. The gene therapy is hoped to be more effective than earlier gene therapies for hemophilia A. [1] [2] [3]
More recently, the first hemophilia gene therapy approved by the U.S. Food and Drug Administration was priced by CSL Behring at $3.5 million; 26 more gene therapies are in late-stage development ...
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