pharmaphorum July 25, 2024
There has been a gene therapy on the market for people with the bleeding disorder haemophilia A since 2022, but take-up has been very slow. Pfizer and Sangamo are hoping for a faster trajectory with their experimental treatment.
The partners’ one-shot giroctocogene fitelparvovec therapy has cleared a phase 3 trial, showing it was better than regular prophylaxis injections with Factor VIII (FVIII) replacement therapies at reducing bleeding episodes, and could now be heading for regulatory review.
In their sights is first-to-market Roctavian (valoctocogene roxaparvovec) from BioMarin, which was approved in Europe in 2022 and the US last year, but has seen glacial sales growth, coming in at $800,000 in the first quarter of the year. Its list price in the...
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