New data show BioMarin's troubled hemophilia gene therapy continues to fade, opening room for rivals
The effects of BioMarin’s vaunted gene therapy for hemophilia A are continuing to fade in the earliest treated patients, renewing questions about the long-term prospects for a drug the company had billed as a one-time cure.
At the International Society on Thrombosis and Haemostasis meeting Wednesday, BioMarin announced that, after five years, the first seven patients treated with the therapy’s high dose expressed a median of just 8.2% the amount of factor VIII that a healthy person would. That’s a small fraction of the 60.3% expression patients saw one year after receiving the therapy, known as valrox.
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