Remove tag haemophilia
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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

Despite their potential multi-million-dollar sticker price, if haemophilia treatments by BioMarin and CSL Behring prove durable, they could help patients save money compared to current treatments, experts note. A fair price for CSL Behring’s haemophilia B treatment etranacogene dezaparvovec would be between $2.93–2.96

Safety 111
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CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. Haemophilia B is a rare condition. per dose, it is the most expensive single-use gene therapy in the US.

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Roctavian okay sets up another gene therapy test for Europe

pharmaphorum

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. million spread over five years.

FDA 109
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Advanced therapies and the high-profile pricing dilemma

pharmaphorum

However, with a price tag of €1.58 However, a recently released report from the organisation could blow the price tag of Zolgensma out of the water. Both of the treatments are gene therapies for haemophilia, and both are one-time therapies. How to navigate the new climate. At more than $2.1

Medicine 124