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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. BioMarin previously said that the treatment’s European price would likely be around EUR1.5 million price tag.

FDA 111
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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. BioMarin has said Roctavian will be available at a ‘net’ price of around €1.5 million spread over five years.

FDA 109
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PharmaShots Weekly Snapshots (March 06 - 10, 2023)

PharmaShots

Vertex Receives the US FDA’s IND Clearance of VX-264 for the Treatment of Type 1 Diabetes Date: Mar 10, 2023 | Tags: Vertex, VX-264, Type 1 Diabetes, Regulatory, US, FDA, IND Alnylam Expands its Collaboration with Medison Pharma to Commercialize RNAi Therapies Date: Mar 10, 2023 | Tags: Alnylam, Medison Pharma, RNAi Therapies, Onpattro, patisiran, (..)

FDA 40
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Sanofi offers refunds for Cablivi in novel model for rebates

Pharmaceutical Technology

Other companies like BioMarin have already announced an intention to offer an outcomes-based warranty program for its hemophilia A drug Roctavian (valoctocogene roxaparvovec). In 2022, the number of risk sharing agreements peaked with 144 agreements being made in the pharmaceutical industry.