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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

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ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

Bio Pharma Dive

Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.

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PTC wins FDA approval for first brain-delivered gene therapy Kebilidi

Fierce Pharma

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.

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CSL closes on EU approval for haemophilia B gene therapy

pharmaphorum

CSL’s gene therapy for haemophilia B has been recommended for approval by the EMA’s human medicine committee, setting up a decision by the European Commission early next year. The two companies are also planning to sell etranacogene dezaparvovec under the Hemgenix brand in the EU, assuming its gets approval.

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Pfizer quickly expands its hemophilia B gene therapy's reach with EU approval

Fierce Pharma

Three months after bagging FDA approval, Pfizer’s hemophilia B gene therapy has been cleared to expand its reach across the pond. | The company's first gene therapy will debut in Europe as Durveqtix. the drug is approved under the brand name Beqvez. In the U.S.,

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UPDATED: After delays, Sarepta's DMD gene therapy Elevidys finally crosses FDA finish line at $3.2M

Fierce Pharma

After several delays and a narrow advisory committee vote, Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy has finally won an accelerated approval. It's now branded as Elevidys.

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Protagen, BioAnalytix, and GeneWerk unveil combined CRO business: ProtaGene

BioPharma Reporter

Protagen, BioAnalytix, and GeneWerk this week announced their unified operations and re-brand as ProtaGene, a CRO partner for the biologics and cell and gene therapy developers, from discovery to commercialization.