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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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Tenaya’s cardiomyopathy gene therapy gets fast track boost

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying gene therapy for MYBPC3-associated HCM.

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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. For now, the list price hasn’t been set and will depend on discussions with reimbursement authorities, according to the biotech.

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Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod

XTalks

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Roctavian, the First Hemophilia A Gene Therapy, Attains EC Approval

XTalks

The hemophilia A gene therapy Roctavian delivers a functional gene to enable the body to make an essential blood clotting protein on its own. A lack of sufficient functioning factor VIII and factor IX blood clotting proteins would result in the development of hemophilia A and hemophilia B, respectively.

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Cell and Gene Therapy Bioassay – A Prerequisite for the ATMPs Development

Roots Analysis

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.

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UCB revs up its gene therapy drive with Handl acquisition

pharmaphorum

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.