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Sanofi’s Qfitlia Gets FDA Approval as First siRNA Therapy for Hemophilia

XTalks

XTALKS WEBINAR: Keys to Success in Clinical Trials: A Strategic Guide for Biotechs and Startups Live and On-Demand: Thursday, May 22, 2025 , at 11am EDT (5pm CEST / EU-Central) Register for this free webinar to learn how biotechs can navigate endpoint challenges in clinical trials and accelerate their path to drug approval.

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Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

XTalks

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

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US FDA approves Krystal Biotech’s Vyjuvek for DEB

Pharmaceutical Technology

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

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STAT+: FDA approves first treatment for Rett syndrome, a genetic neurological disease

STAT News

The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a genetic disease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.

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Hympavzi (Marstacimab): FDA Approves First Once-Weekly and Pfizer’s Second Hemophilia Therapy

XTalks

Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.

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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

XTalks

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

XTalks

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. To date, over 800 patients have received the therapy in clinical trials and real-world settings. Late in 2024, Elevidys was recognized among Times Best Innovations.