Remove Development Remove FDA Approval Remove Genetic Disease
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Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment

XTalks

Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDA approval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.

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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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BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

XTalks

BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

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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.

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Sanofi’s Enzyme Replacement Therapy Xenpozyme Wins FDA Approval for Rare Disease ASMD

XTalks

The FDA approval was based on data from the randomized, double-blind, placebo-controlled ASCEND study involving 31 adult patients. The FDA approval came ahead of its October 3 target date, which had been extended by three months. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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The downside of VC funding for biotech

World of DTC Marketing

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).