Children born with a rare disease that makes their skin as fragile as a butterfly’s wings, leaving them vulnerable to frequent wounding and constant pain, will finally have access to a new treatment that aims to fix the genetic cause of the condition.
The therapy, known as Vyjuvek and made by Krystal Biotech, was approved by the FDA on Friday afternoon for treating patients six months or older with dystrophic epidermolysis bullosa, or DEB. The drug is a long-awaited solution for many families afflicted by the hellish condition, for which there are no approved treatments in the US.
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Genetic medicine is poised to unlock more value for patients and investors. In the latest episode of RBC’s Pathfinders in Biopharma podcast, Biotechnology Analyst Luca Issi highlights the most promising technologies.
Key points
Disruptive technologies emerging in genetics medicine include mRNA – the power behind successful COVID-19 vaccines, now being trialled for melanoma treatment.
After many setbacks, siRNA technology is also starting to deliver new treatments, with potential in areas such as cardiomyopathy.
Gene editing is being trialled against conditions such as sickle cell disease: early data on combating the risks associated with this technology is encouraging.
With an estimated 27 genetic medicines already approved, companies who can combine the right choice of intervention with strong execution stand to benefit.
An FDA independent advisory panel of infectious-disease and vaccine experts voted in general support of recommending Pfizer’s RSV vaccine, Abrysvo, to protect infants from the respiratory virus.
After listening to presentations from scientists from the Centers for Disease Control and Prevention, the FDA, and Pfizer, the panel voted on two questions if the data support the effectiveness and safety of the vaccine for this population. The panel unanimously voted 14-0 in support of efficacy, and 10-4 in favor of the safety data.
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“Given the current situation, to avoid stimulating further demand for this medicine, we’re pausing some key Wegovy promotional efforts,” a Novo Nordisk spokesperson said via email.
In a closely fought patent battle between PCSK9 rivals Amgen and Sanofi/Regeneron, the Supreme Court ruled unanimously on Thursday that Amgen cannot monopolize the market for any and every antibody that binds to areas of the sweet spot of PCSK9.
The win for Sanofi and Regeneron is a setback for companies like Amgen who are looking to patent broad categories of antibodies, but cannot produce each of the antibodies that might fall into such a category.
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Gal made the leap from top Wall Street analyst — and someone I was always careful to read closely — to Novartis insider at a time the big player is reorganizing and rethinking how it works as a global pharma company. And I’m looking forward to picking his brains during our session, which will focus — in a variety of ways — on the key elements biotechs need to thrive in today’s none-too-copacetic environment.
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The startup, Comanche Biopharma, has been operating mostly in stealth mode until this point, and is developing a one-time therapy that it hopes will work for about 90% of preeclampsia cases, said chief medical officer Allison August, a former VP of clinical development for infectious diseases at Moderna. Felix was also recently named a board member.
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A secretive biotech startup developing a new form of gene editing is starting to give curious scientists a peek at its pipeline — without fully divulging how its closely scrutinized technology works.
On Friday, Tessera Therapeutics announced preliminary data from five preclinical programs using its “gene writers” to tweak specific letters or insert entirely new sequences of DNA. The techniques are part of a larger movement in the gene editing field to develop tools that could treat nearly any conceivable genetic disease, and Tessera is one of its top contenders.
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The members of the FDA’s Gastrointestinal Drugs Advisory Committee examined two questions key to the drug’s approval. They voted 12 -2 (with two abstentions) that the benefits don’t outweigh the risks in NASH patients with stage 2 or 3 fibrosis, and 15 to 1 that an FDA decision should be deferred until additional data from the ongoing pivotal trial comes in.
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This content was originally published here.
