July 29, 2024

The need-to-know this morning

GSK and the venture capital firm Flagship Pioneering have created a research collaboration to develop new medicines, focused initially on respiratory disease and immunology.
Syndax Pharmaceuticals said the FDA extended the review of its drug to treat patients with leukemia by three months, to Dec. 26.
The FDA approved a screening blood test to detect colon cancer made by Guardant Health.

gene therapy

Sarepta sought to quiet gene therapy criticism

Sarepta Therapeutics pressed a patient advocacy group to censor a video that included criticism of the company's $3.2 million gene therapy for Duchenne muscular dystrophy, STAT's Adam Feuerstein reports exclusively. Specifically, the video in question showed the mother of a teenager with the degenerative disease confronting a company executive at a recent conference about a lack of efficacy and safety data on Sarepta's treatment, Elevidys.

"You're asking us to put everything on the line but you give us nothing in return," the woman, Catherine Collins, said at the event sponsored by Parent Project Muscular Dystrophy, which receives funding from Sarepta.

A Sarepta spokesperson confirmed the Cambridge, Mass.-based company later asked PPMD to either edit the video or remove it from the group's site entirely. The spokesperson denied Collins' claims that Sarepta had threatened legal action if the advocacy group did not comply.

alzheimer's

Neurologists at odds over new Alzheimer's drugs

There's a sharp divide among neurologists over the new amyloid-targeting Alzheimer's treatments — as evidenced at this year's Alzheimer's Association International Conference. A recent survey showed that 59% of neurologists would prescribe drugs likes lecanemab and donanemab. But nearly half remain skeptical, prioritizing cognitive and functional benefits over plaque reduction. Last week, European regulators said that lecanemab should be rejected due to its potentially dangerous side effects.

"Narrowing the divide in Alzheimer's treatment is crucial to avoid inconsistent practices and care, which prevent many eligible people from accessing potentially transformative medications," opine psychiatrists Murali Doraiswamy of Duke University and Lon Schneider of the University of Southern California. "By making more clinical trial data available, pharmaceutical companies can help restore public trust and enable health care providers to overcome the knowledge gaps that cause disparities in treatment recommendations."

drug pricing

Mark Cuban's company could help shortages more than prices

The Mark Cuban Cost Plus Drug Company tries to tackle two pharmaceutical crises: the high cost of drugs, and the extreme shortages in their availability. However, its focus on off-patent generic drugs, which are already inexpensive, means that the serial entrepreneur's efforts won't make a huge dent in overall drug prices — particularly for the brand name drugs that dominate Medicare spending, opines University of Pennsylvania health policy expert Ezekiel J. Emanuel.

While the company's direct-to-consumer model removes costly intermediaries and offers transparent pricing, it doesn't always provide the the lowest prices compared to competitors. Still, Emanuel says, the company could make a meaningful impact by improving price transparency and addressing generic drug shortages through domestic manufacturing.

marketing

Kaleo reprimanded by FDA for influencer post

The FDA has flagged a social media post from influencer Brittany Mahomes, saying that it contained "false or misleading" information about the Auvi-Q epinephrine autoinjector made by Kaleo. Specifically, it said that the post glowed about the benefits of Auvi-Q, but didn't include any risk information about the product.

Mahomes, who has 2 million followers on Instagram, spoke of how her infant and toddler have severe food allergies — and that a physician prescribed Auvi-Q as it's "the only epinephrine autoinjector out there for infants and toddlers." Although she did refer viewers to another page "for important safety information," regulators determine that wasn't sufficient.

The FDA called on Kaleo to quickly address these concerns and document any similar promotional communications, or it would have to stop distributing the Auvi-Q autoinjector.

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