Vinay Prasad, top FDA official, exits under pressure

Vinay Prasad, a top official at the Food and Drug Administration, resigned yesterday following a series of controversial decisions regarding a gene therapy from Sarepta Therapeutics and pressure from right-wing activists.

Prasad was the head of the FDA division that regulates vaccines, gene therapies, and blood products. He was also the agency's chief medical and scientific officer, making him a top adviser to Commissioner Marty Makary.

Prasad's sudden exit comes amid escalating tensions and blowback involving a regulatory dispute with Sarepta, the maker of a gene therapy for Duchenne muscular dystrophy. The FDA forced Sarepta to halt shipments of the treatment, called Elevidys, following a series of patient deaths. But on Monday, the agency issued a statement saying that Sarepta could begin selling the treatment again.

After the Sarepta saga, Prasad lost support on the right. He was attacked by right-wing activist Laura Loomer, who called him a "progressive leftist saboteur," and he was lambasted by the editorial board of the Wall Street Journal, which described him as a "self-professed Bernie Sanders acolyte."

drug pricing

Judge delivers win to PBMs in closely-watched Arkansas case

A U.S. federal court judge has temporarily blocked a first-in-the-nation Arkansas law that would prohibit companies that own pharmacy benefit managers from also operating both retail and mail-order pharmacies.

Critics have argued that companies such as CVS Health, Cigna, and UnitedHealth Group, which own the largest PBMs, favor their own pharmacy operations and distort the pricing for prescription medicines.

After the Arkansas law was passed in April, it was quickly endorsed by dozens of state attorneys general, who also wrote to federal lawmakers in hopes that Congress would pursue a similar nationwide law.

neurology

Major Alzheimer's group backs certain blood tests for diagnosing

In its first clinical guidelines on blood biomarker testing, the Alzheimer's Association is recommending the use of certain blood tests for diagnosing patients, a move that could lead to more more patients getting diagnosed and potentially also treated

For patients confirmed to have cognitive impairment and are in specialized care, blood tests that have over 90% sensitivity (ability to identify positive results) and 90% specificity (ability to identify negative results) can be used instead of current diagnostic methods like PET scans and cerebrospinal fluid tests, the group said.

Tests that have over 90% sensitivity and 75% specificity can be used to triage patients, meaning negative results rule can rule out Alzheimer's with high probability but positive results should be confirmed with the standard diagnostic methods.

Licensing

Viridian inks deal for autoimmune candidates

From STAT's Allison DeAngelis: Viridian Therapeutics struck a deal around its two leading drug candidates for an autoimmune eye condition, earning $70 million upfront and up to $315 million in additional payments if it meets certain development and regulatory goals.

The Waltham, Mass.-based biotech is handing over the Japanese rights to these drugs to Kissei Pharmaceutical, it announced today. Kissei will also pay Viridian royalties on drug sales.

The drug candidates are both designed to test thyroid eye disease, a condition that can cause inflammation and damage to the tissues around the eye, leading to eye bulging, double vision and discomfort.

Viridian's drugs — veligrotug and VRDN-003 — are both in the late stages of development. Veligrotug hit the mark in Phase 3 trial results reported last year. Similar tests of VRDN-003 are underway. Analysts believe the drug could be a competitor to Amgen's Tepezza. That drug hit the market in 2020 and brought in $1.9 billion in sales last year.

health tech

AI-based medical devices could get streamlined Medicare coverage

There's a renewed push by lobbyists and policymakers to grant automatic Medicare coverage for FDA-designated "breakthrough" medical devices. If the proposal goes through, that means that the many AI-based devices that deemed by the FDA to be breakthroughs could get streamlined reimbursement.

Health agency leaders are supporting this idea. FDA Commissioner Marty Makary has said he is working with CMS Administrator Mehmet Oz to advance immediate Medicare reimbursement, so that breakthroughs "don't have to go through their own repeat duplicative process."

Of the nearly 700 devices that have been deemed "breakthroughs" by the FDA and are listed in STAT's Breakthrough Device Tracker, at least 10% use AI.

But some AI and health policy researchers are calling for caution as manufacturers.

Read more from STAT's Katie Palmer, and check out the new updates to the tracker.

More around STAT