FDA approves Duchenne gene therapy for some

The FDA yesterday conditionally approved Sarepta's gene therapy for Duchenne muscular dystrophy. Regulators said the treatment, called Elevydis, could be given exclusively to younger patients until additional data emerges that could broaden its use.

The one-time treatment is the second-costliest ever in the U.S. — listing at $3.2 million. Although other treatments are available to treat the degenerative condition, their effects are modest — and the hope is that Sarepta's gene therapy might more dramatically alter the course of the disease.

This is the first time the FDA has accepted a sponsor's request to win an accelerated approval for a gene therapy. Sarepta had, however, asked regulators to approve the drugs in all patients who can still walk — so the limited label will prove disappointing for many families whose children do not meet the criteria to receive the medicine. Still, the approval offers hope to some with Duchenne who aren't yet eligible to receive the gene therapy.

"When I was growing up, something like SRP-9001 was for the future. For many boys, it is now the present," one 26-year-old with the disease writes for STAT. "This approval opens up the floodgates for continued research into Duchenne. If one company succeeded through this accelerated approval pathway, then many more can as well."

alzheimer's

Medicare details how it'll cover Leqembi and its kin

It's quite likely Leqembi, Biogen and Eisai's Alzheimer's drug, will soon win approval — which means that soon Medicare could be swamped with requests for the plaque-clearing antibody drug. The agency just released details on how it plans to cover new Alzheimer's drugs.

For example, it will build a patient registry, through which physicians will have to submit data about the drug to Medicare. Details will include the patient's diagnosis, results of cognition tests, whether they're taking blood thinners, any data that indicate amyloid plaque buildup in the brain, and any adverse events like bleeding or swelling.

Ultimately, tools like genotyping will likely help guide which patients receive this class of Alzheimer's drugs. It still remains to be seen if Medicare and other insurers will cover this testing.

podcast

Who's watching the drug price watchmen?

Are RSV vaccines too expensive? And is it finally time for Novavax to shine? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Our colleague Bob Herman joins us to explain his year-long investigation into some brazen conflicts of interest in the world of prescription drug pricing. Then, STAT's Helen Branswell calls in to walk us through a news-packed week for vaccines, including weighty decisions for Covid-19 RSV.

Listen here.

EUROPE

Moderna and U.K. party at a power station

There was no subtlety in the backdrop of Moderna's launch party yesterday: The company celebrated a 10-year partnership with U.K. government in the gold-and-glass control room of the decommissioned Battersea Power Station. "Where better than a power station to celebrate the power of science?" said Will Quince, the country's minister for health and secondary care.

Moderna will invest in mRNA research programs in the U.K., and is building the Moderna Innovation and Technology Center at Harwell, a science campus in Oxfordshire. Per the agreement, a major mRNA vaccine manufacturing site will now be in the U.K. The deal is an economic blessing for the U.K., which has been fighting the perception that it's falling behind in the life sciences. And the event allowed Moderna to showcase its plans beyond Covid-19.