FDA staff wanted to reject Sarepta's gene therapy

Reviewers at the FDA were leaning toward rejecting Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy until a high-ranking agency official intervened, according to three people familiar with the matter.

As STAT's Adam Feuerstein and Jason Mast report, staff members at the FDA's gene therapy division had reached a non-binding conclusion that Sarepta's application did not meet the criteria for a speedy approval, according to the people. Upon hearing that, Peter Marks, a top FDA official and advocate for faster approvals, stepped in and directed staff to schedule a public hearing on gene therapy, the people said.

That, they said, was Marks' second intervention in the review of Sarepta's gene therapy.

Research

How Michael J. Fox 'got one punch in on Parkinson's' with new discovery

A new study, funded by the Michael J. Fox Foundation for Parkinson's Research, has produced the clearest evidence yet that the presence of a particular misfolded protein can determine whether people have the disease, a discovery that could lead to new diagnostics and potential medicines.

As STAT's Matthew Herper reports, the findings establish that testing for alpha synuclein detected Parkinson's 87% of the time. And in volunteers who did not have Parkinson's, the same test showed the absence of the disease 96% of the time. The results, published in The Lancet Neurology last night, are the result of a 1,123-person study that has cost the Fox Foundation hundreds of millions of dollars since it began in 2010.

To Fox, 61, the discovery is a testament to the labors of the foundation he founded two decades ago. And after more than 30 years of battling the disease, "I think I got one punch in on Parkinson's," he said.

Bernie's next guests are the insulin makers

About a month after grilling Moderna CEO Stéphane Bancel on the price of the company's Covid-19 vaccine, Sen. Bernie Sanders plans to call the leaders of the world's largest insulin manufacturers to testify before his health committee.

As STAT's Rachel Cohrs and John Wilkerson report, the CEOs will be summoned to a hearing some time next month, according to people familiar with the matter. No dates have been announced, but Lilly and Novo Nordisk said they were aware of the plan and expect to participate. Sanofi didn't respond to a request for comment.

The hearing comes after all three companies pledged to substantially lower the list prices of their older insulin products, following years of public outcry over lock-step increases.

In the lab

Biogen's next Alzheimer's drug might cut out the bodily middleman

Leqembi, Eisai and Biogen's approved antibody treatment for Alzheimer's disease, requires lengthy infusions twice a month to generate the modest benefits established in clinical trials. That's because you have to pump a lot of antibodies into the veins to get enough of them into the brain, making treatment a relatively imprecise science.

Biogen wants to change that with its next potential Alzheimer's treatment. Yesterday, the company licensed a treatment from Denali Therapeutics that uses that company's technology designed to get large molecules across the blood-brain barrier, a cellular matrix that keeps unwanted materials from reaching the brain.

The idea is to use Denali's approach to ferry antibodies not unlike Leqembi straight into the brain, where they would bind to toxic plaques and slow the progression of Alzheimer's. And if it works, the company could use substantially lower doses and less frequent administrations to do the job.

More around STAT