Why the FDA came down hard on Novartis

The FDA departed from protocol this month when it publicly blasted Novartis over submitting doctored data. The reason, according to a high-ranking agency official, is that the stakes were too high to let the issue pass quietly.

Dr. Peter Marks, who wrote the memo castigating Novartis, said the company’s data issue threatened to undermine the entire field of gene therapy. It so happened that the manipulated data didn’t imperil the safety of Zolgensma, Novartis’s one-time treatment for spinal muscular atrophy, but that doesn’t change the imperative that companies ensure data submitted to the FDA are accurate, Marks said.

“It may sound like we’re kind of bureaucratic paper pushers, but it’s more than that,” Marks told STAT’s Matthew Herper. “It’s making sure that the whole ecosystem understands that when people are working on these things that are highly technically complex, that they have to work truthfully and accurately. Because that’s the foundation upon which the trust that patients put in these products is built.”

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Is Sarepta’s latest failure tied to its earlier success?

We still don’t know the full list of reasons the FDA rejected Sarepta Therapeutics’ latest treatment for Duchenne muscular dystrophy, but history may offer some clues.

As STAT’s Adam Feuerstein points out, Sarepta’s last tangle with the FDA involved scant evidence, agency unrest, and a unilateral decision based at least in part on the company’s financial solvency. The result was a controversial approval for Exondys 51, a drug that now brings in about $300 million a year for Sarepta.

Cut to this week, and the FDA’s consideration of the company’s follow-on drug could well have been motivated by its prior experience. Sarepta is now worth more than $8 billion, and it by all means had the financial wherewithal to run a longer clinical trial before asking for the FDA’s blessing. And thus that rejection could be regulatory payback.

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All you need to know about the peanut allergy race

Immunotherapy isn’t just for cancer. A pair of companies are vying to hit the market with treatments that tinker with the body’s natural defenses to keep kids with peanut allergies safe from dangerous accidental exposure.

But the supporting data leave quite a few questions unanswered.

That’s why we recruited Dr. Erwin Gelfand, a pediatric allergist at National Jewish Health in Denver, to be our guest for the next session of STAT Expert Advantage. Next month, we’ll talk to Gelfand about in-development treatments from Aimmune Therapeutics and DBV Technologies, the merits of their data, and what prescribing physicians want to see from a peanut allergy immunotherapy.

The live call is Aug. 28, and you can sign up right here.

The SEC is serious about Wall Street whispering

If you’re a public company, you can’t play favorites with big news. And according to the SEC, TherapeuticsMD did exactly that on the subject of an all-important meeting with the FDA.

Back in 2017, the company met with the agency to discuss the future of its once-rejected estrogen therapy for postmenopausal women. A day later, one company executive sent an email to Wall Street analysts claiming the sit-down was “very positive and productive,” according to the SEC.

The problem is that TherapeuticsMD didn’t put out a press release or host a conference call to tell the public this apparently positive news, whispers of which sent the stock up nearly 20%. And now the company is paying $200,000 to settle SEC charges that it broke the rules.

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Why the FDA came down hard on Novartis

Is Sarepta’s latest failure tied to its earlier success?

More investors, more funding, and more partnering

All you need to know about the peanut allergy race

The SEC is serious about Wall Street whispering

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