New FDA pathway brings promise, and concerns

Bioethicists including the University of Pennsylvania’s Holly Fernandez Lynch support the FDA’s push to create a new approval pathway for personalized gene-editing treatments like the one given to Baby KJ. But, in an interview with STAT’s Jason Mast, she warned that the “plausible mechanism” framework laid out by FDA Commissioner Marty Makary and CBER chief Vinay Prasad is legally murky — and could be dangerously open-ended.

Writing in Health Affairs with other bioethicists, Fernandez Lynch says unveiling such a major policy shift via a journal article rather than formal FDA guidance sidesteps required process, shuts out broader public input, and leaves unclear which patients would even qualify. Without strict limits, she cautioned that sponsors and patient groups could push the mechanism well past ultra-rare cases — allowing what should be exceptional regulatory flexibilty routinely.

“We know that when the FDA opens the door to these things, there’s a lot of pressure … to then open the door a little wider and then a little wider and then a little wider until you have an exceptional program becoming the norm,” Fernandez Lynch said.

glp-1 drugs

Launch of Wegovy pill looks to be going strong so far

From STAT’s Elaine Chen: The oral version of Novo Nordisk’s obesity drug Wegovy saw about 18,400 prescriptions during its second week of launch, a rapid increase from the roughly 1,300 prescriptions seen in the first week, according to Leerink analyst David Risinger, citing data from IQVIA.

That may even be an undercount. Novo’s investor relations team told Risinger that the IQVIA numbers don’t include all prescriptions coming through the direct-to-consumer cash market. The caveat is there tends to be significant fluctuation in week-to-week prescription numbers. Still, analysts are seeing the new figure as an encouraging sign. For context, Risinger noted that Eli Lilly’s injectable Zepbound saw about 1,300 prescription in its first week of launch and about 8,000 in its second week.

The faster Novo can capture demand, the more it can get ahead of Lilly, which is waiting an FDA decision on its GLP-1 pill orforglipron. Lilly had initially said it expected an approval in March, but is now saying it will come as early as the second quarter.

Novo is focusing intensely on the DTC market with the Wegovy pill. It’s selling the highest doses at $299 a month, undercutting the $399 price that Lilly intends to sell orforglipron at.

startups

The race to finally diagnose endometriosis

After a decades-long odyssey to diagnose endometriosis, a crop of biotech startups is racing to develop noninvasive tests for the condition. Though common, the disease is poorly understood and can take five to 10 years to diagnose. Even then, there’s a lack of effective treatments.

At least a dozen companies are not hunting for biomarkers in blood, menstrual fluid, saliva, stool, or epigenetic signals, STAT’s Theresa Gaffney writes. Several are completing pivotal trials and planning U.S. launches this year — though none of the tests has yet proven reliable enough to replace laparoscopy, the surgical gold standard.

“That's a huge area where a lot of these newer companies are trying to take shortcuts and so you're not actually comparing against the standard of care,” said Heather Bowerman, CEO of endometriosis diagnostic startup DotLab. “Any diagnostic in this space that is not anchored to surgical confirmation is fundamentally speculative.”

synthetic biology

Genyro licenses DNA writing breakthrough from Caltech

Los Angeles-area startup Genyro, whose board includes Nobel laureate Frances Arnold and Moderna co-founder and MIT professor Bob Langer, has licensed a new DNA construction technology called Sidewinder from the California Institute of Technology.

The method, as described last week in Nature, aims to overcome long-standing limits in building large, complex DNA sequences. Developed by Caltech professor and Genyro co-founder Kaihang Wang, Sidewinder uses temporary “assembly instructions” embedded in DNA junctions to dramatically reduce errors, enabling parallel construction of long, repetitive sequences with far greater accuracy than conventional techniques.

“The future of medicine, materials, food production, and the global bioeconomy depends on our ability to write DNA,” Wang said in a statement. “Sidewinder delivers the freedom to construct long DNA sequences irrespective of their complexity — faster, more accurately, and more affordably than has ever previously been possible.”

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