Contamination at Novo manufacturing facility trips up a biotech's drug approval — again

Scholar Rock said this morning that the FDA rejected its treatment for the muscle-wasting disease spinal muscular atrophy due to long-unresolved problems at a contract manufacturing facility where the drug is packaged.

The biotech said its drug will be resubmitted to the FDA for approval once the quality-control issues at the manufacturing facility, owned by the Danish pharma giant Novo Nordisk, are cleared.

Apitegromab is the second drug in recent months to see its marketing clearance indefinitely delayed due to problems at the Novo facility. The FDA has also rejected an application from Regeneron for a high-dose version of its Eylea eye drug for the same reasons.

Read more from my colleague Adam Feuerstein.

politics

FDA brings back shelved GSK drug in highly unusual move

The Food and Drug Administration yesterday re-approved a drug from GSK that the company stopped selling more than 25 years ago, as part of a push from the Trump administration to identify the causes of and potential treatments for autism.

The drug, Wellcovorin, is a branded version of leucovorin, a widely available generic drug that's generally used to help tame the side effects of drugs, including certain cancer treatments. The FDA said that its systematic analysis of literature, including case reports, support the idea that the drug can help people with cerebral folate deficiency, a neurological condition that can have overlapping symptoms with autism.

It appeared to be an unheard-of move by the FDA: re-approving an old branded drug, and doing so in the absence of a request from the drugmaker in question. GSK said, though, that it plans to work with the FDA to update the drug's label.

Read more from STAT's Drew Joseph and Daniel Payne.

biotech

(ARCH-backed) Rome has fallen

From my colleague Jason Mast: ARCH Venture-backed startup Rome Therapeutics is laying off staff and "exploring strategic options," CEO Rosana Kapeller said in an email to STAT.

"Several employees will continue with ROME until we determine the best strategic alternative for the company and our programs," she added.

Rome is the latest startup to fall apparent victim to a protracted biotech downturn. The company launched in 2020 with funding from GV and ARCH to explore how endogenous retroviruses — viruses that have permanently enmeshed themselves in our DNA over eons of evolution — and other DNA elements similar to viruses can drive autoimmune disease and cancer.

The company unveiled preclinical data in Parkinson's and ALS and advanced a program for lupus into "early clinical development," according to its website, but had not announced clinical data. It last raised a $72 million series B extension in 2023.

pharma

Drugmakers try to appease Trump before MFN deadline

Pharma companies seem to be trying to appease President Trump ahead of a Sept. 29 deadline to meet the president's demands related to his most-favored nation policy.

Bristol Myers Squibb said yesterday that it plans to launch its new schizophrenia drug Cobenfy at a list price in the U.K. equal to the U.S. list price (which is $1,850 per month). This seems to fall in line with Trump's goal of making other countries pay more for medicines. "We are asking the UK to step up in recognizing the value of truly innovative therapies," Chief Commercial Officer Adam Lenkowsky said in a statement.

But it's not clear what this announcement will mean for how much the U.K. actually pays. The National Health Service could try to negotiate deep discounts, though the company is "prepared to make the difficult decision to walk away if [NHS] cannot better recognize the value our medicine brings to patients and society," Lenkowsky said. Negotiations are also still a long time away — BMS has not yet submitted the drug for approval in the U.K.

Novartis CEO Vas Narasimhan similarly said in an interview that drug prices in its home country Switzerland are too low. He also said the company is looking at ways to propose price cuts to Trump before the end of the month.

health tech

AI device makers aren't disclosing their payments to doctors, study says

Companies that make drugs and devices that go through FDA approval are required to disclose payments they make to health providers. But manufacturers of AI devices, most of which do not go through the FDA, seem to fall into a regulatory gray area.

In a new study, researchers found that when analyzing Open Payments (the federal database of health industry payments to providers), fewer than 10% of nearly 850 AI devices were linked to payments between 2017 and 2023. Some AI companies likely didn't appear in the database because they simply didn't make any payments, but the there's likely also a large majority of companies have made payments, but did not have to report them, one of the authors said.

The researchers said that policies should change so that AI companies are more transparent about their payments, particularly as the technology is increasingly adopted in medicine.