GLP-1 drug shortage sparks legal action

Oct 11, 2024

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PROGRAMMING NOTE: We’ll be off this Monday for Indigenous Peoples Day but will be back in your inboxes on Wednesday.

Driving The Day

A flag flies above the headquarters campus of Eli Lilly and Company

Eli Lilly is intensifying efforts to stop the compounding of its weight-loss drugs. | Scott Olson/Getty Images

WHAT TO KNOW ABOUT GLP-1 SHORTAGES — It’s been a busy week in the diabetes and weight-loss drug world as Eli Lilly renews its push to shut down the compounding of its tirzepatide products and the pharmacists making the copies plead for flexibility.

Here are three things Lauren is keeping an eye on as this battle plays out in the coming weeks and months:

The legal landscape: The FDA is slated to respond today to the Outsourcing Facilities Association’s emergency motion for a temporary restraining order in connection with its challenge to the shortage list decision. The order would prevent the FDA from enforcing restrictions on compounding against larger facilities that have made copies of Lilly’s Mounjaro and Zepbound for telehealth platforms and smaller pharmacies for two weeks while the court decides whether to grant the group’s request for a preliminary injunction.

Judge Mark Pittman, a Trump-appointed judge in the Northern District of Texas, set a hearing on the matter for Tuesday, the same date OFA requested for a decision. It’s worth noting this district court falls under the 5th Circuit umbrella, meaning any appeal would go to a bench that’s been hostile of late to FDA decisions.

Regardless of how this effort shakes out, we’ll be interested to see how the prospect of litigation affects the FDA’s handling of semaglutide — the other GLP-1 that’s still in shortage — once Novo Nordisk says it can meet demand. One former FDA official noted that the agency could take a wait-and-see approach to that drug while the courts deal with the OFA lawsuit.

Enforcement discretion: Former agency officials and pharmacists agree the law is clear on when compounders must stop making copies of drugs that were previously in shortage: immediately in the case of smaller retail outlets overseen by states or within 60 days of the drug coming off the list for outsourcing facilities covered by the FDA’s strict manufacturing quality rules.

They also agree that the FDA has latitude to tread lightly on enforcement as the tirzepatide market settles. Supply continues to be spotty as brand-name product moves through distributors — a situation compounders say puts patients at risk of missing doses. Plus, price remains a concern given variable insurance coverage, and the agency has exercised compounding enforcement discretion at least once before for preterm birth drug Makena.

But depending on how the OFA case goes, the FDA’s ability to make policy decisions not explicitly outlined in statute might be hamstrung as judges form opinions in a post-Chevron world, where deference to agencies’ legal interpretations isn’t the standard.

Lilly’s next moves: The company has sent hundreds of cease-and-desist letters to companies selling compounded tirzepatide, and it’s already sued compounders and medical spas under state drug laws with mixed results. If the FDA goes easy on compounders, will Lilly turn its pressure campaign toward the agency?

IT’S FRIDAY. WELCOME BACK TO PRESCRIPTION PULSE. The fall weather and sunshine is all the medicine we need (for today, at least).

Send tips to David Lim (dlim@politico.com or @davidalim) and Lauren Gardner (lgardner@politico.com or @Gardner_LM).

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In 2023, four in five Express Scripts by Evernorth patients spent less than $250 a year out-of-pocket for all their medications. Yes, despite the median annual price of new drugs to market soaring to an average of $300K. That's not a middleman. That's an advocate. Express Scripts is advocating to make medications more affordable. Learn how here.

Eye on the FDA

A sign for the Food And Drug Administration is seen.

A drug for a rare genetic disorder narrowly won backing from FDA advisers despite some concerns about the treatment's effectiveness. | Sarah Silbiger/Getty Images

ADVISERS BACK RARE DRUG — The FDA’s advisory panel on heart and kidney drugs voted 10-6 Thursday to recommend the agency approve a novel treatment for an ultra-rare genetic condition despite persistent questions about its effectiveness.

The recommendation came after years of advocacy by patients with Barth syndrome and their caregivers and drugmaker Stealth BioTherapeutics for elamipretide. The discussion underscored the difficulty with generating and interpreting data around potential drugs for rare diseases, which don’t occur in large populations and can make recruitment for gold-standard placebo-controlled studies infeasible.

Barth syndrome — which can cause an enlarged and weakened heart, muscle weakness, recurrent infections and delayed growth — typically affects males and shortens their life expectancy by decades.

FDA reviewers indicated in briefing materials that they weren’t convinced that Stealth had conducted an “adequate and well-controlled” clinical trial with results supporting the drug’s effectiveness.

The independent advisers wrestled with how to approach the vote, with members asking the FDA to clarify whether officials wanted them to base their decisions on the data presented or their desire to give patients access to a treatment that still poses questions. FDA’s top drug regulator, Dr. Patrizia Cavazzoni, said she can accept a level of uncertainty around a drug sponsor’s data if the agency considers “regulatory flexibility” to make an approval decision.

Members who voted to recommend approval said anecdotal evidence from patients and “imperfect” data from Stealth’s studies influenced their decisions.

Those in opposition said they were uncomfortable supporting approval with the data available.

“I could not in good conscience say that I felt convinced that elamipretide was an effective drug,” despite regularly working with children with serious rare conditions, said Dr. Jean-Baptiste Le Pichon, associate division director for neurology at Children’s Mercy Kansas City.

What’s next: The FDA is scheduled to decide on the drug by Jan. 29.

BIRD FLU

FDA TALKS BIRD FLU — Shortly before California announced that the CDC confirmed the state’s fourth avian flu infection Thursday, the FDA’s outside vaccine experts met to discuss pandemic preparedness.

Jerry Weir, director of the FDA’s division of viral products in its Office of Vaccines Research and Review, said if a pandemic emerged, the agency would use every pathway to get vaccines not already licensed to market — including by emergency use authorization. But so-called strain change supplements could be used to rapidly update previously approved vaccines, he added.

“We’ve always thought at the agency that there was an advantage if one could have a vaccine that was licensed,” Weir said. “I think that’s important for the public, and I think we would certainly use that if possible.”

Industry Intel

FDA DELAYS DSCSA COMPLIANCE — Manufacturers, distributors and dispensers have more time to comply with enhanced drug-distribution security requirements required by the Drug Supply Chain Security Act after the FDA extended deadlines to avoid supply chain disruptions.

“While much progress has been made, including establishing electronic system data connections, this exemption is intended to support continued implementation of DSCSA without disrupting patient access to their medications,” the FDA posted on its website.

Manufacturers and repackers have until May 28, 2025; wholesale distributors have until Aug. 27, 2025; and dispensers with 26 or more full-time employees have until Nov. 27, 2025.

Chip Davis, CEO of the Healthcare Distribution Alliance, said the new timeline will allow supply chain players to “better align their data exchange processes to ultimately achieve full implementation.”

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Pharma Worldwide

SHOTS AGAINST AMR — Improving coverage of existing vaccines and ensuring broad uptake of certain immunizations in development could avert half a million deaths linked to antimicrobial resistance and reduce antibiotic use, according to a World Health Organization report.

The report focused on 24 pathogens and 44 vaccines — some already licensed by national regulators and others at different stages of clinical development, POLITICO’s Claudia Chiappa writes. AMR causes more than 1 million deaths each year and contributes to nearly 5 million, a toll that’s expected to grow in the next 25 years without global action.

Document Drawer

FDA’s medical device center published its proposed guidance agenda for fiscal 2025.

WHAT WE'RE READING

New Jersey Gov. Phil Murphy appointed two members to the state’s Drug Affordability Council, POLITICO’s Daniel Han reports.

An errant email to Pfizer CEO Albert Bourla tipped off the executive to an activist investor campaign, Endpoints News’ Drew Armstrong and Max Bayer report.

The Atlantic’s Nicholas Florko explores public health’s relationship with vapes.

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