Lilly’s donanemab gets a red light from the FDA

Presented by The Campaign for Sustainable Rx Pricing

With Erin Schumaker

Driving the Day

LILLY GETS FDA CRL FOR DONANEMAB — Eli Lilly and Co. disclosed late Thursday that the FDA rejected its application for accelerated approval of its Alzheimer’s disease drug candidate, but the company plans to press ahead for traditional approval once it obtains more clinical data.

Lilly received a Complete Response Letter from the FDA for donanemab, rejecting the company’s application for accelerated approval because of a lack of data on a sufficient number of patients for at least 12 months of continued treatment.

But the drug, a monoclonal antibody that reduces certain brain proteins thought to be associated with cognitive decline, may still be on track for traditional approval later this year. Donanemab is the third monoclonal antibody therapy for Alzheimer’s treatment to seek FDA approval in the past two years.

Lilly is conducting a Phase III trial of donanemab with roughly 1,800 participants and expects to have preliminary results by the second quarter of this year. The company anticipates the late-stage trial to “form the basis of donanemab’s application for traditional approval shortly thereafter.”

IT’S FRIDAY. WELCOME TO PRESCRIPTION PULSE. It’s time to announce something you may have picked up on via Twitter: Lauren is going on hiatus later this month as she picks up a new title — Mom!

Send tips, feedback and late-night TV recommendations to David Lim ([email protected] or @davidalim), Lauren Gardner ([email protected] or @Gardner_LM) or Katherine Ellen Foley ([email protected] or @katherineefoley).

TODAY ON OUR PULSE CHECK PODCAST, Ruth Reader talks with Ben Leonard about Congress’ notice to HHS that the agency needs to better oversee its funding of research into “enhanced potential pandemic pathogens” — and why this adds fuel to a contentious debate in Washington, D.C., over so-called gain-of-function research.

In Congress

PALLONE: AGENCIES MUST ENFORCE TRIAL REPORTING REQUIREMENTS — The FDA and NIH must do a better job policing companies that do not comply with requirements to report clinical trial information to ClinicalTrials.gov, said Rep. Frank Pallone (D-N.J.), the top Democrat on the House Energy and Commerce Committee.

Citing recent studies and an August HHS Office of Inspector General report that show many clinical trial sponsors have failed to report results on time, the lawmaker argued the FDA and NIH need to deploy rarely used enforcement tools, such as trial termination and civil money penalties, to compel compliance with the law.

“These actions have only been taken with regard to a tiny fraction of the trials that have been or remain in violation of ClinicalTrials.gov requirements. Additionally, it appears FDA has not sent any compliance letters to NIH, despite the many trials NIH runs or oversees,” Pallone wrote in a Thursday letter to FDA Commissioner Robert Califf and acting NIH Director Lawrence Tabak. “FDA has also not yet imposed any civil money penalties on any trial sponsors. The collection of these penalties would provide a stronger incentive for trial sponsors to comply.”

Trials on the mind: Earlier this week, Califf published a research article making the case for an overhaul of postmarket research on medical products to improve patient health outcomes.

To learn real-world best practices for medical interventions, “continued improvements in data science and management, the ethics of data sharing, and alignment of purpose in integrating research and clinical practice are the central areas where focus is needed,” he wrote.

SANDERS BRINGS HELP ONTO HELP — Incoming Senate HELP Committee Chair Bernie Sanders (I-Vt.) is building up his health policy staff with several longtime aides and alums, POLITICO’s Megan R. Wilson scoops.

For pharma watchers: Sanders is bringing on Zain Rizvi from progressive advocacy group Public Citizen, where he’s focused on access to affordable drugs, to work on pharmaceutical policy as the committee’s senior health counsel.

Eye on the FDA

STATE AGS PROTEST FDA ABORTION PILL POLICY — Nearly two dozen state attorneys general are asking the FDA to reverse its decision removing the in-person dispensing requirement for medication abortion. In a letter made public Thursday, the 22 attorneys general stressed that the agency’s updated regs don’t supersede their states’ laws prohibiting telehealth abortions.

“Nothing in the FDA’s recent changes affects how we will protect our people,” they wrote to Califf.

FDA: DRUG DEMAND REPORTING REQS MAY HELP MITIGATE IMPACTS — While high-profile shortages of brand-name drugs, such as Adderall and the diabetes drug Ozempic, continue to plague Americans who rely on them, demand-driven shortages are the exception rather than the rule, according to the FDA.

“Demand shortages have been kind of a new challenge for us since the pandemic,” Valerie Jensen, associate director of the drug shortage staff at the FDA, told Erin. “We’ve had some unusual circumstances.”

A “tripledemic” of respiratory ailments, including Covid-19, caused a run on children’s cold and flu medicine. Meanwhile, TikTok influencers touted the off-label use of Ozempic for weight loss, which caused the drug to become so popular that people with diabetes had a hard time getting it.

And a surge in Adderall prescriptions, combined with strict DEA regulations for Schedule II controlled substances and manufacturing delays, have made the ADHD drug all but impossible to get. While the manufacturing delays have been resolved — according to Jensen — it remains unclear when the shortage will end.

Supply-side disruptions are a more pervasive problem. In addition to drug discontinuations and delays, supply interruptions at manufacturing facilities are a major problem, according to the FDA. There are 192 drugs actively in shortage or being resolved, per the FDA.

While the FDA works closely with manufacturers to identify disruptions and prevent shortages before they occur, more transparency from drug manufacturers could mitigate demand-driven shortages.

Right now, drug companies are required to notify the FDA about supply or manufacturing disruptions. Disclosing demand spikes remains voluntary.

“We do think that would be helpful,” Jensen said of advanced warnings about demand spikes. “What that would do is help us to use our tools earlier and hopefully prevent shortages before they occur,” she said.

Drug Pricing

JANUARY DRUG PRICE HIKES DRAW CSRxP IRE — A basket of 587 brand-name drugs saw an average price increase of 5.5 percent for 2023, according to data from AnalySource. The data drew fresh calls from the Campaign for Sustainable Rx Pricing — whose members include hospitals, insurers and pharmacy benefit managers — for policymakers to take further action to curb the cost of prescription drugs.

But PhRMA spokesperson Brian Newell argued the complaint is off-base. In December, the consumer price index showed prices rose 6.5 percent over the past 12 months.

“Premiums are going up, along with deductibles and other out-of-pocket costs that insurers force patients to pay, and a front group for the insurance industry is pushing out a report that shows drug prices are growing below the rate of inflation?” Newell said. “Net drug prices after rebates and discounts are falling.”

Industry Intel

J&J ENDS LATE-STAGE HIV VACCINE TRIAL AFTER CANDIDATE NOT EFFECTIVE — An HIV vaccine candidate from Janssen, a subsidiary of Johnson & Johnson, was found to be safe but ineffective at preventing infection, the company said Wednesday.

As a result, the company halted the Phase III trial, which had been ongoing since 2019. The trial included 3,900 men and transgender people who received four doses of the vaccine candidate, which was designed to provide protection against multiple virus subtypes. They also received HIV prevention tools, including PrEP, or preexposure prophylaxis, and immediate treatment if they contracted the virus.

Pharma Moves

Ben Pearlman joined the National Association of Chain Drug Stores as director of state government affairs. Most recently, he served as senior director of government and public affairs for Shipt, which is owned by Target.

Meetali Desai, Rosland Fisher McLeod and Tim Lynch have joined hospital advocacy group 340B Health’s board of directors.

Quick Hits

An analysis from Jennifer Golan at ProPublica found that websites that sell abortion pills share data with Google and other third parties, which could inform law enforcement of when people terminate their pregnancies.

Walgreens has agreed to pay West Virginia $83 million for its role in the opioid crisis, bringing the state’s total opioid settlement money to more than $950 million, Leah Willingham reports for The Associated Press.

Document Drawer

The FDA published draft guidance for developing mpox treatments Friday.

The Office of Information and Regulatory Affairs is reviewing final guidance on compounding certain ibuprofen oral suspension products.

On Thursday, the FDA announced it extended the shelf life for at-home Covid-19 tests made by Abbott, iHealth and MaximBio.

The FDA is requesting comments on whether it should give Medical Device User Fee Amendments funding intended to boost premarket real-world evidence activities to an organization other than the National Evaluation System for Health Technology.

Nonprofit ECRI named inadequate communication of recalls for at-home medical devices as its top health technology safety issue for 2023.