FDA panel recommends approval of donanemab

An FDA advisory panel recommends approval of Eli Lilly’s Alzheimer's drug donanemab, parents explain why we need to reauthorize the Rare Pediatric Disease Priority Review Voucher Program (PPRV), and we recap BIO 2024 sessions on women’s health. (611 words, 3 minutes, 3 seconds)

FDA panel recommends approval of donanemab for early-stage Alzheimer’s

A Food and Drug Administration (FDA) advisory panel voted 11-0 to recommend approval of Ely Lilly’s Alzheimer's drug donanemab yesterday.

What they said: Donanemab is safe and effective in slowing cognitive decline among people in the early stages of Alzheimer’s, according to the Peripheral and Central Nervous System Drugs Advisory Committee, which was tasked by the FDA to review Eli Lilly’s 2023 biologics license application.

The drug: Donanemab reduces amyloid plaque, a protein found in high levels in the brains of Alzheimer’s patients.

The clinical trial: Donanemab showed a 37% lower risk of progression of a clinical measure of dementia over 76 weeks, according to data presented by Eli Lilly.

What they’re saying: “It’s encouraging to see that some patients essentially enter remission, where they achieve full amyloid clearance with donanemab, with no resurgence in substantial plaque buildup for nearly four years,” said Dr. Howard Fillit of the Alzheimer’s Drug Discovery Foundation.

What’s next: The FDA is expected to decide on donanemab’s application for approval by the end of the year. The FDA usually follows the recommendations of its advisory panels.

‘Curing rare childhood diseases will falter unless Congress steps up’

Describing their family’s struggle with an ultra-rare form of muscular dystrophy, two parents call on Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher Program (PPRV).

Their journey: Joe and Courtney Dion’s 10-year-old son and 6-year-old daughter have a condition that can lead to inability to walk. Like 95% of the many rare diseases impacting 30 million Americans, the condition has no treatment, they write in STAT News.

“Thankfully, we haven’t gone it alone: Over the years, Congress has passed a number of important reforms aimed at encouraging more research into rare diseases. One of the great victories of these efforts is the rare pediatric disease priority review voucher program,” the Dions write.

How it works: Companies gaining approval for drugs targeting rare pediatric diseases can receive a voucher that grants expedited review of another drug in the future. Companies can fund current research by selling the voucher to another company.

Why it matters: “Priority review vouchers have attracted an impressive amount of attention and private investment to this underserved area of medical research. To date, the program has led to the development of nearly four dozen pediatric rare disease medicines,” the Dions explain.

The PPRV program “is set to expire in September. Without swift action by Congress, the difficult task of curing and treating rare childhood diseases will get even harder,” the Dions write.

Read the whole thing at STAT News.

BIO 2024 puts spotlight on women’s health

ICYMI: Women’s health receives less than 1% of all VC investment in the U.S.—but by addressing the women’s health gap, we could not only improve women’s health but also boost the global economy by $1 trillion annually by 2040, says a recent McKinsey report.

Why it matters: “I honestly don’t see dramatic differences in the understanding of women’s healthcare conditions now versus a decade ago,” noted Julia Owens, Ph.D., CEO of Verto Therapeutics, at the 2024 BIO International Convention, speaking during a panel that explored the lack of investment in women’s health and what’s needed to improve it.

Speaking of women’s health…we CAN eliminate cervical cancer in our lifetimes, said another panel featuring BIO members Roche and Merck, which developed the Gardasil vaccine. But we still have a lot of women who need to be reached and told of the benefits of screening and vaccination.

Catch up on BIO 2024 with Good Day BIO Live 

• Keep an eye on Bio.News for panel recaps, exclusive Q&As, and much more.
• Join the conversation on X and LinkedIn, with hashtag #BIO2024. Watch for more exclusive video content and repost-worthy quotes.

President Biden’s Tuesday: Addressing the Everytown for Gun Safety Action Fund’s “Gun Sense University,” in Washington D.C., per CNN.

What’s Happening on Capitol Hill: The House Appropriations Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies will mark up an FY25 spending bill.