Vinay Prasad

Stories

Disc Medicine's rejected sun-sensitivity drug clears Phase 3, reopening FDA path

Left the FDA; no longer involved in the bitopertin review

People with erythropoietic protoporphyria feel stabbing pain in strong light, and no oral drug is approved to help them. Disc Medicine's bitopertin passed a 183-patient Phase 3 trial called APOLLO, the company said on Oct. 19, 2026, reopening a path to the FDA after a rejection in February.

Updated Oct 25

Moderna's mRNA flu vaccine clears Phase 3 with superior efficacy

New Capabilities

As of May 2026: Reversed the initial refusal to review mRNA-1010

For 80 years, flu vaccines grew in chicken eggs — a process effective against roughly 40% of infections in good years. On May 8, the New England Journal of Medicine published Moderna's Phase 3 data: mRNA-1010 reduced influenza illness by 26.6% more than a standard shot in 40,805 adults aged 50 and older. Moderna's stock rose 16% that day.

Updated May 31

FDA creates new approval pathway for gene therapies tailored to individual rare disease patients

Rule Changes

As of May 2026: Returned to CBER director role after brief departure in mid-2025

For decades, the FDA required all drugs to prove effectiveness through controlled trials with sufficient sample sizes, a standard that made sense for common diseases but created an impossible barrier for ultra-rare conditions. On February 23, 2026, the FDA issued draft guidance for a new standard—the 'plausible mechanism' framework. Developers of gene-editing and ribonucleic acid (RNA) therapies could win full approval by showing that their treatment targets the root genetic cause, engages it, and improves outcomes compared to the disease's natural course.

Updated May 29

FDA shifts to single-study standard for drug approvals

Rule Changes

As of May 2026: Active, serving in dual roles

For 63 years, the FDA required drugmakers to prove their products worked in at least two rigorous clinical trials. On February 18, 2026, Commissioner Marty Makary formally ended that standard. Now one trial is the 'default position' for all new drugs—from rare diseases to common conditions affecting millions of patients. In accompanying articles published in the New England Journal of Medicine and JAMA, Makary and Deputy Commissioner Vinay Prasad emphasized that the single-trial standard doesn't eliminate evidence requirements. Instead, sponsors must provide 'confirmative evidence' through mechanistic data, findings from related indications, animal models, real-world evidence, or data from drugs in the same class.

Updated May 29

The six-month cure: gene editing goes bespoke

New Capabilities

As of May 2026: Co-developing guidance on personalized therapy approvals

In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy designed, manufactured, approved, and delivered in 180 days.

Updated May 19

Notable Quotes

"Designing treatments unique to individual patients has always been the promised goal of personalized medicine. After 25 years, the FDA has, for the first time, outlined a framework to facilitate these approvals. The Plausible Mechanism framework is a revolutionary advance in regulatory science." — FDA press announcement, February 2026

from FDA creates new approval pathway for gene therapies tailored to individual rare disease patients · Updated May 29

"Dropping the two-trial requirement reflects modern advances that have made drug research increasingly precise and scientific." — NEJM, February 2026

from FDA shifts to single-study standard for drug approvals · Updated May 29