Product Reviews

The headlines showed a robust tally of new drug approvals from the US FDA in 2025, but cracks are beginning to show that could turn into fissures in the year ahead.

The European Medicines Agency has recommended new uses for nine approved medicines, including Incyte's immunotherapy Zynyz which is poised to become the first EU treatment for squamous cell carcinoma of the anal canal.

Accelerated approvals of novel products rebounded in 2025 as drug development adapted to major pathway changes enacted in the FDA Omnibus Reform Act, but pending applications suggest a potentially bigger impact outside oncology in 2026.

The European Medicines Agency said that Sanofi’s Rezurock should be approved on a conditional basis for patients with chronic graft-versus host disease after reconsidering its initial decision not to recommend the drug.

The unusual revision of Corcept’s complete response letter suggests the FDA may be writing for a different audience now that unapproved product CRLs are being made public.

The FDA starts 2026 with 55 novel agents under review, which are detailed in Pink Sheet's interactive chart.

Review times for novel agents approved in 2025 stayed remarkably steady, hugging PDUFA timelines, maintaining high approval volumes, and foreshadowing a new debate over whether ultra‑fast, politically driven reviews will result from the Commissioner's National Priority Voucher program.

Pink Sheet editors discuss Richard Pazdur’s concerns about the Commissioner’s National Priority Voucher program and the idea that the FDA could release action packages for unapproved products in addition to complete response letters.

Saol Therapeutics cannot conduct a new clinical trial of SL1009 in ultra-rare mitochondrial disorder, but aims to answer the FDA's complete response letter with new looks at available data.

FDA reviewers cited several limitations of the videos, including a lengthy recall period, use of the term “today” in questions, and a lack of standardization in interview conduct.

The Pink Sheet’s Drug Review Profile looks at the clinical development and US FDA review timeline for Stealth's elamipretide for Barth syndrome.

Stealth BioTherapeutics' diligent response to the FDA’s accelerated approval suggestion, its justification for proposed confirmatory trial milestones, and the rarity of Barth syndrome gave the agency confidence to grant approval before the postmarketing study began.

The European Medicines Agency has recommended new uses for nine approved medicines, including Incyte's immunotherapy Zynyz which is poised to become the first EU treatment for squamous cell carcinoma of the anal canal.

The EMA has voted in favor of EU approval for five new products, including treatments for a rare, life‑threatening genetic disorder and for non‑cirrhotic MASH, and has also recommended reversing its earlier rejection of an orphan drug for chronic graft‑versus‑host disease.

The European Medicines Agency said that Sanofi’s Rezurock should be approved on a conditional basis for patients with chronic graft-versus host disease after reconsidering its initial decision not to recommend the drug.

The European Medicines Agency is set to adopt opinions on whether marketing approval should be granted to five new products, including therapies for a serious liver disease and an ultra‑rare genetic mitochondrial disorder.

Amgen's refusal to voluntarily withdraw the rare disease treatment could force the FDA to employ its formal process to remove it from the market.

Two flu vaccine labels already include febrile seizure in the clinical trial and postmarketing experience sections of the label, but the FDA wants all of them to add a warning of the adverse event.

The US FDA’s comprehensive review found no increased risk of suicidal ideation and behavior with the use of GLP-1s to treat obesity, leading the agency to request existing warnings based on older drugs be removed from labeling.

The FDA outlined its plans for “Sentinel 3.0,” including a data hub that would speed queries, during PDUFA VIII negotiations as industry questions whether user fee revenue should continue funding the program.

New terms of reference for the Advisory Committee on Immunization Practices’ overhauled work groups on influenza, HPV and RSV reflect some members' broader concerns about the potential long-term adverse effects of vaccination.

A workshop co-hosted by the US FDA and Duke on ideas to expand access to epinephrine for anaphylaxis is the latest example of the agency holding a public meeting that could have been an advisory committee – but without the formalities.

After conducting more than 30 meetings for drugs, biologics and related issues in 2023 and 2024, less than half as many were held in 2025.

The tilt toward approval extended beyond patients and advocates to clinicians, professional societies and industry.