Product Reviews

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.

The disruption of CDC’s Advisory Committee on Immunization Practices meeting schedule adds fuel to concerns about potential alterations of the vaccine schedule raised by former FDA Commissioner Scott Gottlieb.

Martin Makary said he would re-evaluate the topics that deserve a Vaccines and Related Biological Products Advisory Committee review, but did not commit to rescheduling the cancelled meeting on influenza vaccine strain selection.

The Vaccines and Related Biological Products Advisory Committee meeting next month was scheduled to select strains for the 2025-2026 flu vaccine. The cancellation could mean delays in vaccine availability.

The European Medicines Agency has recommended five drugs for EU-wide approval , including Averoa’s Xoanacyl for concomitant hyperphosphatemia. Two companies have withdrawn their marketing authorization applications.

Eli Lilly’ will request a re-examination after the European Medicines Agency declined to recommend its Alzheimer’s disease drug Kisunla for EU approval.

The European Medicines Agency’s qualification of the AIM-NASH tool is said to signify a major advancement for clinical trials for metabolic dysfunction-associated steatohepatitis. The market size for MASH treatments is expected to grow substantially in the coming years.

Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.

Levodopa/carbidopa (ND0612), Mitsubishi Tanabe Pharma’s investigational drug-device combination therapy for the treatment of motor fluctuations in people with Parkinson’s disease, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Japan recommends eight new drugs for approval, including two for cardiomyopathy, and will also raise prices for multiple products, including Enhertu, on 1 April.

Unofficial January results showed the US FDA continued to issue full and tentative ANDA approvals in line with previous months, but recent layoffs may not help the agency maintain that pace.

While CMC glitches linger over a US NDA for Elevar/Hengrui’s novel liver cancer combination following a second complete response letter, the separate issue of underrepresentation of US patients in multiregional trials is looming large after new FDA draft guidance last year.

Acting Commissioner Sara Brenner rejected the company’s request for a hearing, saying the lack of an adequate and well-controlled trial showing tasimelteon's effectiveness in treating sleep-onset insomnia is “fatal to the application.”

Prospects are promising for many of the 16 novel candidates that received complete response letters in 2024 after half of the 2023 CRL class was approved the following year.

Vanda’s fight against a complete response letter for its gastroparesis drug results in a notice of opportunity for hearing on a formal FDA proposal to refuse to approve the NDA.

The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.

The Pink Sheet Drug Review Profile breaks down the FDA's long reckoning with testosterone cardiovascular safety concerns

Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.

The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

Regulatory authorities from multiple regions are considering extending a pilot project related to collaborative assessments of post-approval chemistry, manufacturing and controls changes, with a focus on the supply of critical medicines.

The pharmaceutical industry was not sufficiently involved in designing updates to England’s health data infrastructure, leading to a “misalignment” between company needs and what is currently being delivered, according to the results of a survey by the Association of the British Pharmaceutical Industry.

Patient advocates and sponsors want the US to reconsider a 20-year-old class warning against the use of antipsychotics in patients with dementia. The agency is weighing the issue, but also defending the current labels of newer agents.

Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.