Product Reviews

Pink Sheet editors and special guest Michael McCaughan of Prevision Policy discuss Robert F. Kennedy Jr.’s first act as HHS Secretary, former FDA Commissioner Scott Gottlieb’s suggestion that industry extend the existing user fee agreement rather than negotiate a new one, and President Trump’s policies impacting FDA advisory committee schedules and staffing.

HHS communications restrictions are keeping the FDA from publicly scheduling expected advisory committee meetings. The lack of upcoming meetings is raising concern about missed user fee dates or lower-quality agency decision-making.

A Trump Administration communications freeze and travel ban may be complicating efforts to convene FDA advisory committees. A 5 February meeting on postmarketing requirements for extended-release/long-acting opioids was postponed, while a 24 February meeting on a Novartis rare disease drug was cancelled.

The injection for radicular leg pain appears likely to be approved, but with a very prescriptive label to help balance safety concerns and a lack of long-term outcomes data.

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

Mavorixafor, X4 Pharmaceuticals/Norgine's treatment for WHIM syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

GSK’s depemokimab, if approved, could become the first ultra-long-acting drug for treating asthma and chronic rhinosinusitis with nasal polyps. Meanwhile, Sanofi’s teplizumab, which the US approved as the first disease-modifying therapy for type 1 diabetes in 2022, has now been filed for review by the European Medicines Agency.

Insight obtained by the Pink Sheet relating to the European Medicines Agency’s stricter approach to requests for clock-stop extension shows that refusals have not led to any companies withdrawing their marketing applications. On the contrary, companies were able to provide responses within the agreed timeframe after all.

The US FDA approval of new meningococcal and chikungunya vaccines arrived in time for the 26-28 February meeting of the CDC’s Advisory Committee On Immunization Practices, if political developments allow.

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include nine new products, one of which is Kostaive (zapomeran), CSL/Arcturus Therapeutics’ self-amplifying mRNA COVID-19 vaccine for individuals 18 years and older.

Mavorixafor, X4 Pharmaceuticals/Norgine's treatment for WHIM syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

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.

Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.

An FDA complete response letter followed a bioresearch warning letter citing the integrity of electronic data capture and handling of dosing errors in a pivotal trial of govorestat for galactosemia.

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.

The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.

Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.

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.

The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.