Product Reviews
US Advisory Committees
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.
An FDA advisory committee will consider modifications to the indicated patient population, as well as the appropriate health professionals and settings to administer the proposed treatment for radicular leg pain.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
The development of a joint document is not iterative and there is no discussion between the agency and sponsor, according to new guidance seemingly aimed at differentiating the frequent use of joint backgrounders in oncology from the single document developed for the Alzheimer's drug Aduhelm.
EU CHMP
Marketing authorization applications for five new advanced therapy medicinal products were filed with the European Medicines Agency in 2024, the highest number in any year since 2020. The Pink Sheet looks at these and two other ATMPs that might get the thumbs up from the European Medicines Agency in 2025.
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.
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
The European Medicines Agency has recommended several new products for pan-EU approval, including CSL Behring’s drug for hereditary angioedema and BridgeBio’s treatment for transthyretin amyloidosis in cardiomyopathy patients.
Approvals
The European Medicines Agency wants to work with other regulators as well as industry, scientists and patients to “find solutions for the most pertinent problems in psychedelic research” and improve treatment options for psychiatric disorders more broadly, says the agency.
Novel agents from the biologics center had a median review time of 10.6 months, beating the drugs center's median of 11.8 months thanks to fewer multicycle and more priority reviews.
Review times for all 61 novel agents the US FDA approved in 2024.
Marketing authorization applications for five new advanced therapy medicinal products were filed with the European Medicines Agency in 2024, the highest number in any year since 2020. The Pink Sheet looks at these and two other ATMPs that might get the thumbs up from the European Medicines Agency in 2025.
Complete Response Letters
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.
The US FDA rejected Intercept’s bid to convert Ocaliva’s accelerated approval in primary biliary cholangitis, meaning withdrawal prospects loom.
Drug Review Profiles
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.
Post-Marketing Regulation & Studies
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.
The Therapeutic Goods Administration is advising companies to get up to speed with its new recall procedure, which is designed to improve the timeliness of recalls, alerts and corrections, and reduce regulatory burden for sponsors.
Himal Thakur said the required long-term safety study can replicate a clinical trial, which is not the intent.