Product Reviews

At the advisory committee review of Stealth’s Barth syndrome treatment elamipretide, Office of New Drugs Director Peter Stein clarified the circumstances where clinical data in a related indication could serve as confirmatory evidence for a single adequate and well-controlled study.

Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.

Pink Sheet reporter and editors discuss experts’ experience helping drug sponsors negotiate prices with CMS and the FDA’s proposal for a new pathway to update vaccines before a pandemic is declared.

US FDA Office of New Drugs Director Peter Stein says review divisions have made the case that a discussion-only meeting would solicit the necessary input.

Concizumab sponsor Novo Nordisk and a number of other companies could soon learn whether or not the European Medicines Agency will give their respective drugs the marketing thumbs up.

A newly published draft implementing act sets out the procedural rules for the joint scientific consultations that are foreseen by the EU’s Health Technology Assessment Regulation.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add four new products, including Anzupgo, LEO Pharma's treatment for moderate to severe chronic hand eczema in adults for whom topical corticosteroids are inadequate or inappropriate.

The company’s decision to withdraw the drug was based on “the totality of clinical data that now indicates the overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population.”

Nipocalimab is an investigational FcRn blocker for treating generalized myasthenia gravis that was also recently filed for regulatory review in the US.

Companies will initially be able to submit their dossiers to the Health Sciences Authority via the eCTD portal on a voluntary basis.

New guidance from the European Medicines Agency explains how in vitro and in vivo models may be used instead of clinical data for the purpose of establishing therapeutic equivalence in a stepwise approach.

The UK regulator approved 16 medicines containing a new active substance in the first half of this year, while the European Commission issued marketing authorizations for 20 NAS-containing drugs.

The company will file a new drug application with the US FDA by the end of the year for tradipitant in a different indication, motion sickness.

Sponsor of psychedelic PTSD treatment will try rarely successful formal dispute resolution process after receiving a complete response letter. 

US FDA would need to approve 44 novel agents by year-end to match 2023’s big total, but only 40 candidates are known to have user fee goals in the second half of 2024.

A Pink Sheet analysis finds US approval preceded European Union clearance for 80% of products approved in both areas, but when EU approval came first, it beat the FDA by a median of 13 months. US-first approvals came close to six months before the EU.

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 US FDA Pediatric Advisory Committee uses web-posted reviews of “low safety risk” products to keep up with postmarketing monitoring requirements amid rising interest in pediatric development.

The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion.

Long-term follow-up requirements have taken a conservative approach, but could be ripe for re-examination and global harmonization, Kite Pharma executive director says, while former FDA gene/cell therapy office head Wilson Bryan wants the classwide REMS eliminated.

An FDA question to a patient concerned about the negative impacts of the CAR-T boxed label warning during a recent listening session indicates the agency may be thinking about improving its dissemination of the information.