Approval Standards
The US FDA says its release of complete response letters for products subsequently approved by the agency is just a first step toward releasing rejection letters when they are issued, assuming legal issues can be resolved.
New US FDA drugs center director George Tidmarsh’s data-centric comments at a Reagan-Udall Foundation meeting on unapproved fluoride products could suggest patients’ and caregivers’ experiences will carry less weight in benefit-risk decisions under his leadership.
The European Medicines Agency’s human medicines committee, the CHMP, has recommended 14 new medicines for pan-EU approval this month, including five orphan medicines.
The European Medicines Agency recommended against pan-EU marketing authorization of Roche/Sarpeta’s gene therapy Elevidys for the treatment of Duchenne muscular dystrophy as the US reported another death in an Elevidys patient.
Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.
The US FDA's Psychopharmacology Advisory Committee emphasized high standards over regulatory flexibility while turning down Otsuka's brexpiprazole plus serotonin for post-traumatic stress disorder based on conflicting Phase III trials and post-hoc Phase II analyses.
US FDA's 25 novel approvals in 2025 fell short of the agency's five-year average count, driven by a drop in drugs center approvals; 45 user fee goals remain in second half of year.
FDA staff are trying to plan for program implementation in the absence of critical eligibility and other details, although guidance may be coming soon.
Bayesian statistics could help clinical trial sponsors to include external information in their analyses, but concerns around bias and incorrect conclusions remain, the European Medicines Agency said.
Companies can propose specific issues for discussion during the regulators’ monthly teleconferences, which focus on finding areas of agreement and reasons for nonalignment on specific pediatric development plans or general issues.
Extended-release stimulants will add a new ‘Limitation of Use’ about weight loss in children under 6 years, reflecting concerns about overuse from the Make America Healthy Again commission, a concern multiple studies did not find valid.
Roche’s filing for Elevidys in the EU followed applications in Middle Eastern countries because those markets accept applications based on approval by the US Food and Drugs Administration.
An ongoing proof-of-concept study by the European Medicines Agency testing the use of US-style standards for submitting non-clinical raw data in marketing applications has received positive feedback from both industry and regulatory assessors so far.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Tepezza, Amgen's treatment for moderate to severe thyroid eye disease.
US FDA critics have long decried the practice of using informal communications to convey regulatory expectations as podium policy. In the Makary era, the phrase may need to be updated because the agency now seems to be specializing in policy by podcast.
While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.
The FDA will use electronic health records to “get eyes” on drugs immediately after approval, Commissioner Martin Makary told the DIA 2025 Global Annual Meeting. He also wants to reduce development time with better communication and new approval pathways.
The group had been laid off as part of the 1 April reduction-in-force, which led to missed guidance publication deadlines.
Julian Beach, interim executive director of healthcare quality and access at the UK drug regulator, the MHRA, tells the Pink Sheet how the agency is responding to increased efforts to develop bacteriophage therapies.
The US FDA ended efforts to regulate lab-developed tests as medical devices for now when it did not appeal a decision from the Eastern District of Texas that tossed out the agency's final rule.