Approval Standards
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.
The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.
CBER's Nicole Verdun wants rare disease sponsors with stalled treatments to circle back because the FDA's evolved thinking on clinical trial designs may offer another opportunity for cast away products.
A European Medicines Agency official provided an overview and update of the support the EMA offers developers of innovative products and discussed, among other things, its Innovation Task Force, Quality Innovation Group and a scientific advice pilot for clinical trials.
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 Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.
The European Medicines Agency’s chief medical officer Steffen Thistrup argued that patients are able to understand the “complex regulatory and scientific issues” that the EMA committees discuss when they assess drugs.
The lack of a formal analysis of efficacy in castration-resistant prostate cancer patients without HRR mutations doomed Pfizer’s Talzenna at the US FDA's Oncologic Drugs Advisory Committee, as FDA oncology chief Richard Pazdur says chance cannot be ruled out.
The use of J&J’s Darzalex Faspro in high-risk smoldering multiple myeloma could result in overtreatment, but the FDA committee voted in favor of the new claim for delay in development of multiple myeloma.
The FDA may have violated laws and regulations along with many norms in how it released its planned changes for COVID-19 vaccine approvals.
The Oncologic Drugs Advisory Committee will vote on the risk-benefit of UGN-102 for low-grade, intermediate-risk, non-muscle invasive bladder cancer based on data from a single-arm trial, but the FDA also seeks input on requiring randomized studies for future treatments.