Post-Marketing Regulation & Studies

The confirmatory trial for the anticoagulant reversal agent raised safety and efficacy concerns from the agency.

The European Medicines Agency is encouraging companies to submit the type I variations they want to make to their marketing authorizations by the end of this month.

Updated guidance issued by the EU regulator explains how to comply with the provisions of the amended Variations Regulation, which will apply to new submissions from January 2025 and is intended to make lifecycle management “more efficient and future-proof.”

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. 

Pink Sheet reporters and editors discuss Pfizer’s abrupt decision to withdraw Oxbryta, the US FDA’s Oncologic Drugs Advisory Committee bringing sponsors of approved products back to discuss labeling changes while competitors are pending, and the new CEO of a generic industry trade association.

Maturing data supporting first-line indications for Merck’s Keytruda, Bristol’s Opdivo and BeiGene’s Tevimbra show inadequate efficacy for patients at the lowest level of PD-L1 expression in esophageal and gastric cancers.

The withdrawal amid concerns about the risk-benefit ratio reduces treatment options for sickle cell disease, but it is not expected to have much effect on the SCD market, including the gene therapies.

Recent ODAC meetings suggest the FDA will expand focus beyond applications with pending approval decisions to refining trial designs and drug labels for competitors. 

Obeticholic acid has not confirmed clinical benefit and the benefit-risk profile is not favorable in primary biliary cholangitis, the FDA advisory committee said. The agency must now decide whether to keep Ocaliva on the market with new study requirements or seek withdrawal.

A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.  

The European Medicines Agency’s safety committee has concluded that the benefits of medicines containing metamizole continue to outweigh the risks but that new measures are needed to minimize the serious outcomes of agranulocytosis.

The US Medicare agency is encouraging use of observational trials to fulfill Coverage With Evidence Development requirements, but details will await an upcoming guidance on “fit for purpose” studies.

Covis’ preterm birth prevention drug was withdrawn in April 2023. The agency now is considering whether hydroxyprogesterone caproate should be added to the list of products withdrawn or removed from the market after being found to be unsafe or not effective.

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.

Even as US FDA expressed concern about the perioperative trial design, AstraZeneca’s ability to snag NSCLC indication without demonstrating the benefits of both the neoadjuvant and adjuvant phases of therapy highlights slow pace of agency policy shifts.

The first engineered cell therapy for a solid tumor could be submitted for full approval in synovial sarcoma next year.

The European Medicines Agency is seeking feedback on how to standardize the prospective planning and reporting of cardiovascular safety endpoints in oncology trials.