Post Market Regulation & Studies

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.

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.