Post-Marketing Regulation & Studies
Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.
The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.
The Therapeutic Goods Administration is advising companies to get up to speed with its new recall procedure, which is designed to improve the timeliness of recalls, alerts and corrections, and reduce regulatory burden for sponsors.
Himal Thakur said the required long-term safety study can replicate a clinical trial, which is not the intent.
The landscape for primary biliary cholangitis or cirrhosis, PBC, is set to change if Intercept withdraws Ocaliva, which received its fourth safety alert from the US FDA and lost conditional marketing authorization in EU. Pink Sheet studies data from Citeline’s Pharmaprojects and separately Evaluate Pharma to reveal a promising clinical trials landscape and a sizeable market
Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.
The US FDA seeks to better understand and predict insertional mutagenesis in gene therapies like bluebird bio's Skysona, which use integrating viral vectors, but a workshop on integration site analysis highlighted the approach's complexity and limitations.
The agency will focus less on new capabilities and more on meeting explicit mandates for the Sentinel active surveillance system, in part due to budgetary realities.
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.
Sponsors Aim To Avoid Confirmatory Trial Hurdles That Plagued Ocaliva In Primary Biliary Cholangitis
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.