Post-Marketing Regulation & Studies

Gene Therapy And Secondary Malignancies: Integration Site Analysis Can Only Be Part Of The Answer

 

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.

‘Hard Truths’ For Sentinel: US FDA Scales Back Active Surveillance Ambitions

 
• By 

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.

AstraZeneca’s Andexxa Could Be Headed For Withdrawal After US FDA’s Negative Advisory Committee Preview

 

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

EMA Urges Variation Submissions By 30 November Before Holiday Closure

 

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.


‘Unforeseen’ Variations & ‘Super-Grouping’ Addressed In New EU Guidance

 
• By 

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.”

Growth Spurt: Pediatric Labeling Is On The Rise, Taxing US FDA Safety Monitoring

 

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

 
• By 

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.

CAR-T Therapies: Stakeholders Urge Rethinking Of Postmarketing Requirements And REMS

 
• By 

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.


US FDA Acknowledges Patient Frustration With CAR-T Boxed Warning

 

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 Podcast: Pfizer Pulls Sickle Cell Drug, ODAC Calls Back Approved Products, New AAM CEO

 
• By 

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.

All Comers No More? US FDA AdComm Supports PD-L1 Threshold In Esophageal, Gastric Cancer

 

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.

Pfizer Withdraws Oxbryta, But Overall Market Impact May Be Limited

 

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.


New US FDA Adcomm Trend: Approved Cancer Meds Get Another Look When Competitors Coming

 

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

Intercept’s Ocaliva Falls At US FDA Panel; Is Accelerated Approval Withdrawal On The Horizon?

 
• By 

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.

Ocaliva Confirmatory Trial Failed To Verify Benefit In Primary Biliary Cholangitis, US FDA Says

 
• By 

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.  

EMA Probe Prompts New Safety Measures For Decades-Old Drug Metamizole

 

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.


US Medicare To Offer Guidance On Real World Evidence To Support Coverage

 
• By 

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.

US FDA Panel To Consider Compounding Ban For Makena’s Active Ingredient

 
• By 

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.

EU Revokes Intercept/Advanz’s Ocaliva Approval As Moment Of Reckoning Nears in US

 
• By 

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.

AstraZeneca’s Imfinzi Squeezes Through: Is FDA Reversing Course Or In A Transition Period?

 

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.