World Regulators Wrestle With Advanced Therapies

 
• By Ian Schofield
  
Generate a summary of this article
Listen to this article
Beta

The development of advanced therapy medicinal products is dogged by a lack of regulation and guidance and differences in what should and should not be deemed an ATMP, according to speakers at last month’s pre-ICDRA gathering of world regulators.

Cooperation
There are still gaps in the regulation of advanced therapy products • Source: Shutterstock

Following their recent groundbreaking approvals, products like Novartis AG’s Kymriah (tisagenlecleucel) and Gilead Sciences Inc.’s Yescarta (axicabtagene ciloleucel) may be hogging the limelight where advanced therapies are concerned, but behind the scenes there are still many gaps to bridge before the whole gamut of cell, tissue and gene therapy products can be adequately regulated.

While a great deal of progress has been made so far on dedicated pathways and guidelines, the regulation of advanced...

Pink Sheet Podcast

Learn what's happening at the US FDA. On the go.

Derrick Gingery and the team bring you a weekly "Drug Fix".

SpotifyApple PodcastsWebsite

More from United States

COVID Vaccines: US FDA Changes Course, Wants New Formulas To Target KP.2 Subvariant ‘If Feasible’

 
• By Sue Sutter

Revised recommendation is based on most recent data on circulating strains in US, where KP.3 is now dominant. Novavax sticks with plans targeting the JN.1 ‘parent strain.’

Diversity Action Plans: What To Submit And When

 
• By Nielsen Hobbs

With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.

Stealth’s Elamipretide: Efficacy In Barth Syndrome Not Shown, US FDA Says

 
• By Sue Sutter

Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.

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

 
• By Sarah Karlin-Smith

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

More from North America

Stealth’s Elamipretide: Efficacy In Barth Syndrome Not Shown, US FDA Says

 
• By Sue Sutter

Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.

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

 
• By Sarah Karlin-Smith

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

Clinical Trial Diversity Action Plans Need Timely FDA Feedback, Groups Say

 
• By Sue Sutter

The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.