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US FDA Again Asked To Hold Off On Approval Of Ryoncil Pediatric Cell Therapy

 
• By Brenda Sandburg

Attorney says new information Mesoblast submitted following ‘complete response’ letter does not include data from randomized controlled trial, which he says is needed to provide substantial evidence of efficacy in treatment of children with graft-versus-host disease.

Graft-versus-host disease
Additional study is needed for approval of cell therapy for pediatric graft-versus-host disease, petitioner tells FDA • Source: Shutterstock

Two years after receiving a ‘complete response’ letter from the US Food and Drug Administration on its biologics license application for Ryoncil (remestemcel-L) for treatment of pediatric steroid-refractory acute graft-versus-host-disease, Mesoblast Limited submitted new information to the agency. But an attorney contends that the submission is inadequate because Mesoblast did not conduct a randomized controlled study of the allogeneic cell therapy product.

The Australian company announced on 3 October that it had submitted “substantial new information on clinical and potency assay items identified in the

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More from Approval Standards

US FDA Advisory Committee Misperceptions Abound … At HHS

 
• By Michael McCaughan

HHS Secretary Robert F. Kennedy Jr. regularly decries the “conflicts of interest” he believes abound in advisory committees, but his concerns, as well as a recent “policy directive” eliminating industry representatives, seem driven by a misunderstanding of the panels’ jobs.

US FDA Guidance Roadblock: Writing Continues, But Publication Has Slowed Or Stopped

 
• By Derrick Gingery

Cuts to the FDA’s policy and legal personnel have prevented dozens of product-specific guidances for generic drug development from being published.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 
• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
• By Kate Rawson

Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

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US FDA Expands Surprise Foreign Inspections But Loses Associate Commissioner Michael Rogers

 
• By Sue Sutter

Commissioner Martin Makary’s repeated characterization of foreign facilities as being subject to lower standards than domestic counterparts may have contributed to Rogers’ decision to retire as head of the recently formed Office of Inspections and Investigations.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
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Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

Moderna Pivots To Increase Focus On Cancer Amid US Vaccine Policy Changes

 
• By Joseph Haas

During its first quarter earnings call, Moderna avoided criticizing vaccine policy changes, but de-emphasized its flu/COVID-19 vaccine for those under age 50 and prioritized cancer programs.