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Novartis CAR-T Therapy's Swift Approval Aided By REMS And New US FDA Review Model

 
• By Brenda Sandburg

Risk Evaluation and Mitigation Strategy for Kymriah requires the acute lymphoblastic leukemia treatment be administered by certified centers, while a postmarketing study will assess safety over 15 years. FDA's early approval reflects efforts by the biologics center and new cross-cutting Oncology Center for Excellence to implement a more collaborative review model.

Decorin protein

FDA's swift approval of Novartis AG's chimeric antigen receptor T-cell therapy Kymriah (tisagenlecleucel) was aided by the company's Risk Evaluation and Mitigation Strategy (REMS), which requires hospitals dispensing the treatment to be certified.

FDA approved Kymriah, the first gene therapy to clear the agency, on Aug. 30 for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse

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More from Approvals

New EU Filings

 
• By Neena Brizmohun

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
• By Sue Sutter

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
• By Sue Sutter

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
• By Sue Sutter

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

More from Product Reviews

FDA Adcomms Are Back: Four Cancer Drugs, COVID-19 Vaccine Formulations To Get Reviews

 
• By Sue Sutter and Bridget Silverman

The Oncologic Drugs Advisory Committee will meet for two days in mid-May, followed by a Vaccines and Related Biological Products Advisory Committee’s review of the 2025-2026 COVID-19 vaccine formulation.

Unlocking Opportunities: How To Engage With The EMA On Animal Testing Alternatives

 
• By Neena Brizmohun

The European Medicines Agency, like its counterpart in the US, is increasingly focusing on the use of alternatives to animal testing.

Opioid Safety: US FDA Hears Mixed Messages On Adding Posmarketing Studies To Labeling

 
• By Sue Sutter

Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.