3. >>Approvals

China Approves More Drugs For Cancer, Rare Diseases

 
• By Brian Yang

BeiGene's BTK inhibitor for leukemia/lymphoma and Sanofi/Genzyme's enzyme replacement therapy for MPS I among novel drugs newly approved in China.

Red Approved Stamp with Wooden handle Rubber Stamper Isolated on White Background.
CHINA APPROVES NEW CANCER, RARE DISEASE TREATMENTs • Source: Shutterstock

China's National Medical Products Administration (NMPA) has just approved Genzyme Corp.'s injected enzyme replacement therapy Aldurazyme (laronidase) for the long-term treatment of patients with mucopolysaccharidosis type I (MPS I).

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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.

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