China has granted its first approval for a stem cell therapy, to domestic firm Platinum Life Biotech’s umbilical cord-derived mesenchymal stem cell product for corticosteroid-failed acute graft-versus-host disease with gut involvement.
China has granted its first regulatory approval for a stem cell therapy despite mixed results from the supporting Phase II clinical trial on which the marketing green light was based.
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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.
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.
The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.
Novo Nordisk’s application seeking EU marketing approval to use its GLP-1 receptor agonist to treat cirrhotic metabolic dysfunction-associated steatohepatitis is one of 15 new drug filings the European Medicines Agency has started reviewing. Meanwhile, Arrowhead’s filing for its familial chylomicronemia syndrome, plozasiran, is being fast tracked.
A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.
India is pressing ahead with “big shifts” in its regulatory approach for cell and gene therapies. Reduced “layers of review”, tighter internal timelines, upcoming guidelines to facilitate CGT development and reforms in the constitution of expert committees are some of the key changes.
Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.
