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EMA recommends five new medicines, including a first-in-class small interfering RNA treatment

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The EMA’s April meeting of the Medicinal Products for Human Use Committee (CHMP) recommended five new medicines and nine existing medicines with extended therapeutic indications for approval.

The new medicines include a gene therapy for spinal muscular atrophy (SMA) - Itvisma (onasemnogene abeparvovec, Novartis), a treatment for non-relapsing secondary progressive multiple sclerosis - Cenrifki (tolebrutinib, Sanofi), and a first-in-class RNA interference (RNAi) mechanism-based medicine to help block a harmful protein produced in the liver that inhibits the breakdown of triglycerides, for the treatment of a rare inherited disease...

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