Pharma Focus Europe

CHMP Approves Evrysdi® for Infants Under Two Months with Spinal Muscular Atrophy

Saturday, July 22, 2023

PTC Therapeutics, Inc. (NASDAQ: PTCT) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for the extension of the Evrysdi® (risdiplam) marketing authorization in the European Union (EU) to include infants under two months of age. The final decision on approval is expected from the European Commission later this year.

Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed that the CHMP opinion marks a significant milestone in the development of Evrysdi, emphasizing the importance of providing oral, at-home administered treatments early in the disease to presymptomatic babies.

The CHMP opinion is based on the RAINBOWFISH interim analysis, which involved 18 participants, including six babies with two or three copies of the SMN2 gene who completed at least one year of study assessments. Among these babies, after one year of treatment with Evrysdi, 100% (6/6) were able to sit, 67% (4/6) could stand, and 50% (3/6) could walk independently. Notably, all infants were alive at 12 months without requiring permanent ventilation.

Evrysdi is developed based on PTC's splicing platform and is marketed by Roche in collaboration with the SMA Foundation and PTC Therapeutics. In the United States, Genentech, a member of the Roche Group, markets Evrysdi.

Spinal Muscular Atrophy (SMA) is a severe, progressive neuromuscular disease caused by a mutation in the survival motor neuron 1 (SMN1) gene, leading to a deficiency of SMN protein. Evrysdi is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that result in SMN protein deficiency. The drug is designed to distribute evenly throughout the body, including the central nervous system, and is administered daily at home in liquid form by mouth or feeding tube.

Evrysdi's approval and recognition have been granted in various countries, and it has been hailed for its potential in treating SMA. The RAINBOWFISH study, an open-label, single-arm, multicenter study, has investigated the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies from birth to six weeks of age with genetically diagnosed SMA who are not yet presenting symptoms. The study is now fully enrolled.

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