Therapy Areas: Central Nervous System
FDA Approves Genentech's Evrysdi for Use in Babies Under Two Months with Spinal Muscular Atrophy
1 June 2022 - - The US Food and Drug Administration has approved a label extension for Evrysdi (risdiplam) to include babies under two months old with spinal muscular atrophy, US-based biotechnology company Genentech, a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY), said.

The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. Evrysdi is now approved to treat SMA in children and adults of all ages.

Of the babies with 2 or 3 copies of the SMN2 gene, 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently.

All infants were alive at 12 months without permanent ventilation.

As part of the label extension, the Evrysdi prescribing information has also been updated to include recent two-year pooled data from Parts 1 and 2 of the FIREFISH study, which demonstrate long-term efficacy and safety in symptomatic infants with Type 1 SMA.

The study enrolled babies aged 1-7 months and after two years of treatment with Evrysdi at the recommended dose, 60% of infants were able to sit without support for 5 seconds, 40% for 30 seconds and 28% of infants were able to stand.

Without treatment, infants do not achieve these milestones in the natural history of the disease.

There were no treatment-related adverse events leading to withdrawal. The most common adverse reactions were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting and cough.

Evrysdi is approved in 81 countries and the dossier is under review in a further 27 countries. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings.

Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Evrysdi is a survival motor neuron 2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein in the central nervous system and peripheral tissues as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Evrysdi was granted PRIME designation by the European Medicines Agency in 2018 and Orphan Drug Designation by the U.S Food and Drug Administration in 2017.

In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery. Evrysdi is currently approved in 81 countries and the dossier is under review in a further 27 countries.
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