FDA grants Evrysdi priority review for treatment of spinal muscular atrophy in infants

pharmafile | January 25, 2022 | News story | Manufacturing and Production  

The FDA has granted priority review of a supplemental new drug application (sNDA) for Evrysdi (risdiplam), to expand the indication to include pre-symptomatic infants under two months old with spinal muscular atrophy (SMA). If approved, Evrysdi would be the first medicine administered at home for pre-symptomatic babies with SMA.

The sNDA submission included interim data from the RAINBOWFISH study, which showed 80 percent of pre-symptomatic infants with SMA treated with Evrysdi for at least 12 months achieved motor milestones such as sitting without support, rolling, crawling, standing unaided, and walking independently.

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies, and when untreated is the leading genetic cause of infant mortality. It is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is essential to the function of nerves that control muscles and movement, and without it, nerve cells are unable to function correctly.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body, and is essential to the maintenance of healthy motor neurons and movement. Evrysdi was previously based on PTC’s splicing platform.

“The results demonstrating that almost all of the pre-symptomatic infants achieved motor milestones comparable to healthy infants is tremendous,” said Stuart W Peltz, PhD, CEO, PTC Therapeutics. “The granting of the Priority Review for Evrysdi recognises this and the significant need to treat babies with SMA as early as possible. We are proud that such a transformative treatment for patients living with SMA came from our splicing platform.”

Lina Adams

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