FDA Approves Roche, PTC’s SMA Treatment, Evrysdi (Risdiplam)

WASHINGTON, D.C., The United States – The Food and Drug Administration (FDA) has approved Roche and PTC Therapeutics’ Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 months and up.

In FIREFISH and SUNFISH, two clinical trials containing more than 450 patients with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA, Evrysdi showed clinically-meaningful improvement in motor function. In addition, Evrysdi improved survival without permanent ventilation at 12 and 23 months, compared to natural history.

Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development said, “Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease.

“The strength and resolve of the SMA community have continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them.”

According to Roche, Evrysdi will be available in the U.S. within two weeks for direct delivery to patients’ homes through Accredo Health Group. Evrysdi is a liquid medicine administered daily at home either by mouth or feeding tube.

Reuters reported that the list price of Evrysdi will be based on weight until the patient reaches 44 pounds at around 6 years of age, at which point the price reaches a maximum of $340,000 per year. The annual price of treatment for a patient would be less than $100,000 for an infant under 2 years old and weighing 15 pounds.

Back in April, Roche submitted additional data on Evrysdi from the SUNFISH Part 2 study to the FDA, extending the Prescription Drug User Fee Act (PDUFA) date for review of the New Drug Application (NDA) to August 24, 2020; originally, a decision was expected by May 24, 2020. Prior, it was granted Priority Review and Orphan Drug and Fast Track Designations by the FDA.

Ahead of any regulatory approvals, Roche kicked off an early access program, the global Pre-Approval Access/Compassionate Use Program, for Evrysdi in Europe for Type 1 SMA patients who cannot receive an approved treatment for their disease.

Evrysdi was provided free of cost to patients who could not be treated with other available drugs for medical or regulatory reasons.

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