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EU regulators to review Evrysdi for SMA

EU regulators have agreed to review Roche’s Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA), a severe, inherited, progressive neuromuscular disease that causes muscle atrophy and disease-related complications.

The European Medicines Agency previously granted PRIME (PRIority MEdicines) designation to the drug for the treatment of SMA, thus providing a pathway for its accelerated evaluation.

Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that cause SMN protein deficiency. The drug is administered daily at home in liquid form by mouth or by feeding tube.

The submission is based on data from the dose-finding Part 1 and confirmatory Part 2 of the FIREFISH and SUNFISH studies which evaluated the efficacy and safety of Evrysdi (risdiplam) in symptomatic infants with type 1 SMA aged 2 to 7 months and in people with types 2 or 3 SMA aged 2 to 25 years, respectively.

It also incorporates safety data from JEWELFISH, a trial in people with all types of SMA aged 1 to 60 years previously treated with other SMA therapies.

In FIREFISH, 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least five seconds as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).

Additionally, 90% (19/21) of infants were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older. Untreated infants would not be expected to be able to sit independently, and only 25% would be expected to survive without permanent ventilation beyond 14 months of age.

In SUNFISH, children and adults treated with Evrysdi experienced a clinically-meaningful and statistically significant improvement in motor function at 12 months (1.55 point mean difference) compared to placebo, as measured by a change from baseline in the Motor Function Measure-32 (MFM-32) total score.

“The acceptance of the MAA for Evrysdi marks an important milestone as we continue towards the goal of making this ground-breaking therapy available globally to a broad range of SMA patients,” said Stuart Peltz, PTC’s chief executive.

“Evrysdi has consistently demonstrated clinically meaningful results in multiple clinical trials with a favorable safety profile. Evrysdi is an oral medicine that can be delivered and taken at-home, which is particularly important for SMA patients during the COVID-19 global pandemic.”

The EMA’s acceptance of the drug’s marketing application, which comes just days after Evrysdi’s approval in the US, triggers a $15 million payment to PTC Therapeutics from Roche.