PTC Therapeutics, Inc. PTCT announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA). SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control movement.
Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche Holding AG RHHBY. Roche leads the development of the drug.
Shares of PTC Therapeutics have gained 5% so far this year against the industry’s decline of 4.7%.
The submission was based on data from the pivotal FIREFISH and SUNFISH studies, which evaluated Evrysdiacross a broad patient population suffering from type 1, 2 or 3 SMA.
In FIREFISH study, 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least five seconds. Additionally, 90% (19/21) of infants were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older.
In the SUNFISH study, children and adults treated with Evrysdi experienced a clinically-meaningful and statistically significant improvement in motor function at 12 months compared to placebo. SUNFISH is the first and only placebo-controlled study to include adults with types 2 and 3 SMA.
In addition to FIREFISH and SUNFISH, Evrysdi is being evaluated in a broad range of people with SMA, including safety data from JEWELFISH, a study in people with all types of SMA aged 1 to 60 years previously treated with other SMA therapies. The results of an exploratory efficacy analysis from SUNFISH Part 1 study showed that Evrysdi significantly improved motor function after 24 months of treatment compared to the natural history data.
We remind investors that on Aug 10,the FDA granted approval to Roche’s Evrysdi for the treatment of patients (adults and children two months of age and older) with SMA. With the approval, the drugbecame the first at-home administered liquid medicine for people with SMA.
However, competition is stiff in the SMA market from the likes of Biogen’s BIIB Spinraza and Novartis’ NVS gene therapy, Zolgensma.
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