European MAA for Evrysdi accepted

PTC Therapeutics (Nasdaq: PTCT) today announced that the European Medicines Agency has accepted the Marketing Authorization Application (MAA) for Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA), with the news, hot on the heels of US approval for the drug just over a week ago, lifted the firm’s shares 3.6% to $50.79 in mid-morning trading.

The MAA was filed by PTC’s development partner Swiss pharma giant Roche (ROG: SIX). The EMA previously granted PRIME (PRIority MEdicines) designation to risdiplam for the treatment of people with SMA, providing a pathway for accelerated evaluation by the agency. The milestone triggers a $15 million payment to PTC from Roche.

"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, chief executive of PTC Therapeutics. "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," he noted.