The European Commission (EC) has approved Roche’s spinal muscular atrophy (SMA) treatment Evrysdi for use in patients aged two months and older.
Evrysdi (risdiplam), which is administered daily at home in liquid form by mouth or by feeding tube, is indicated for use in patients with 5q SMA, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
The approval is based on data from two clinical studies – FIREFISH in symptomatic SMA Type 1 infants aged two to seven months and SUNFISH in symptomatic SMA Type 2 and 3 children and adults aged two to 25 years old.
In the FIREFISH study, 29% of infants treated with Evrysdi for 12 months were able to sit without support for at least five seconds, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition.
In the SUNFISH study, children and adults who received Evrysdi treatment also experienced clinically meaningful and statistically significant improvement in motor function at 12 months compared to placebo, as measured by a change from baseline in the Motor Function Measure-23 (MFM-32) total score.
Roche said that it is working with reimbursement and assessment bodies across Europe to ensure ‘broad and rapid access to patients in need’.
In Germany, Evrysdi will be accessible to patients in the coming days and in France from early April, via the cohort Temporary Authorisation for Use.
"We welcome today’s approval of Evrysdi for people with SMA in Europe, and are proud of the role we have played in its development and of our partnership with Roche,” said Nicole Gusset, president of SMA Europe.
“A recent survey conducted by SMA Europe showed that a large proportion of people with SMA in the EU were not receiving an approved treatment which leaves them feeling helpless and frustrated. It is vital that we work together with health authorities, regulators and industry to ensure we can get this medicine to the patients who need it as soon as possible,” she added.