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The EC has approved Roche's treatment for 5q spinal muscular atrophy (SMA), Evrysdi (risdiplam).
Roche has announced in a March 30, 2021 press release, that the European Commission (EC) has approved its treatment for 5q spinal muscular atrophy (SMA), Evrysdi (risdiplam), in patients aged two months and older with a clinical diagnosis of SMA Type 1, Type 2, or Type 3, or with one to four SMD2 copies.
The approval decision was made based on data from two clinical studies in which Evrysdi demonstrated a favorable efficacy and safety profile in a broad spectrum of patients living with SMA. Roche is now working with health authorities across the European Union (EU) to achieve extensive and rapid access to the therapy for patients with SMA.
“Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU,” said Levi Garraway, Roche’s chief medical officer and head of Global Product Development, in the press release. “By avoiding the need for in-hospital administration, Evrysdi can reduce the treatment burden on those living with SMA, their caregivers, and healthcare systems. We thank the SMA community for their partnership, the trust they have placed in us and their unyielding commitment to achieve this significant milestone.”
“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,” added Dr Nicole Gusset, president of SMA Europe, in the press release. “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.”