Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older - Seite 2

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; p=0.0156) compared to placebo (1.36 points [95% CI: 0.61, 2.11]; -0.19 points [95% CI: -1.22, 0.84], respectively), as measured by a change from baseline in the Motor Function Measure-32 (MFM-32) total score. Children and adults also experienced significant improvement in upper limb function, a key secondary endpoint, at 12 months (1.59 point mean difference; p=0.0469) compared to placebo (1.61 points [95% CI: 1.00, 2.22]; 0.02 points [95% CI: -0.83, 0.87] respectively), as measured by a change from baseline in the Revised Upper Limb Module (RULM). The median age at enrolment was nine years.

Evrysdi demonstrated a favourable efficacy and safety profile, with the safety profile established across the FIREFISH and SUNFISH trials. The most common adverse events were upper respiratory tract infection, pneumonia, nasopharyngitis, pyrexia, constipation, rhinitis, diarrhoea, headache, cough and vomiting. There were no treatment-related safety findings leading to withdrawal from either study.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

More than 2,500 patients have now been treated with Evrysdi in clinical trials, compassionate use and real world settings, with patients ranging from birth to over 70 years of age and including those previously treated with other SMA-targeting therapies. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Roche is actively engaged with reimbursement and assessment bodies in European countries to enable broad and rapid access to patients in need. Reimbursement dossiers are being submitted in many countries in advance of an expected European Commission decision to minimise any delay in patient access.

*Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders

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