Roche has secured approval from the US Food and Drug Administration (FDA) for its Evrysdi (risdiplam) to treat spinal muscular atrophy (SMA) in adults and children aged two months of age and older.
Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier created for the treatment of SMA caused by mutations in chromosome 5q, which lead to SMN protein deficiency.
The company evaluated Evrysdi in over 450 people as part of a large clinical trial programme in SMA.
The approval was based on data from two clinical studies, including FIREFISH in symptomatic infants aged two to seven months and SUNFISH in children and adults aged two to 25 years. SUNFISH is claimed to be the first and only placebo-controlled trial to include adults with types 2 and 3 SMA.
Evrysdi demonstrated clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity such as types 1, 2, and 3 SMA.
The trial programme is comprised of infants aged two months to adults aged 60 with varying symptoms and motor functions such as people with scoliosis or joint contractures and those earlier treated for SMA with another medication.
Roche will start selling Evrysdi in the US within two weeks via Accredo Health Group, an Express Scripts speciality pharmacy.
Risdiplam secured PRIME designation from the European Medicines Agency (EMA) in 2018, while an orphan drug designation from the FDA and EMA in 2017 and 2019, respectively.
Roche chief medical officer and global product development head Dr Levi Garraway said: “Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favourable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease.
“The strength and resolve of the SMA community has continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them.”
In July this year, Roche and Blueprint Medicines have signed a global licencing and collaboration deal worth up to $1.7bn for the latter’s pralsetinib, an investigational treatment for RET-altered cancers.