The oral medication is available for adults and children two months and older.
Roche announced in June 2022 that the Health Sciences Authority (HSA) in Singapore has approved Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease3. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition.
“With Evrysdi, people in Singapore with SMA now have a treatment option that can be taken at home, offering meaningful benefits for many living with this rare neurological disease,” said Dr. Sivabalan Sivanesan, Director of Medical & Regulatory Affairs at Roche Pharmaceuticals, Singapore. “By avoiding the need for in-hospital administration, Evrysdi can also help to reduce the treatment burden on those living with SMA, their caregivers and healthcare systems. We look forward with working with the SMA community to get this option to people with SMA as soon as possible.”
Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people from 2 months to 25 years of age, and various levels of disease severity, including Types 1, 2, and 3 SMA4. Infants achieved the ability to sit without support for at least five seconds, a key motor milestone not normally seen in the natural course of the disease. Evrysdi also improved survival without permanent ventilation at 12 and 23 months, compared to natural history.
Evrysdi works to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein, which is a protein found throughout the body and is critical for maintaining healthy motor neurons and movement. A liquid medicine, Evrysdi is administered daily at home by mouth or feeding tube.
The approval is based on data from two clinical studies, designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3 children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA. Evrysdi demonstrated a favourable efficacy and safety profile, with the safety profile established across both trials. [APBN]