Roche Malaysia has announced that the National Pharmaceutical Regulatory Agency (NPRA) in Malaysia has approved Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children two months and older.
Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy is a rare disease affecting patients two months of age and older. SMA is one of the genetic causes of death in infants. 5q SMA is one form of the disease1. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition.
“Children with SMA may start showing symptoms as early as the first few days of life and lose muscle strength over time, so early treatment is crucial before the weakness begins. While the age of onset and range and severity of symptoms varies from each individual, the physical and emotional challenges from SMA impacts the entire family,” said Dr Ch’ng Gaik Siew, Paediatrician specializing in Clinical Genetics. “Until recently, there have been no medical advances to alleviate the debilitating symptoms from SMA. With more treatment options available, people with SMA and their families now have more hope that they may be able to overcome the challenges from their disease.”
“We are proud to offer children with this rare neurological disease a new treatment option that can provide meaningful benefits and can be taken at home instead of visiting the hospital and this can help reduce the burden of hospital administration. We look forward to working with the healthcare community to drive more awareness around this disease with the hopes of improving outcomes,” said Deepti Saraf, General Manager of Roche Malaysia Sdn. Bhd.
“A call to policymakers and insurance providers to ensure a clear and committed policy direction, covering regulations and incentives, and the provision of genetic non-discrimination insurance, is also being made. Lastly, increasing community education and awareness, as well as future research on SMA, is also recommended,” added Deepti.
Evrysdi is administered daily at home in liquid form by mouth or by feeding tube. The approval is based on data from two clinical studies, designed to represent a broad spectrum of people living with SMA: 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 SMA2. Evrysdi demonstrated a favorable efficacy and safety profile, with the safety profile established in the trial.
How Evrysdi Works
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.
Reference: 1. Arnold, W., Kassar, D., & Kissel, J. (2014). Spinal muscular atrophy: Diagnosis and management in a new therapeutic era. Muscle & Nerve, 51(2), 157-167. https://doi.org/10.1002/mus.24497
2. Mercuri, E., Deconinck, N., Mazzone, E., Nascimento, A., Oskoui, M., & Saito, K. et al. (2022). Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. The Lancet Neurology, 21(1), 42-52. https://doi.org/10.1016/s1474-4422(21)00367-7