KUALA LUMPUR, June 16 – The National Pharmaceutical Regulatory Agency (NPRA) has authorised Roche’s Evrysdi (risdiplam), the first drug approved in Malaysia to treat spinal muscular atrophy (SMA).
SMA is a rare genetic disease that can kill infants and causes muscle weakness and progressive loss of movement, particularly for adults living with the condition, affecting one’s ability to walk, eat, or even breathe.
“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,” Dr Ch’ng Gaik Siew, a paediatrician specialising in clinical genetics, said in a statement released by pharmaceutical company Roche Malaysia yesterday.
“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.”
Roche Malaysia general manager Deepti Saraf urged policymakers and insurance providers to provide clear policy directions on regulations, incentives, and non-discrimination genetic insurance on the newly registered SMA treatment.
“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,” she said.
Evrysdi, developed by Swiss pharmaceutical company Roche, is a liquid medicine that is administered daily at home by mouth or feeding tube.
The drug increases and sustains the production of the survival motor neuron (SMN) protein, treating SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.
Roche Malaysia said NPRA’s approval was based on data from two clinical studies, called SUNFISH, that looked at the effect of Evrysdi on symptomatic Type 2 and 3 SMA in children and in adults.
The SUNFISH trials saw significant improvement in motor function and upper limb function at 12 months for children and adults treated with Evrysdi. The medication also showed a favourable safety profile across the trials.
The most common adverse events reported were upper respiratory tract infection, pneumonia, common cold, fever, diarrhoea, headache, cough and vomiting.
Patient group Spinal Muscular Atrophy Malaysia estimates 3,200 SMA-affected individuals and 640,000 SMA carriers in the Malaysian population, based on SMA prevalence in the United States of one in 10,000 babies and one in every 50 Americans being a genetic carrier.