기업
MFDS approved Roche's oral SMA drug 'Evrysdi'
by Dongyoung Park
Roche received an approval of 'Evrysdi' from Korean MFDS after 3 months of FDA’s approval..."Improving motor function in patients of various ages and disease types"
The oral medication for Spinal Muscular Atrophy (SMA) developed by Roche was approved in Korea three months after it was approved by the US Food and Drug Administration (FDA).
Roche Korea announced on the 3rd that its oral spinal muscular atrophy drug 'Evrysdi(Risdiplam)' has been approved for marketing by the Ministry of Food and Drug Safety.
“We are pleased to be able to provide treatment options to a wider range of patients in the field of rare diseases where drug development is difficult,” said Nick Horridge, CEO of Roche Korea. "We will contribute to the improvement of public health by continuously supplying innovative medicines in the neuroscience field."
Spinal Muscular Atrophy is a rare hereditary neuromuscular disease caused by a deficiency of the Survival Motor Neuron (SMN) protein essential for motor function. This is characterized by degeneration of various muscles, including motor function.
Evrysdi binds to the pre-mRNA of the abnormal SMN2 gene increasing the concentration of the normal SMN protein to treat spinal muscular atrophy. Roche Korea explained that Evrysdi can cross the blood-brain barrier (BBB), thereby increasing the expression of systemic normal SMN proteins, including the central nervous system. In addition, it is possible to provide customized prescription for patients based on age and weight, making possible to self-manage with once a day recommended dose as a liquid formulation.
Lee Seung-hoon, medical director of Roche Korea, said, “Evrysdi is a liquid formulation that is taken orally, so that even patients with spinal deformity can be treated without restrictions. We have confirmed the effect of improving motor function in various disease types and patients with wide range of age groups, from infants to adolescents and adults.”
Meanwhile, Evrysdi was approved by the U.S. FDA in August, and was designated as a drug for priority review by the European Medicines Agency (EMA) in 2018.
