Risdiplam received orphan drug designation from the MHLW on
'As potentially the first oral drug for SMA, risdiplam demonstrated clinically meaningful treatment effects across infants and adults in two clinical studies for Types 1, 2 and 3 SMA,' said Chugai's President and COO, Dr.
This application is based on the results from the FIREFISH study in infants with symptomatic SMA Type 1 and SUNFISH study in children and young adults with SMA Type 2 or 3. SUNFISH is the only placebo-controlled trial to include children, teenagers and adults with SMA.
About risdiplam
Risdiplam is an investigational, oral medicine designed to increase and sustain survival motor neuron (SMN) protein levels in the central nervous system and throughout the body. It is designed to help the SMN2 gene produce more functional SMN protein to better support motor neurons and muscle function. Risdiplam was approved in the
About spinal muscular atrophy (SMA)
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease that causes muscular atrophy and muscle weakness due to degeneration of the spinal motor nerve cells.1) It is the most frequently observed life-threatening genetic disease in infants.2) The incidence of SMA from infancy to childhood is one to two in 100,000 individuals.3) The causative gene for SMA is the survival motor neuron (SMN) gene. The disease develops because of insufficient production of functional SMN protein from SMN2 genes alone, in addition to the dysfunction of the SMN1 gene.4)
Contact:
Tel: +81-3-3273-0881
Email: pr@chugai-pharm.co.jp
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