Swiss pharmaceutical giant Roche (SWX: ROG) has announced that its blockbuster nervous system drug Evrysdi (risdiplam) has received market approval from the National Medical Products Administration (NMPA) to treat spinal muscular atrophy (SMA) in patients aged 16 days and above. This marks an expansion of the drug’s indication in China, where it was first approved for SMA in patients aged 2 months in June 2021 and entered the National Reimbursement Drug List (NRDL) in 2022.
Approval Based on RAINBOWFISH Study Results
The approval is supported by the interim results of the RAINBOWFISH study, an open-label, single-arm, multi-center study that assessed the efficacy, safety, tolerance, pharmacokinetics, and pharmacodynamics of Evrysdi in children born up to 6 weeks with a genetic diagnosis of SMA and no symptoms. The study demonstrated significant milestones among infants with 2 or 3 copies of the SMN2 gene: 100% achieved sitting alone, 67% achieved standing alone, and 50% achieved walking alone after one year of treatment with Evrysdi. All children survived without the need for permanent ventilation, and all patients maintained the ability to swallow solid food, indicating the drug’s positive impact on various aspects of SMA patients’ lives.
Understanding Spinal Muscular Atrophy (SMA)
SMA is one of the most common genetic diseases leading to infant death, primarily linked to a lack or mutation of the SMN1 gene, which results in insufficient expression of the systemic functional SMN protein. This deficiency affects patients’ exercise, breathing, swallowing, spleen, heart, pancreas, and other organs, making treatments like Evrysdi crucial for improving their quality of life.-Fineline Info & Tech
