Chugai's Risdiplam Receives MHLW's Orphan Drug Designation for Spinal Muscular Atrophy (SMA)
Shots:
- The MHLW’s ODD follows P-II/III (FIREFISH- SUNFISH) study assessing Risdiplam in patients with spinal muscular atrophy (SMA) in Japan
- Chugai & Roche collaborated for the clinical development Risdiplam- first oral systemic therapy for SMA
- Risdiplam (RG7916) is survival motor neuron-2 (SMN2) splicing modifier targeted to increase SMN protein levels in CNS and has received ODD in EU- Switzerland & the US- FDA’s FT designation- EMA’s PRIME designation in Dec’18. In 2020 Chugai also plans to file a regulatory application for risdiplam in Japan
Ref: Chugai | Image: Twitter
Click here to read the full press release
This content piece was prepared by our former Senior Editor. She had expertise in life science research and was an avid reader. For any query reach out to us at connect@pharmashots.com