Chugai’s Risdiplam Receives MHLW’s Orphan Drug Designation for Spinal Muscular Atrophy (SMA)

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

Click here to read full press release/ article | Ref: Chugai | Image: Twitter