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)

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

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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