Shots:
- PTC Therapeutics to receive $650M as upfront in cash in exchange for ~43% of the risdiplam royalties, up to a specified amount while PTC maintains the majority of the risdiplam royalty stream and retains all economics associated with up to ~$400M in remaining regulatory and commercial milestones
- The collaboration enables PTC to further develop and expand its innovative rare disorder portfolio, including its validated splicing, Bio-e and gene therapy platform
- Risdiplam is an investigational SMN2 splicing modifier for SMA, its NDA is under the US FDA’s Priority Review with anticipated PDUFA date as Aug 24, 2020. The company is planning to submit MAA to the EMA as well as filings in other international markets
Click here to read full press release/ article | Ref: PTC Therapeutics | Image: RTTNews
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