2024-05-22 07:53:10 ET
Summary
- Translarna remains conditionally approved in Europe, despite a negative CHMP opinion and lacking U.S. approval.
- PTC's diverse pipeline includes potential treatments for PKU and FA, but faces significant commercial challenges.
- Financial analysis shows a decent cash runway and manageable short-term liabilities, but PTC is still unprofitable.
- PTCT is a high-risk investment; recommended to hold, as current risks outweigh potential high returns.
Translarna Tiptoes Through European Red Tape
PTC Therapeutics ( PTCT ) received some welcomed news earlier this week. Its lead product, Translarna for Duchenne muscular dystrophy [DMD], was permitted to stay on the market by the European Commission [EC]. Translarna is thought to work by promoting the readthrough of premature stop codons in the mRNA, allowing the production of functional dystrophin protein in muscle cells. This is theorized to slow disease progression in DMD patients. The EC green light came despite a negative opinion from the Committee for Medicinal Products for Human Use [CHMP] in January. As Translarna was only "conditionally approved" in 2014, it is subject to annual renewals by the EC. Translarna has yet to be approved by US regulators due to insufficient proof of benefit, among other reasons ....
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PTC Therapeutics: Riding The Regulatory Rollercoaster With Translarna