Sephience Sales Momentum Prompts Citi to Upgrade PTC Therapeutics to Buy
Citi turns bullish on PTCT, citing strong Sephience sales in PKU and potential $1B+ peak, raising target to $108.
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PTC Therapeutics Beats Wall Street Estimates and Hikes 2026 Outlook on Strong Sephience Launch
PTC beat estimates, Sephience sales surged, losses small, outlook raised, and analysts upgraded stock on strong growth momentum.
Royalty Pharma Completes $240M Deal for Full Evrysdi Royalty Rights
Royalty Pharma acquires complete royalty interest in Roche's Evrysdi for $240M upfront plus milestones, consolidating ownership of the 8-16% tiered royalty stream.
FDA Feedback Rocks uniQure: Gene Therapy for Huntington’s Disease Delayed
UniQure's stock plummeted after the FDA unexpectedly decided that the current clinical trial data for its promising Huntington's disease therapy, AMT-130, is not adequate for immediate marketing application (BLA).
FDA Rejects Friedreich's Ataxia Drug Vatiquinone
The FDA declined PTC Therapeutics' vatiquinone for Friedreich's Ataxia, citing insufficient efficacy data and requiring more studies.
PTC SEPHIENCE: A New PKU Treatment Approved
FDA-approved SEPHIENCE (sepiapterin), an oral drug, treats PKU in patients 1 month+. SEPHIENCE aims to be the future standard of care.