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.
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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.