PTC Misses Phase III In Friedrich Ataxia, But Isn’t Giving Up
Company Ends Gene Therapy R&D, Cuts Headcount
Pointing to data showing benefit in patients who completed 72 weeks of therapy, PTC thinks vatiquinone still could be viable for pediatric FA patients. Restructuring won’t affect Upstaza.
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PTC’s Phase III Data Sets Up PKU Competition With BioMarin
Phase III data for PTC’s sepiapterin shows statistically significant reduction of blood phenylalanine levels in patients with phenylketonuria. BioMarin has the only two approved drugs for PKU.
ICER Says Gene Therapies For Sickle Cell Likely To Improve Lives, But Points To Uncertainties
The drug pricing watchdog said improvement to patients’ quality and length of life from lovo-cel and exa-cel were likely, but noted ongoing uncertainty around durability and toxicity concerns.
Reata Lays Commercial Path For Skyclarys With First-Ever Friedreich’s Ataxia Nod
The company forecasts an addressable US patient market that comprises 90% of the total Friedreich’s population of 5,000 based on a broad label covering patients aged 16 and older.