PTC Readies China Plan Amid US FDA Translarna Setback
Executive Summary
PTC is engaging physicians and Duchenne muscular dystrophy patients in China to access a largely untapped market but regulatory and reimbursement-related uncertainties could pose challenges.
You may also be interested in...
Pfizer Bets On Gene Therapy For Rare Diseases In China
After excluding China from its early development plans for domagrozumab, Pfizer’s rare disease R&D team is now gearing to commence a clinical study of the investigational compound on Duchenne muscular dystrophy patients soon.
Set, Ready, Walk: Gilead Unveils Sovaldi Strategy In China
Rapid regulatory changes in China are expected to trigger a flurry of new drug launch activities. As Q3 earnings season plays out, Gilead COO Kevin Young talks about doing ‘the right thing’ while rolling out products like the high-priced Sovaldi in China's private-pay market.
PTC To Appeal Translarna's Complete Response Letter From US FDA
PTC Therapeutics appears to be hoping that CDER Director Woodcock will do for ataluren what she did for Sarepta's exon-skipping Duchenne muscular dystrophy treatment Exondys 51 since dispute resolution request would first go to Office of New Drugs, where Woodcock is acting director.