Topic: Duchenne muscular dystrophy
Without any new data, Sarepta appealed an FDA rejection and got a surprising nod for Vyondys 53—its second med approved on a surrogate marker.
Duchenne muscular dystrophy drug Emflaza sparked controversy under its previous owner, but its new owner, PTC Therapeutics, is forging ahead.
Sarepta Therapeutics’ DMD therapy Exondys has already been knocked for its skimpy clinical data. Now a price watchdog says it’s also too expensive.
Emgality will likely chase Amgen and Novartis’ first-in-class Aimovig and Teva's Ajovy in the CGRP migraine field.
Sarepta CEO Douglas Ingram may have netted biopharma's biggest 2017 compensation package after six months on the job.
Takeda and Shire struck a buyout deal worth $64B; Nektar and Takeda teamed up for a combo cancer trial; and more.
PTC is facing questions about an issue that has dogged the company since it bought DMD drug Emflaza from Marathon last March: the drug’s rising price.
JLABS lands first ex-North America site in Shanghai, Sanofi's Dengvaxia situation quickly worsens in the Philippines, Astellas buys DMD biotech.
Sarepta’s new CEO pick, Doug Ingram, wins plaudits for commercial savvy, CNS expertise—and sets the stage for more M&A chatter.