Sarepta Must Balance Exondys 51 Confirmatory Trials And Sales
Executive Summary
How will the confirmatory trial necessary to convert accelerated approval to full approval affect sales given the small size of the target population?
You may also be interested in...
PTC Tumbles On Ataluren Failure In Cystic Fibrosis
With a second Phase III failure in nonsense mutation CF, the New Jersey biotech ends development of ataluren for CF, but continues with the drug in three smaller indications.
Sarepta Winning Payer War Over Duchenne Muscular Dystrophy Drug Coverage
Exondys 51 has been met with recommendations for restrictions by many insurers, but after consultations with experts and Sarepta execs coverage is expanding.
Sarepta Eyes Patient Outcomes To Boost Exondys 51's European Review
EMA and payers want to know more about daily life improvements from US FDA-approved Duchenne muscular dystrophy treatment.