Sarepta Must Balance Exondys 51 Confirmatory Trials And Sales
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...
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.
Exondys 51 has been met with recommendations for restrictions by many insurers, but after consultations with experts and Sarepta execs coverage is expanding.
EMA and payers want to know more about daily life improvements from US FDA-approved Duchenne muscular dystrophy treatment.