Alnylam's Onpattro: Not Following FDA Guidance Amplifies Clinical Meaningfulness Concerns
Even if Alnylam can get a favorable advisory committee vote and is able sneak out an approval for Onpattro for ATTR-associated cardiomyopathy, its lack of data on hard clinical outcomes makes it unlikely to unseat Pfizer’s tafamidis for the rare disease.
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A restrictive label could be the result of the lukewarm endorsement from the majority of the agency’s Cardiovascular and Renal Drugs Advisory Committee for use of Onpattro for the treatment of ATTR-associated cardiomyopathy.
US FDA Guidance On Endpoints Incorporating Clinical Outcome Assessments Fulfills Patient-Focused Commitment
Stakeholders can weigh in during 4 May 2023 webinar as US FDA rounds out a PDUFA VI commitment with a draft of the fourth of four methodological guidances on patient-focused drug development.
US FDA advisory panel feels there could be a place for Intarcia’s exenatide drug-device combo – if the company is willing to put in the work.