Stealth BioTherapeutics Inc.’s elamipretide moved a step closer to US approval with a Food and Drug Administration advisory committee’s positive vote that the drug is effective for the treatment of Barth syndrome.
Stealth’s Elamipretide Gets US FDA Panel Nod, But Not A Glowing Endorsement, For Barth Syndrome
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
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Sanofi’s Dupixent, Amgen’s Uplizna, and Bristol’s Opdivo seek new indications, while J&J hopes to start a franchise with nipocalimab and Stealth’s day of reckoning approaches.
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March features nearly 20 user fee goal dates, including a crowd of novel candidates for crowded areas like hemophilia, heart disease and urinary tract infections, as well as the potentially first therapies for a rare eye disease and hyperphagia of Prader-Willi syndrome.
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The US FDA approval of new meningococcal and chikungunya vaccines arrived in time for the 26-28 February meeting of the CDC’s Advisory Committee On Immunization Practices, if political developments allow.
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The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.
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The February meeting of US CDC’s Advisory Committee on Immunization Practices was indefinitely delayed to accommodate public comment, which did not open as scheduled on 3 February.
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Pink Sheet editors and special guest Michael McCaughan of Prevision Policy discuss Robert F. Kennedy Jr.’s first act as HHS Secretary, former FDA Commissioner Scott Gottlieb’s suggestion that industry extend the existing user fee agreement rather than negotiate a new one, and President Trump’s policies impacting FDA advisory committee schedules and staffing.
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HHS communications restrictions are keeping the FDA from publicly scheduling expected advisory committee meetings. The lack of upcoming meetings is raising concern about missed user fee dates or lower-quality agency decision-making.