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Mitochondrial Disease Workshop Shows US FDA Commitment To First Approvals

 
• By Kate Rawson

First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.

Mitochondria (red) are organelles found in most cells. They generate a cell's chemical energy.
Mitochondrial disorders include Barth syndrome, mitochondrial cardiomyopathies, Leber’s hereditary optic neuropathy, Friedreich’s ataxia, Alpers syndrome and Leigh syndrome. • Source: NIH National Institute of Child Health and Human Development

A US Food and Drug Administration scientific workshop on mitochondrial diseases signaled the agency’s continued interest in helping drug developers get to the first approvals in disorders like Barth syndrome and Friedreich’s ataxia, but also underscored the continued challenges in designing clinical trials in such a genetically heterogeneous group of diseases.

In one sign of the agency’s willingness to encourage mitochondrial disorder drug development, FDA Office of Pediatric Therapeutics pediatric ethicist...

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