Stealth Biotherapeutics

FDA refusal of Stealth Bio drug shows challenges of ultra-rare disease studies

The FDA refused to review Stealth BioTherapeutics’ Barth syndrome drug, telling the company results in a study of just eight patients are insufficient to support its submission. The impasse highlights the challenges of testing drugs for ultra-rare diseases. Barth is so rare that Stealth is unsure it can recruit patients to run a new study.

Stealth casts a wide net with experimental treatment for mitochondrial diseases

Stealth is moving towards Phase 3 trials in primary mitochondrial myopathy – a common denominator in many mitochondrial diseases – with an emphasis on improving patients' quality of life.