The FDA has approved UCB’s Kygevvi for treating children and adults with thymidine kinase 2 deficiency, an inherited mitochondrial disorder. It’s the drugmaker’s first ultra-rare disease product.
A Phase 2b-ready Cyclerion Therapeutics drug candidate for a rare mitochondrial disease gets new life under a new startup led by a familiar face. Tisento Therapeutics acquired the Cyclerion drug and is backed by a Series A round financing.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
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 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.