Small Biotech Contemplates Abandoning Ultra-Rare Disease Drug Due to FDA Frustration

A small biotech company may abandon its development of an experimental medicine for Barth syndrome, an ultra-rare disease, due to frustration with the FDA's approval process. If the drug fails to gain market approval, patients like Declan, a toddler who has benefited from the medicine, would lose access to it through a special access program called compassionate use.