Tag

Compassionate Use

All articles tagged with #compassionate use

FDA opens compassionate path to investigational pancreatic cancer drug
health25 days ago

FDA opens compassionate path to investigational pancreatic cancer drug

The FDA has approved an expanded-access protocol for the investigational pancreatic cancer drug daraxonrasib, allowing eligible patients with metastatic pancreatic ductal adenocarcinoma to access it before full approval. In clinical data, patients taking daraxonrasib showed a median survival of about 13 months versus six months with chemotherapy, and supporters like former Sen. Ben Sasse have reported improved pain and tumor shrinkage. The 300 mg daily pill works by blocking a signal that drives cancer growth, and the FDA’s April 30 protocol approval aims to give seriously ill patients earlier access while trials continue.

Small Biotech Contemplates Abandoning Ultra-Rare Disease Drug Due to FDA Frustration
healthcare2 years ago

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.