Blueprint Medicines slips as it changes endpoint for trial in aim to greatly broaden Ayvakit label
Blueprint is forging ahead on its slow crawl to broaden its market for Ayvakit.
Having nabbed its fourth approval for the drug in 2021 for advanced systemic mastocytosis (SM) — a very rare disease in which a type of white blood cells known as mast cells accumulate in the body — Blueprint is targeting the non-advanced form of the disease, which impacts the vast majority of SM patients.
However, for the second part of its Phase II trial in non-advanced SM, Blueprint announced that it will be swapping its primary endpoint with a key secondary endpoint based on an FDA recommendation. The trial’s primary endpoint was originally the proportion of patients with a ≥30% decrease in total symptom score, or TSS, but the new primary endpoint will become the mean change in TSS.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.