FDA panel wants clinical endpoints for AAT

FDA's Blood Products Advisory Committee voted 17-0 to recommend that change in lung density measured by high resolution computer tomography (HRCT) could be used as a primary endpoint in pivotal studies of inhaled alpha 1-antitrypsin ( AAT, A1AT) augmentation therapies. For intravenous products, the panel voted unanimously to recommend that FDA focus on clinically meaningful endpoints, such as HRCT, FEV1, pulmonary exacerbations or mortality. FDA's Jay Epstein, director of the Office of Blood Research and Review, told the panel any decision about endpoints will not affect IV products already in development.

Marketed AAT products, as well as an IV product from Kamada Ltd. (Tel Aviv:KMDA), used serum AAT levels and/or other biochemical surrogate endpoints. It is not possible to use serum AAT levels to measure efficacy of inhaled products and, according to FDA's briefing documents, recent findings have "called into question the target serum levels previously used." ...