FDA Questions Drug Study

FDA Staff Question Study on Blood Drug

Mon Jan 11, 2010 - US FDA questions Actelion rare disease drug data FDA questions effectiveness, see no big safety issue WASHINGTON, Jan 11 (Reuters) - U.S. health regulatorsquestioned a study by Recordati SpA (RECI.MI), casting doubt onthe ability of the company's proposed drug to treat elevatedlevels of ammonia in the blood. The Italian pharmaceutical company is seeking to market thetreatment, called Carbaglu, in patients with N acetyl glutamatesynthetase, or NAGS, deficiency, an inherited disorder thatcauses ammonia to accumulate in the blood. U.S. Food and Drug Administration staff reviewers pointedto a number of problems with the drug's study design, includinga lack of controls and potential bias, although they added thatno serious safety issues were found in patients overall. "Clear conclusions about the effectiveness of carglumicacid cannot be made because of the severely limited quantityand quality of the data," FDA staff wrote in the documentsreleased on Monday. The agency released its staff review ahead of a publicadvisory meeting on Wednesday to discuss whether the FDA shouldapprove the product made by Recordati's Orphan Europe unit. Carbaglu contains carglumic acid and aims to work in a moretargeted manner than other available drugs by replacing aspecific chemical compound that NAGS patients lack. The condition affects roughly one per 30,000 live births,according to the company. In a separate document also released on Monday, OrphanEurope said the data offered "significant proof" of its drug'seffectiveness. While other medicines exists, they can be risky, OrphanEurope said, adding that hemodialysis, liver transplants anddiet changes can also be used. Carbaglu's safety profile was"highly acceptable" and would not require any specificfollow-up to monitor risks, the company wrote. The FDA plans to seek advice from its panel of outsideadvisors over the company's studies. The main issue is "whether the quality and quantity of datasubmitted constitutes sufficient evidence" to conclude "aclinical meaningful effect of Carbaglu," the FDA wrote. Shares of Recordati earlier closed up less than 1 percenton the Milan stock exchange.

(Reporting by Susan Heavey; Editing by Lisa Von Ahn and DerekCaney)