There could be trouble ahead for Italian drug developer Recordati. FDA staff reviewers have some questions about Carbaglu, the company's treatment for patients with NAGS deficiency. NAGS, which is found in about 1 in 30,000 births, is a disorder that causes excess ammonia to gather in the blood. The deficiency can result in a serious neonatal disorder that can be fatal if not detected immediately. Carbaglu seeks to replace the specific chemical compound that is missing in patients with the disorder.
The FDA pointed out that problems with the study design could have interfered with the company's findings. "Clear conclusions about the effectiveness of carglumic acid cannot be made because of the severely limited quantity and quality of the data," the FDA says in its documents, as reported by Reuters. At the advisory panel on Wednesday, experts will decide whether Recordati has submitted enough strong evidence that the drug is effective. The drug is made by Recordati's Orphan Europe unit, a rare-disease company that it acquired in 2007.
- check out the Reuters report