US FDA advisory panel back approval of Recordati's Carbaglu and new use for Actelion's Zavesca

A US Food and Drug Administration advisory panel has unanimously recommended the approval of Italian drugmaker Recordati SpA's drug for a rare blood condition, causing the firm's shares to rise 2% on the Milan Stock Exchange, while the same committee backed the use of Swiss firm Actelion's Gaucher disease drug for a new indication.

The agency's Endocrinologic and Metabolic Drugs Advisory Committee voted 12-0 in support of Recordati's Carbaglu (carglumic acid) for the treatment of hyperammonemia due to an autosomal genetic disorder resulting in the deficiency of the liver enzyme N-acetylglutamate synthase (NAGS). A very rare disease, NAGS deficiency involves extremely high plasma levels of ammonia, which leads to permanent and irreversible damage of the central nervous system. The drug, which treats a lifelong and life-threatening clinical condition, is already marketed by Recordati in Europe.

"The potential usefulness of this drug is phenomenal," said panelist Karl Roth, a Nebraska-based researcher at MDS Pharma Services, quoted by Reuters. "This is one way we can salvage some of the children born with NAGS deficiency," he added.
However, earlier, some FDA staff scientists had been concerned that the company's data was unclear given issues with the studies' design. But panelists called the data not only sufficient but impressive in showing the drug targeted the ammonia problem, said Reuters. Some panelists acknowledged problems with the company's method of retrospectively analyzing data but said the results were persuasive given the difficulty of studying such a rare and deadly condition.