FDA panel OKs Recordati's Carbaglu

An FDA panel has unanimously backed the approval of Carbaglu, Recordati's treatment for patients with a rare and fatal disease called NAGS deficiency. The vote of confidence comes despite earlier concerns that the study design could have interfered with the company's findings. "The potential usefulness of this drug is phenomenal," said one panel member, quoted by Reuters. "This is one way we can salvage some of the children born with NAGS deficiency." The data, the panel said, were impressive given how difficult it is to study patients with the disease. Report