A panel of federal experts recommended approval for Chelsea Therapeutics’ experimental blood-pressure drug Northera, which is designed to prevent dizziness and fainting in Parkinson’s disease patients.
The Food and Drug Administration’s panel of outside experts voted 16-1 on Tuesday in favor of approving the drug, despite concerns from the FDA about a lack of evidence regarding its long-term effects. Some panelists said their endorsement came with the condition that the drug only be approved for short-term use and in a narrowly defined group of patients. The experts stressed that the drug should not be used generally to treat any patient reporting dizziness.
The FDA is not required to follow the group’s advice, though it often does.
The recommendation is an unexpected win for Chelsea Therapeutics Ltd., and its shares jumped 10.9 percent to $2.55 in after-hours trading. The stock is down 48 percent in January, but it has still more than doubled over the past 12 months.
The FDA’s own scientists had raised major questions about the drug’s effectiveness in documents posted ahead of the meeting. Chelsea shares fell nearly 30 percent Friday on the agency’s negative review. While the drug appears to have a strong short-term effect, the FDA said there is no data showing that the drug’s benefits last beyond one week. FDA staffers also pointed to several issues that raise questions about the reliability of the company’s studies, including a high drop-out rate among patients taking the drug.
Despite those shortcomings, more than a dozen patients with Parkinson’s and similar movement disorders spoke before the panel during a public-comment period Tuesday, urging regulators to approve the drug.
Northera is designed to treat neurogenic orthostatic hypotension, which is a sudden drop in blood pressure that occurs after standing. It’s a common symptom of Parkinson’s disease that can cause dizziness, fainting and fall-related injuries. The FDA is scheduled to make an approval decision on the drug in February.
The FDA previously rejected the drug in March 2012, asking the company for an additional study to demonstrate Northera’s effectiveness. Charlotte, N.C.-based Chelsea Therapeutics International Ltd. has been seeking approval for the drug from the FDA since 2011.
Northera, known chemically as droxidopa, is Chelsea’s most advanced drug candidate. The company has no drugs approved for sale.