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The for the treatment of neurogenic orthostatic hypotension, a rare disease that results in a drop in blood pressure when patients try to stand up.
The drug was passed up for approval 2 years ago when the FDA .
Approval stems from two clinical trials, each 2 weeks long, in which patients on droxidopa reported less dizziness, lightheadedness, feeling faint, or feeling as if they might black out than those on placebo.
The FDA noted, however, that the drug was OK'd under its accelerated approval program, which allows it to go on-market for demonstrating an effect on an "intermediate clinical measure" -- in this case, short-term relief of dizziness.
Drugmaker Chelsea Therapeutics, based in Charlotte, N.C., will have to demonstrate the drug's long-term benefit of extended dizziness relief in post-approval trials.
In its press release, the FDA emphasized that the "durability of the improvement in patient symptoms beyond 2 weeks has not been demonstrated."
Droxidopa will come with a boxed warning on the risk of increased blood pressure while lying down, which can cause stroke. FDA also warned it's essential that clinicians remind patients to sleep with their head and upper body elevated, and that supine blood pressure needs to be monitored before and during treatment, and more frequently when increasing doses. The drug is available in 100 mg, 200 mg, and 300 mg capsules.
In the two trials, the most common adverse events were headache, dizziness, nausea, high blood pressure, and fatigue.
Last month an FDA panel voted overwhelmingly in favor of approval (16 to 1) for droxidopa, but 2 years earlier nearly that same panel had been split on approval.
Following that advisory committee meeting, the FDA did not approve the drug, telling Chelsea Therapeutics it
Many panel members voting in favor of approval at the most recent advisory committee meeting said FDA should limit the drug to short-term use only, given the lack of long-term data.
Droxidopa is a pro-drug for norepinephrine, converted both peripherally and centrally, as it crosses the blood-brain barrier. It acts as a vasoconstrictor which, in theory, should help patients retain adequate blood pressure when they stand up from sitting or supine positions.