FDA grants accelerated approval to NORTHERA (droxidopa) for patients with symptomatic NOH

Chelsea Therapeutics International, Ltd. (Nasdaq: CHTP) today announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval of NORTHERATM (droxidopa) for the treatment of symptomatic neurogenic orthostatic hypotension (NOH). NORTHERA is the first and only therapy approved by the FDA which demonstrates symptomatic benefit in patients with NOH.

NORTHERA is indicated for the treatment of orthostatic dizziness, lightheadedness, or the "feeling that you are about to black out" in adult patients with symptomatic NOH caused by primary autonomic failure (Parkinson's disease, multiple system atrophy and pure autonomic failure), dopamine beta hydroxylase deficiency and non-diabetic autonomic neuropathy.

"The approval of NORTHERA is an extraordinary achievement, one for which I thank patients and their families, investigators and the medical community, our partner Dainippon Sumitomo, and our employees," said Joseph G. Oliveto, President and Chief Executive Officer of Chelsea Therapeutics. "We will now turn our efforts towards delivering NORTHERA to patients with NOH in the United States, an important goal we expect to achieve in the second half of the year."

Symptomatic NOH is an autonomic nervous system disorder caused by failure to produce and or release adequate amounts of norepinephrine upon standing. Norepinephrine deficiency results in an inability for a person to maintain adequate blood pressure and blood flow to the brain when upright, frequently resulting in dizziness, lightheadedness, blurred vision, fatigue, poor concentration and fainting episodes. These symptoms often severely limit a person's ability to perform routine daily activities that require standing or walking for both short or long periods of time. 1,2 The disorder affects an estimated 80,000 to 150,000 individuals in the United States.

"Symptomatic NOH is a commonly debilitating disorder with limited treatment options and no new therapeutic choices introduced in nearly two decades," commented Dr. Robert A. Hauser, Professor of Neurology, Molecular Pharmacology, and Physiology, and Director of the Parkinson's Disease Movement Disorders Center, University of South Florida. "NORTHERA has a distinct mechanism of action affecting the root cause of NOH and represents an important new treatment choice for patients. It is the first and only therapy approved to address specific underlying symptoms of NOH, namely orthostatic dizziness, lightheadedness, or feeling like you might black out. I look forward to offering this new and novel treatment approach to patients who may benefit from it."

"This approval is a significant step forward for people with MSA forced to cope with the debilitating effects of NOH," said Judy Biedenharn, Co-President of the Multiple Systems Atrophy (MSA) Coalition. "Addressing the major symptoms of NOH can have an impact on a person's ability to perform even the simplest of daily activities -- like taking a shower or standing to brush one's teeth -- offering an important benefit to both individuals and their caregivers."

The NORTHERA approval was granted under the FDA's accelerated approval program, which allows for conditional approval of a medicine that fills a serious unmet medical need, provided additional confirmatory studies are conducted. The package insert indicates that effectiveness beyond 2 weeks of treatment has not yet been demonstrated, therefore the continued effectiveness of NORTHERA in patients should be assessed periodically. A multi-center, placebo-controlled, randomized study, which includes a 4 week randomized withdrawal phase preceded by a three month open label run-in phase, designed with the goal of definitively establishing the durability of the clinical benefits of NORTHERA, has been preliminarily agreed to with the FDA. Based on the contemplated study design, the trial would include approximately 1,400 patients, which the FDA has agreed may be enrolled over a six year period.

Source:

Chelsea Therapeutics International, Ltd.

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