Chelsea Therapeutics Announces FDA Accelerated Approval of NORTHERA™ (Droxidopa) for the Treatment of Symptomatic NOH

  Chelsea Therapeutics Announces FDA Accelerated Approval of NORTHERA™
  (Droxidopa) for the Treatment of Symptomatic NOH

     First New Treatment Option for Symptomatic NOH in Nearly Two Decades

  First and Only FDA Approved Therapy to Demonstrate Symptomatic Benefit in
                              Patients with NOH

Business Wire

CHARLOTTE, N.C. -- February 18, 2014

Chelsea Therapeutics International, Ltd. (Nasdaq: CHTP) today announced that
the U.S. Food and Drug Administration (FDA) granted accelerated approval of
NORTHERA^TM (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(TM) (droxidopa) is available in 100mg, 200mg and 300mg capsules.
(Photo: Business Wire)

NORTHERA(TM) (droxidopa) is available in 100mg, 200mg and 300mg capsules.
(Photo: Business Wire)

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.

Please see NORTHERA full Prescribing Information including Most Serious Side
Effects for additional Important Safety Information at
http://www.chelseatherapeutics.com.

For product information, or to report SUSPECTED ADVERSE REACTIONS, contact
Chelsea Therapeutics, Inc. at 1-855-351-2879 or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.

References: 1. Freeman R, Wieling W, Axelrod FB, et al. Consensus statement on
the definition of orthostatic hypotension, neurally mediated syncope and the
postural tachycardia syndrome. Clin Auton Res 2011;21:69-72. 2. Freeman R.
Clinical practice. Neurogenic orthostatic hypotension. N Engl J Med
2008;358:615-624. 3. Goldstein DS, Holmes C, Kaufmann H, Freeman R. Clinical
pharmacokinetics of the norepinephrine precursor L-threo-DOPS in primary
chronic autonomic failure. Clin Auton Res 2004;14:363-368.

About NORTHERA

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. Effectiveness
beyond 2 weeks of treatment has not been demonstrated. The continued
effectiveness of NORTHERA should be assessed periodically.

Droxidopa was initially developed by Dainippon Sumitomo Pharma Co., Ltd. (DSP)
and first commercialized in Japan in 1989.

About Chelsea Therapeutics

Chelsea Therapeutics (Nasdaq:CHTP) is a biopharmaceutical development company
that acquires, develops and commercializes innovative products for the
treatment of a variety of human diseases, including central nervous system
disorders. Chelsea acquired global development and commercialization rights to
droxidopa (L-DOPS), or NORTHERA, from Dainippon Sumitomo Pharma Co., Ltd. in
2006, excluding Japan, Korea, China and Taiwan. For more information about the
Company, visit www.chelseatherapeutics.com.

This press release contains forward-looking statements regarding future events
including our intention to pursue the development of NORTHERA . These
statements are subject to risks and uncertainties that could cause the actual
events or results to differ materially. These include the risk that we are
unable to successfully commercialize NORTHERA; the risks associated with
fulfilling sales, marketing and distribution requirements; reliance on key
personnel and our ability to attract and/or retain key personnel; the risk
that FDA will not agree that our clinical trial results demonstrate the safety
and effectiveness of droxidopa or fulfill the post marketing requirements to
achieve full approval; the risk that our resources will not be sufficient to
conduct any study of NORTHERA that will be acceptable to the FDA; the risk
that we cannot complete Study 401 or any other additional study for NORTHERA
without the need for additional capital; the risks and costs of drug
development and that such development may take longer or be more expensive
than anticipated; our need to raise additional operating capital in the
future; our reliance on our lead drug candidate droxidopa; the risk that we
will not be able to obtain regulatory approvals of droxidopa or our other drug
candidates for additional indications; the risk of volatility in our stock
price, related litigation, and analyst coverage of our stock; reliance on
collaborations and licenses; intellectual property risks; our history of
losses; competition; market acceptance for our products when approved for
marketing.

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