Strongbridge Biopharma plc Launches the Uncovering Periodic Paralysis Genetic Testing Program
September 25 2017 - 7:30AM
Strongbridge Biopharma plc, (Nasdaq:SBBP), a global
commercial-stage biopharmaceutical company focused on the
development and commercialization of therapies for rare diseases
with significant unmet needs, today announced the launch of a
genetic testing program for hyperkalemic or hypokalemic periodic
paralysis. Primary Periodic Paralysis is a rare inherited disorder
characterized by recurrent, progressive, and debilitating episodes
of muscle weakness and temporary paralysis that affects
approximately 4,000 to 5,000 diagnosed individuals in the United
States.
“We are pleased to launch the Uncovering
Periodic Paralysis genetic testing program, which builds upon our
current suite of patient and physician services already being
offered through the Strongbridge CareConnection Program,” said
Matthew Pauls, president and chief executive officer of
Strongbridge Biopharma. “Primary Periodic Paralysis patients have
historically been underserved, and we are proud to offer them
robust support from Strongbridge’s patient- and physician-focused
programs. Additionally, given the early market interest in KEVEYIS®
(dichlorphenamide), the first and only FDA-approved treatment for
hyperkalemic, hypokalemic, and related variants of primary periodic
paralysis, and to better serve the needs of patients and the
physicians who treat them, we are expanding our 12-person field
force team to 21 employees by early November.”
Uncovering Periodic Paralysis is a no-cost
program that offers genetic testing for those who qualify. Although
genetic testing can confirm a suspected diagnosis, the absence of a
genetic alteration does not preclude diagnosis of this disease. The
Periodic Paralysis Panel includes testing of the three genes that
are most commonly-associated with periodic paralysis: SCN4A,
CACNA1S, and KCNJ2.
“Strongbridge’s genetic testing program will
provide an early diagnostic testing measure for Primary Periodic
Paralysis, a serious neuromuscular disease that sometimes takes up
to 20 years to accurately diagnose,” said Deborah Cavel-Greant,
president, Periodic Paralysis International. “The availability of
this test may help relieve patients and their families of a
significant emotional burden and pave the way for more timely and
effective treatment.”
“There is a growing awareness of periodic
paralysis and access to tests such as this may help shorten the
diagnostic journey for patients,” said Steve Cannon M.D., PhD,
neurologist and professor of Physiology at UCLA. “Access to
more reliable genetic testing will empower medical professionals
with the right information to facilitate improved clinical
management of the disease.”
Periodic Paralysis Panel
EligibilityAn individual must:
- Be at least 18 years old
- Have experienced more than one occurrence of episodic muscle
weakness/paralysis attacks, or episodic pain after attacks
- Episodes are provoked by at least one of the common triggers
for hyperkalemic or hypokalemic periodic paralysis, e.g. potassium,
carbohydrates, rest after exercise, cold exposure, or stress
Family Variant Testing For any
patient that is newly diagnosed via Uncovering Periodic Paralysis,
Strongbridge will offer no-cost Family Variant Testing to any
first-degree relative. Requirements include:
- A pathogenic or likely pathogenic variant was found on the gene
panel test
- Order must occur within 90-days of new diagnosis
For more information about the program, please
visit: UncoveringPeriodicParalysis.com.
About Strongbridge Biopharma
Strongbridge Biopharma is a
commercial-stage global biopharmaceutical company focused on the
development and commercialization of therapies for rare diseases
with significant unmet needs. Strongbridge's first commercial
product is KEVEYIS® (dichlorphenamide), the first and
only FDA-approved treatment for hyperkalemic, hypokalemic, and
related variants of Primary Periodic Paralysis. KEVEYIS has orphan
drug exclusivity status in the U.S. through August 7, 2022. In
addition to establishing this neuromuscular disease franchise, the
Company has a clinical-stage pipeline of therapies for rare
endocrine diseases. Strongbridge's lead compounds include
RECORLEV™ (levoketoconazole), a cortisol
synthesis inhibitor currently being studied for the treatment of
endogenous Cushing's syndrome, and veldoreotide, a next-generation
somatostatin analog being investigated for the treatment of
acromegaly, with potential additional applications in Cushing's
syndrome and neuroendocrine tumors. Both RECORLEV and veldoreotide
have received orphan designation from the U.S. Food and Drug
Administration and the European Medicines Agency. For more
information, visit www.strongbridgebio.com.
About KEVEYIS
KEVEYIS® (dichlorphenamide) is indicated
for the treatment of primary hyperkalemic periodic paralysis,
primary hypokalemic periodic paralysis, and related variants. In
clinical studies, the most common side effects of KEVEYIS were a
numbness or tingling, difficulty thinking and paying attention,
changes in taste, and confusion. These are not all of the possible
side effects that you may experience with KEVEYIS. Talk to your
doctor if you have any symptoms that bother you or do not go away.
You are encouraged to report side effects to Strongbridge
Biopharma at 1-855-324-8912, or to the FDA at
1-800-FDA-1088 or visit www.fda.gov/medwatch/. For additional
KEVEYIS important safety information and the full prescribing
information visit www.keveyis.com.
Forward-Looking StatementsThis
press release contains forward-looking statements that involve
substantial risks and uncertainties. All statements, other than
statements of historical facts, contained in this press release,
are forward-looking statements. These statements relate to future
events and involve known and unknown risks, including, without
limitation, uncertainties regarding Strongbridge's strategy, plans,
size of patient population and objectives of management for future
operations. The words "anticipate," "estimate," "expect," "intend,"
"may," "plan," "potential," "project," "target," "will," "would,"
or the negative of these terms or other similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. These
forward-looking statements are based on current expectations,
estimates, forecasts and projections and are not guarantees of
future performance or development and involve known and unknown
risks, uncertainties and other factors. The forward-looking
statements contained in this press release are made as of the date
of this press release, and Strongbridge Biopharma does
not assume any obligation to update any forward-looking statements
except as required by applicable law.
Contacts:
Corporate and Media Relations Elixir Health
Public Relations Lindsay Rocco +1 862-596-1304
lrocco@elixirhealthpr.com
Investor RelationsU.S.:The Trout Group Marcy
Nanus +1 646-378-2927 mnanus@troutgroup.com
Europe:First HouseMitra Hagen Negård+47 21 04
62 19strongbridgebio@firsthouse.no
USA 900 Northbrook Drive Suite 200 Trevose,
PA 19053 Tel. +1 610-254-9200 Fax. +1 215-355-7389
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