Novelion Therapeutics Announces Acceptance of Marketing Authorization Application for Metreleptin by European Medicines Agen...
January 23 2017 - 8:45AM
Novelion Therapeutics Inc. (NASDAQ:NVLN)
(TSX:NVLN), a biopharmaceutical company dedicated to developing new
standards of care for individuals living with rare diseases, today
announced that the European Medicines Agency (EMA) has accepted for
review the Marketing Authorization Application (MAA) for
metreleptin as replacement therapy to treat complications of leptin
deficiency in patients with congenital or acquired generalized
lipodystrophy (GL) and in a subset of patients with partial
lipodystrophy (PL). The MAA was filed in December 2016. An opinion
from the Committee for Medicinal Products for Human Use (CHMP) is
expected in December of 2017, and decision is expected in the first
quarter of 2018.
“The MAA filing acceptance marks important
progress in our priority initiative to deliver MYALEPTA® to adult
and pediatric GL and a subset of PL patients in Europe,” said John
Orloff, M.D., executive vice president, head of research and
development for Novelion Therapeutics. “There are more than 100
patients on therapy via a pre-approval compassionate use program in
certain markets in Europe, the majority of whom are within the
proposed indications. Additionally, there are a meaningful number
of patients who currently have limited options for treating
complications of these rare forms of lipodystrophy. We look forward
to working with European regulatory authorities with the objective
of achieving marketing authorization and bringing this needed
therapy to patients.”
Metreleptin was granted orphan drug designation
by the European Commission in July 2012, and, if approved, will
carry 10 years of orphan drug exclusivity in the EU. The company
estimates the prevalence of GL is approximately one in one million
people, and the prevalence of the subset of PL being targeted for
approval is approximately one half to one in one million people.
The company, through a subsidiary, will seek to market metreleptin
in the EU, if approved, under the tradename MYALEPTA.
U.S. INDICATIONS AND IMPORTANT SAFETY
INFORMATION
MYALEPT® (metreleptin) for injection is a
leptin analog indicated as an adjunct to diet as replacement
therapy to treat the complications of leptin deficiency in patients
with congenital or acquired generalized lipodystrophy. LIMITATIONS
OF USE: The safety and effectiveness of MYALEPT for the
treatment of complications of partial lipodystrophy or for the
treatment of liver disease, including nonalcoholic steatohepatitis
(NASH), have not been established.
Anti-metreleptin antibodies with neutralizing
activity have been identified in patients treated with MYALEPT.
T-cell lymphoma has been reported in patients with acquired
generalized lipodystrophy, both treated and not treated with
MYALEPT. For more detailed information, please see
additional Important Safety Information and
the Prescribing Information for MYALEPT.
Forward Looking
Statements:Certain statements in this press release
constitute “forward-looking statements” and constitute
“forward-looking information” within the meaning of applicable
securities laws, including statements regarding the approval,
orphan drug exclusivity and the market potential of metreleptin in
the EU, and the prevalence of the proposed metreleptin indications.
Forward-looking statements are based on estimates and assumptions
made by Novelion in light of current conditions and expected future
developments, as well as other factors that Novelion believes are
appropriate in the circumstances, including but not limited to, our
financial position and execution of our business strategy, receipt
of regulatory approvals, indication prevalence, and product
competition, market acceptance, sales, pricing, reimbursement and
side effects. These forward-looking statements are neither promises
nor guarantees of future performance, and are subject to a variety
of risks and uncertainties, many of which are beyond our control,
which could cause actual results to differ materially from those
contemplated in these forward-looking statements. Many such
risks, uncertainties and other factors are taken into account as
part of our assumptions underlying these forward-looking statements
and include, among others, the following: the risks that
metreleptin may not be approved by the EMA at all or for the
desired indications; that, if approved, metreleptin may not obtain
reimbursement approvals on a timely basis or at all or at the
levels necessary to support commercialization; that the prevalence
estimates for GL and a subset of PL may be inaccurate; the risk
that orphan drug, data and marketing exclusivity or the patent
protection for metreleptin may not be sufficient or may be
invalidated; and the other risks common to seeking regulatory
approval for, developing and commercializing orphan
drugs.
For additional disclosure regarding these and
other risks we face, see the disclosure contained in the "Risk
Factors" section of Aegerion's Quarterly Report on Form 10-Q filed
on November 4, 2016, Novelion’s Annual Report on Form 10-K filed on
February 25, 2016 (and amended on April 29, 2016) and Quarterly
Report on Form 10-Q filed on November 1, 2016 and each company’s
other public filings with the SEC, available on the SEC's website
at www.sec.gov. Except as required by law, we undertake no
obligation to update or revise the information contained in this
press release, whether as a result of new information, future
events or circumstances or otherwise.
Investors and others should note that we
communicate with our investors and the public using our company
website www.novelion.com, including, but not limited to, company
disclosures, investor presentations and FAQs, SEC filings, press
releases, public conference calls transcripts and webcast
transcripts. The information that we post on these websites could
be deemed to be material information. As a result, we encourage
investors, the media and others interested to review the
information that we post there on a regular basis. The contents of
our website shall not be deemed incorporated by reference in any
filing under the Securities Act of 1933, as amended.
CONTACT:
Amanda Murphy, Director, Investor Relations & Corporate Communications
Novelion Therapeutics
857-242-5024
amanda.murphy@novelion.com
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