ZTALMY is the first treatment approved by the
European Commission for seizures associated with CDKL5 deficiency
disorder in children and adolescents
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced that the European
Commission (EC) has granted approval of ZTALMY® (ganaxolone) oral
suspension for the adjunctive treatment of epileptic seizures
associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency
disorder (CDD) in patients two to 17 years of age. ZTALMY may be
continued in patients 18 years of age and older.
“The approval by the European Commission represents a
significant milestone for children, families and physicians who,
until now, have long been challenged by the impact of seizures and
lack of treatments available for CDD,” said Scott Braunstein, M.D.,
Chairman and Chief Executive Officer of Marinus. “This achievement
reflects our organization’s unwavering commitment to bring an
effective treatment option to individuals living with CDD and we
are grateful to all the stakeholders who made the approval
possible.”
The EC approval of ZTALMY in CDD is supported by data from the
Phase 3 Marigold double-blind placebo-controlled trial, in which
101 patients were randomized and individuals treated with ZTALMY
showed a median 30.7% reduction in 28-day major motor seizure
frequency, compared to a median 6.9% reduction for those receiving
placebo, achieving the trial’s primary endpoint (p=0.0036). In the
Marigold open label extension study, patients treated with ZTALMY
for at least 12 months (n=48) experienced a median 49.6% reduction
in major motor seizure frequency. In the clinical development
program, ZTALMY demonstrated efficacy, safety and tolerability with
the most common adverse reactions (incidence ≥5% and at least twice
the rate of placebo) in the ZTALMY group being somnolence, pyrexia,
salivary hypersecretion and seasonal allergy. In May 2022, the
results from the Marigold study were published in The Lancet
Neurology.1
The approval follows a positive opinion issued in May 2023 by
the Committee for Medicinal Products for Human Use of the European
Medicines Agency and is applicable to all 27 European Union member
states plus Iceland, Norway and Liechtenstein. In July 2021,
Marinus and Orion Corporation entered into a collaboration
agreement which grants Orion the right to commercialize ZTALMY in
Europe.
About CDKL5 Deficiency Disorder
CDKL5 deficiency disorder (CDD) is a serious and rare genetic
disorder characterized by early‑onset, difficult‑to‑control
seizures and severe neuro‑developmental impairment.2 It is caused
by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene,
located on the X chromosome. The CDKL5 gene produces a protein that
is important for normal brain development and function.3
About ZTALMY®
ZTALMY (ganaxolone) is a neuroactive steroid GABAA receptor
modulator that acts on a well-characterized target in the brain
known to have anti-seizure effects. ZTALMY is an oral prescription
medicine approved by the European Commission for the adjunctive
treatment of epileptic seizures associated with CDKL5 deficiency
disorder in patients two to 17 years of age. ZTALMY may be
continued in patients 18 years of age and older.
Full European Summary of Product Characteristics for ZTALMY
will be available from the EMA website at
www.ema.europa.eu.
About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated
to the development of innovative therapeutics for seizure
disorders. The Company first introduced FDA-approved prescription
medication ZTALMY® (ganaxolone) oral suspension CV in the U.S. in
2022 and continues to invest in the potential of ganaxolone in IV
and oral formulations to maximize therapeutic reach for adult and
pediatric patients in acute and chronic care settings. For more
information about Marinus visit www.marinuspharma.com.
About Orion Corporation
Orion is a globally operating Finnish pharmaceutical company – a
builder of well-being. Orion develops, manufactures and markets
human and veterinary pharmaceuticals and active pharmaceutical
ingredients. The company has an extensive portfolio of proprietary
and generic medicines and self-care products. The core therapy
areas of its pharmaceutical R&D are oncology and pain.
Proprietary products developed by Orion are used to treat cancer,
neurological diseases and respiratory diseases, among others.
Orion's A and B shares are listed on Nasdaq Helsinki.
Forward-Looking Statements
To the extent that statements contained in this press release
are not descriptions of historical facts regarding Marinus, they
are forward-looking statements reflecting the current beliefs and
expectations of management made pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995.
Words such as "may", "will", "expect", "anticipate", "estimate",
"intend", "believe", and similar expressions (as well as other
words or expressions referencing future events, conditions or
circumstances) are intended to identify forward-looking statements.
Examples of forward-looking statements contained in this press
release include, among others, our commercial and clinical
strategy, development plans and timelines and other future
events.
Forward-looking statements in this press release involve
substantial risks and uncertainties that could cause our clinical
development programs, future results, performance or achievements
to differ significantly from those expressed or implied by the
forward-looking statements. Such risks and uncertainties include,
among others, unexpected market acceptance, payor coverage or
future prescriptions and revenue generated by ZTALMY; the pricing
and reimbursement process can be time consuming and may delay
commercialization of ZTALMY in one or more European countries; our
dependence on Orion to commercialize ZTALMY in Europe pursuant to
the exclusive collaboration agreement; unexpected actions by the
FDA or other regulatory agencies with respect to our products;
competitive conditions and unexpected adverse events or patient
outcomes from being treated with ZTALMY, uncertainties and delays
relating to the design, enrollment, completion, and results of
clinical trials; unanticipated costs and expenses; the company’s
cash and cash equivalents may not be sufficient to support its
operating plan for as long as anticipated; our ability to comply
with the FDA’s requirement for additional post-marketing studies in
the required time frames; the timing of regulatory filings for our
other product candidates; clinical trial results may not support
regulatory approval or further development in a specified
indication or at all; actions or advice of the FDA or EMA may
affect the design, initiation, timing, continuation and/or progress
of clinical trials or result in the need for additional clinical
trials; the size and growth potential of the markets for the
company’s product candidates, and the company’s ability to service
those markets; the company’s expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
and the availability of and the need for additional financing;
delays, interruptions or failures in the manufacture and supply of
our product candidates; the company’s ability to obtain additional
funding to support its clinical development and commercial
programs; the company’s ability to protect its intellectual
property; and the effect of the COVID-19 pandemic on our business,
the medical community, regulators and the global economy. This list
is not exhaustive and these and other risks are described in our
periodic reports, including our annual reports on Form 10-K,
quarterly reports on Form 10-Q and current reports on Form 8-K,
filed with or furnished to the Securities and Exchange Commission
and available at www.sec.gov. Any forward-looking statements that
we make in this press release speak only as of the date of this
press release. We assume no obligation to update forward-looking
statements whether as a result of new information, future events or
otherwise, after the date of this press release.
1 The Lancet Neurology, Volume 21, Issue 5, P417-427, May 01,
2022
2 Olson H et al. 2019 Pediatric Neurology
3 Jakimiec M et al. 2020 Brain Sci.
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version on businesswire.com: https://www.businesswire.com/news/home/20230731081674/en/
Company Molly Cameron Director, Corporate Communications
& Investor Relations Marinus Pharmaceuticals, Inc.
mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
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