Marinus Pharmaceuticals Receives Positive CHMP Opinion for ZTALMY® (ganaxolone) for the Adjunctive Treatment of Seizures Associated With CDKL5 Deficiency Disorder
26 Maio 2023 - 9:10AM
Business Wire
The European Commission decision is expected
within 67 days of the CHMP opinion
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has adopted a positive opinion recommending the
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 CHMP’s positive opinion reinforces the safety and efficacy
profile of ZTALMY and is a testament to the tireless efforts and
dedication of our team and the CDD community,” said Scott
Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
“This recommendation brings us one step closer to addressing a
significant unmet need for CDD patients with treatment-resistant
seizures in Europe. We look forward to the European Commission’s
decision and working with our commercial partner, Orion
Corporation, to bring this important treatment option to more
patients who may benefit.”
The CHMP recommendation will now be reviewed by the European
Commission (EC), which has the authority to approve medicines for
the European Union (EU). A final decision by the EC is expected
within 67 days of receipt of the CHMP opinion and will be
applicable to all 27 EU member states plus Iceland, Norway and
Liechtenstein. If approved, ZTALMY will be the first treatment in
the EU indicated for the adjunctive treatment of seizures
associated with CDD and will be commercialized by Orion
Corporation.
Marinus’ application for the 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 Neurology1.
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 Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated
to the development of innovative therapeutics for seizure
disorders. The Company’s commercial product, ZTALMY® (ganaxolone)
oral suspension CV, has been approved by the U.S. FDA for the
treatment of seizures associated with CDKL5 deficiency disorder in
patients two years of age and older. The potential of ganaxolone is
also being studied in other rare seizure disorders, including in
Phase 3 trials in tuberous sclerosis complex and refractory status
epilepticus. Ganaxolone is a neuroactive steroid GABAA receptor
modulator that acts on a well-characterized target in the brain
known to have anti-seizure effects. It is being developed in IV and
oral formulations to maximize therapeutic reach for adult and
pediatric patients in acute and chronic care settings. For more
information 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 expectations regarding approval
or marketing authorization by the EC 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, including the
timing thereof; ganaxolone’s potential across a range of seizure
disorders, our commercial strategy for ganaxolone, our 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, the risk that the EC does not grant marketing
authorization to ZTALMY on the timeline that we expect, or at all;
unexpected actions by the EMA, FDA or other regulatory agencies
with respect to our products; changes in regulatory policy
impacting orphan drug designations; 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 regulatory
authorities’ requirements 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 regulatory authorities 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; 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/20230525005802/en/
Sasha Damouni Ellis Senior Vice President, Corporate Affairs
& Investor Relations Marinus Pharmaceuticals, Inc.
sdamouni@marinuspharma.com
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