Marinus Pharmaceuticals Announces Ganaxolone Approved in China as First Treatment for Seizures Associated with CDKL5 Deficiency Disorder
18 Juillet 2024 - 1:00PM
Business Wire
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced that the China National
Medical Products Administration (NMPA) has approved ganaxolone oral
suspension for the treatment of epileptic seizures in patients two
years of age and older with CDKL5 deficiency disorder (CDD).
In November 2022, Marinus and Tenacia Biotechnology entered into
a collaboration agreement which granted Tenacia the right to
develop and commercialize ganaxolone in Mainland China, Hong Kong,
Macau and Taiwan in exchange for royalties and other payments
associated with net sales and commercial and regulatory
milestones.
“CDD is a severe and rapidly progressive neurodevelopmental
disorder and before ganaxolone, there had been no treatments
approved for this condition in China,” said Dr. Xiaoxiang Chen,
Chief Executive Officer of Tenacia Biotechnology. “There is an
urgent need to bring innovative new therapies to patients and
families living with CDD, particularly as diagnosis rates continue
to rise. We look forward to delivering the first and only treatment
option for these patients in China.”
The approval is supported by data from Marinus’ Phase 3 Marigold
trial in CDD, a double-blind placebo-controlled trial in 101
patients. Patients treated with ganaxolone showed a 30.7% median
reduction in 28-day major motor seizure frequency, compared to a
6.9% reduction for those receiving placebo, achieving the trial’s
primary endpoint (p=0.0036). Patients in the open-label extension
study treated with ganaxolone for at least 12 months (n=48)
experienced a median 49.6% reduction in major motor seizure
frequency. In the Marigold trial, ganaxolone was generally
well-tolerated and showed a safety profile consistent with previous
clinical trials, with the most frequent adverse event being
somnolence.
“CDD brings unpredictability and increased healthcare needs and
costs to patients and their families, and can significantly impact
their quality of life,” said Kimberly McCormick, PharmD., Chief
Regulatory and Quality Assurance Officer of Marinus. “The approval
in China represents an important step forward for patients living
with CDD and underscores our commitment to bringing innovative
medicines to people living with rare genetic epilepsies across the
globe. We’re grateful for the collaboration and support of Tenacia
and regulatory authorities in China to bring this important new
treatment option to patients with CDD.”
Ganaxolone has received regulatory approval in the U.S.,
European Union and China for appropriate patients with CDD. Under
its collaboration agreements, Marinus supplies ganaxolone for all
global markets.
About ZTALMY® (ganaxolone) oral suspension 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. It is a prescription medicine that has been
approved by the U.S. Food and Drug Administration and the European
Commission for appropriate patients with CDKL5 deficiency
disorder.
U.S. Prescribing Information for ZTALMY® (ganaxolone)
oral suspension CV.
European Union Summary of Product Characteristics for
ZTALMY.
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. For
more information about Marinus visit www.marinuspharma.com.
About Tenacia Biotechnology Based in China and a
portfolio company of Bain Capital, Tenacia is a clinical-stage
biopharmaceutical company focused on developing and commercializing
innovative treatments to address unmet medical needs of nervous
system disorders. Tenacia is led by industry leaders with deep
experience in life sciences and expertise in CNS, global and
China-specific drug development and commercialization and
demonstrated success in company formation. The company is advancing
a portfolio of innovative, best-in-class therapies.
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, statements regarding our clinical development and
commercialization plans and the timing thereof; our expectations
regarding our collaboration with Tenacia and Tenacia’s
commercialization plans and the timing thereof; and other
statements regarding our future operations, financial performance,
financial position, prospects, objectives and other future
events.
Forward-looking statements in this press release involve
substantial risks and uncertainties that could cause our clinical
development and commercialization 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 results or
delays in the commercialization of ZTALMY; unexpected market
acceptance, payor coverage or future prescriptions and revenue
generated by ZTALMY; unfavorable pricing regulations, third-party
reimbursement practices or healthcare reform initiatives;
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 varying interpretation of clinical data; 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; the potential that
regulatory authorities, including the FDA and EMA, may not grant or
may delay approval for our product candidates; early clinical
trials may not be indicative of the results in later clinical
trials; 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; our ability to obtain
and maintain regulatory approval for our product candidate; our
ability to develop new formulations of ganaxolone or prodrugs; our
ability to obtain, maintain, protect and defend intellectual
property for our product candidates; the potential negative impact
of third party patents on our or our collaborators’ ability to
commercialize ganaxolone; delays, interruptions or failures in the
manufacture and supply of our product candidate; the size and
growth potential of the markets for our product candidates, and our
ability to service those markets; our ability to continue as a
going concern; our cash and cash equivalents may not be sufficient
to support our operating plan for as long as anticipated; our
expectations, projections and estimates regarding expenses, future
revenue, capital requirements, and the availability of and the need
for additional financing; our ability to obtain additional funding
to support our clinical development and commercial programs; the
potential for our ex-U.S. partners to breach their obligations
under their respective agreements with us or terminate such
agreements in accordance with their respective terms; the risk that
drug product quality requirements may not support continued
clinical investigation of our product candidates and result in
delays or termination of such clinical studies and product
approvals; and the availability or potential availability of
alternative products or treatments for conditions targeted by us
that could affect the availability or commercial potential of our
product candidate. This list is not exhaustive and these and other
risks are described in our periodic reports, including the annual
report 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240718265934/en/
Investors Sonya Weigle SVP, IR, HR
& Corporate Affairs Marinus Pharmaceuticals, Inc.
sweigle@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
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