ZTALMY is approved by the U.S. Food and Drug
Administration and the European Commission for appropriate patients
with CDKL5 deficiency disorder
The program enables physicians to request
ZTALMY for eligible patients in geographies where the product is
not commercially available and as supported by local regulatory
requirements
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced the initiation of the
Marinus Access Program, a global managed access program for ZTALMY®
(ganaxolone) oral suspension CV. The new program enables physicians
to request ZTALMY for eligible patients with seizures associated
with CDKL5 deficiency disorder (CDD) in geographies where the
product is not commercially available and as supported by local
regulatory requirements. The Marinus Access Program will be managed
by Durbin, a leader in the international distribution of
specialized pharmaceuticals.
“Consistent with our company mission, we are committing
appropriate resources to help facilitate broader access to ZTALMY
for patients with critical unmet medical needs,” said Scott
Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
“With the launch of the Marinus Access Program and commercial
collaboration agreements in place in Europe, China and the MENA
region, we are proud to advance our global access strategy and
provide pathways to make ZTALMY available for appropriate CDD
patients in geographies where there are no approved treatment
options and local regulations allow.”
The U.S. Food and Drug Administration and European Commission
approvals of ZTALMY in CDD are 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
About the Marinus Access Program
The Marinus Access Program enables physicians to request ZTALMY®
(ganaxolone) oral suspension CV for eligible patients with seizures
associated with CDKL5 deficiency disorder in geographies where the
product is not commercially available and as supported by local
regulatory requirements. The program does not provide free of
charge access to ZTALMY and does not affect patients already taking
ZTALMY as part of a clinical trial. The Marinus Access Program is
managed by Durbin, a leader in the international distribution of
specialized pharmaceuticals.
Access requests in geographies where Marinus has a commercial
arrangement in place are managed by the local commercial
organization and are not eligible for the Marinus Access
Program:
- European Economic Area, United Kingdom, Switzerland: Orion
Corporation
- Algeria, Bahrain, Egypt, Iraq, Jordan, Kingdom of Saudi Arabia,
Kuwait, Lebanon, Libya, Morocco, Oman, Qatar, Tunisia, and United
Arab Emirates: Biologix FZco
- Mainland China, the Hong Kong Special Administrative Region,
Macau Special Administrative Region, and Taiwan: Tenacia
Biotechnology Co., Ltd.
Physicians can learn more about the Marinus Access Program for
ZTALMY by sending an email to MarinusMAP@durbinglobal.com.
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 ZTALMY®
ZTALMY (ganaxolone) oral suspension is a neuroactive steroid
GABAA receptor modulator that acts on a well-characterized target
in the brain. 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.
Full European Summary of Product Characteristics for ZTALMY®
(ganaxolone) oral suspension is available at
www.ema.europa.eu.
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 Durbin
A part of Uniphar Group’s Product Access Division, Durbin is a
specialist pharmaceutical services provider, distributing critical
medications to over 160 different countries. Durbin works in
partnership with global pharmaceutical and biotech companies to
provide Early Access Programs (EAPs), including Named Patient
Supply and Cohort Programs. The company has over 25 years’
experience designing and implementing EAPs and specializes in
strategic consultancy, asset specific program design and
innovative, customized delivery models.
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 the potential
benefits of the Marinus Access Program and the availability of
ZTALMY under the program; our commercialization and marketing plans
for ZTALMY; the potential benefits ZTALMY will provide for
physicians and patients; statements regarding our expected clinical
development plans, enrollment in our clinical trials, regulatory
communications and submissions for ganaxolone, and the timing
thereof; our expectations and beliefs regarding the FDA and EMA
with respect to our product candidates; our expectations regarding
our strategic partners; the pl safety and efficacy of ganaxolone,
as well as its therapeutic potential in a number of indications;
and other statements regarding the company’s future operations,
financial performance, financial position, prospects, objectives
and other future event.
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, our ability to successfully launch the Marinus Access
Program; unexpected results or delays in the commercialization of
ZTALMY; unexpected market acceptance, payor coverage or future
prescriptions and revenue generated by ZTALMY; 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 the company’s product candidates, and the
company’s ability to service those markets; the company’s cash and
cash equivalents may not be sufficient to support its operating
plan for as long as anticipated; the company’s expectations,
projections and estimates regarding expenses, future revenue,
capital requirements, and the availability of and the need for
additional financing; the company’s ability to obtain additional
funding to support its clinical development and commercial
programs; the potential for our ex-US 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; the effect of the COVID-19 pandemic on our business, the
medical community, regulators and the global economy; 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.
___________________________ 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231107340710/en/
Company
Investors Jim DeNike Senior
Director, Investor Relations Marinus Pharmaceuticals, Inc.
jdenike@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
Historical Stock Chart
Von Apr 2024 bis Mai 2024
Marinus Pharmaceuticals (NASDAQ:MRNS)
Historical Stock Chart
Von Mai 2023 bis Mai 2024