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
RADNOR, Pa., Nov 8 (Bernama-BUSINESS WIRE) — 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 regulatoryrequirements. 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.
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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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Jim DeNike
Senior Director, Investor Relations
Marinus Pharmaceuticals, Inc.
jdenike@marinuspharma.com
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Molly Cameron
Director, Corporate Communications & Investor Relations
Marinus Pharmaceuticals, Inc.
mcameron@marinuspharma.com
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