28 July 2022
First and only FDA-approved treatment for seizures associated with CDKL5 deficiency disorder in patients 2 years of age and older
ZTALMY One™ offers comprehensive patient services, including access and prescription drug support
RADNOR, Pa.--(BUSINESS WIRE)-- Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced the U.S. commercial launch of ZTALMY® (ganaxolone) oral suspension, CV for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in patients two years of age and older1.
“ZTALMY represents an important first for both Marinus and the CDD community and we are proud to make it available for patients by physician prescription,” said Christy Shafer, Chief Commercial Officer of Marinus. “With our highly experienced commercial team, designated specialty pharmacy and comprehensive patient support program, we are well-prepared to bring ZTALMY to market. We look forward to engaging with healthcare providers and supporting patient access to ZTALMY as we raise awareness about CDD and this important new treatment option.”
Physicians can now discuss the availability of ZTALMY as a treatment option with their patients and can access important information at ZTALMYhcp.com about prescribing ZTALMY and the prescription fulfillment process through Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases and gene therapies.
As part of Marinus’ commitment to helping people who are prescribed ZTALMY receive the support they need, the Company has established ZTALMY One™, a comprehensive patient support program that facilitates access to treatment and provides ongoing prescription drug support and education throughout the treatment journey. This includes prescription benefit and prior authorization support, medication delivery, financial support programs for patients with no insurance, limited insurance or a gap in coverage and a co-pay savings program that helps commercially insured eligible patients pay as low as $0 per fill for a ZTALMY prescription. Visit ZTALMY.com to learn about full program offer and terms and conditions. The ZTALMY One support team is available Monday through Friday, 8:00 a.m. to 8:00 p.m. Eastern Time, with pharmacists available 24/7.
ZTALMY was approved by the U.S. Food and Drug Administration in March 2022 based on data from the Phase 3 Marigold double-blind placebo-controlled trial of 101 patients with CDD, published in The Lancet Neurology2, in which ZTALMY significantly reduced the frequency of monthly major motor seizures by a median of 30.7% compared with 6.9% for placebo (p=0.0036). In this trial, 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.
To learn more about ZTALMY and ZTALMY One, please visit ZTALMY.com.
CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a mutation of the cyclin‑dependent kinase‑like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment.
ZTALMY® is the first and only FDA-approved treatment indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. ZTALMY, a neuroactive steroid anticonvulsant that acts as a positive allosteric modulator of GABAA receptors in the central nervous system, is a sugar-free cherry-flavored oral suspension taken three times daily.
ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.
Warnings and Precautions
Somnolence and Sedation: ZTALMY can cause somnolence and sedation. In a clinical study somnolence and sedation appeared early during treatment and were generally dose related. Other CNS depressants, including opioids, antidepressants, and alcohol, could potentiate these effects. Monitor patients for these effects and advise them not to drive or operate machinery until they have gained sufficient experience on ZTALMY to gauge whether it adversely affects their ability to drive or operate machinery.
Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including ZTALMY, increase the risk of suicidal thoughts or behavior. Monitor patients taking ZTALMY for the emergence or worsening of depression, suicidal thoughts or behavior, or any unusual changes in mood or behavior. Advise patients, caregivers, and their families to be alert for these behavioral changes and report behaviors of concern immediately to healthcare providers. When considering ZTALMY, or any other AED, balance the risk of suicidal thoughts or behaviors with the risk of untreated illness. If these symptoms emerge during treatment, consider whether it may be related to the AED or the underlying illness.
Withdrawal of Antiepileptic Drugs: As with most AEDs, withdraw ZTALMY gradually to minimize the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse event, rapid discontinuation can be considered.
The most common adverse reactions (incidence of at least 5% and at least twice the rate of placebo) were somnolence, pyrexia, salivary hypersecretion, and seasonal allergy.
Cytochrome P450 inducers will decrease ganaxolone exposure. Avoid concomitant use with strong or moderate CYP3A4 inducers; if unavoidable, consider a dosage increase of ZTALMY, but do not exceed the maximum recommended dosage.
Use In Specific Populations
Pregnancy: Use caution when ZTALMY is administered to pregnant women as there are no adequate data on the developmental risk associated with use in pregnant women. In animal studies, developmental adverse effects were observed following exposure during organogenesis or throughout gestation and lactation.
Lactation: ZTALMY is excreted in human milk at concentrations resulting in a dose to the breastfed infant of less than 1% maternal dose. The effects of ZTALMY on milk production and the breastfed infant are unknown.
Hepatic Impairment: Monitor patients with hepatic impairment for the incidence of adverse reactions. Patients with hepatic impairment may require a reduced dosage of ZTALMY.
Drug Abuse and Dependence
ZTALMY contains ganaxolone, a Schedule V controlled substance (CV). Advise patients of the potential for abuse and dependence. It is recommended that ZTALMY be tapered according to the dosage recommendations unless symptoms warrant immediate discontinuation.
Full Prescribing Information for ZTALMY® is available here.
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.
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 commercial launch of ZTALMY; the potential benefits ZTALMY will provide for physicians and patients; our expectations regarding the ZTALMY One program; our expected clinical development plans, enrollment in our clinical trials, regulatory communications and submissions for ganaxolone, and the timing thereof;; 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 commercialization and 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 results or delays in the commercial launch of ZTALMY, including any potential delays caused by the current COVID-19 global pandemic, 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; and the company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated. 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 ZTALMY (ganaxolone) oral suspension prescribing information. Marinus Pharmaceuticals, Inc. 6/2022.
2The Lancet Neurology, Volume 21, Issue 5, P417-427, May 01, 2022
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