Late this afternoon, Marinus Pharmaceuticals Inc. (MRNS:NASDAQ), a pharmaceutical company focused on developing medications to treat seizure disorders, announced that "the U.S. Food and Drug Administration (FDA) has approved ZTALMY® (ganaxolone) oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD), a rare form of genetic epilepsy, in patients two years of age and older."
The firm explained that ZTALMY is "a neuroactive steroid that acts as a positive allosteric modulator of the GABAA receptor," and noted that ZTALMY is the first FDA approved treatment approved specifically for use in treating CDD in patients ages two and older. The company advised that following scheduling by the U.S. Drug Enforcement Administration it expects that ZTALMY will be commercially available in July 2022 through a designated specialty pharmacy.
Marinus Pharmaceuticals' CEO Scott Braunstein, M.D. commented, "Today is a historic milestone not only for Marinus but for CDD patients, families, and caregivers who have long been navigating the unpredictable, often devastating reality of living with uncontrolled seizures…We are grateful and humbled by the opportunity to bring the first and only FDA-approved treatment for seizures associated with CDD to this community."
The firm explained that "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."
The Marigold trial's Principal Investigator, Scott Demarest, M.D., a neurologist and Clinical Director of Precision Medicine at Children's Hospital Colorado, remarked, "There has been a great unmet medical need for treatments that address seizures associated with CDKL5 deficiency disorder given their prominent role and profound impact on patients…To date, antiseizure treatment decisions have been based on very limited clinical evidence in this patient population and the resulting outcomes underscore the need for therapies that further improve seizure control."
"Thanks to our research and this trial, we now have the first treatment specifically approved for seizures associated with CDKL5 deficiency disorder that was shown to have a positive benefit-risk profile," Dr. Demarest added.
The firm noted that the FDA's approval of ZTALMY in CDD was based upon data collected during its Phase 3 Marigold clinical trial. In the randomized study of 101 patients, ZTALMY demonstrated a median 30.7% reduction in 28-day major motor seizure frequency, versus a median 6.9% reduction in the control group. Results from the open label extension study were even better as patients given ZTALMY over a period of 12 months or longer showed a median 49.6% reduction in major motor seizure frequency.
International Foundation for CDKL5 Research Co-founder and President Karen Utley stated, "As the mother of a daughter living with CDD, I've experienced first-hand the devastating impact seizures can have on these patients…This approval is monumental for the CDD community—bringing not only the first approved treatment option specifically for CDD patients, but renewed hope to those who have struggled to find medications that are effective in significantly reducing the number of seizures these patients experience on a daily basis."
The company stated that "the FDA reviewed ZTALMY under Priority Review and granted ZTALMY orphan drug and Rare Pediatric Disease designations for the treatment of CDD and added that with the approval, the FDA awarded a Rare Pediatric Disease Priority Review Voucher (PRV), which Marinus plans to monetize."
The company advised that it has scheduled a virtual conference call with investors on Monday, March 21, 2022, at 8:00 a.m. ET to discuss the FDA's approval of ZTALMY along with other recent developments at the company during Q4/21. Those interested in joining the call can do so via the investors 'Events & Presentations' section on the Marinus website.
Marinus Pharmaceuticals is based in Radnor, Pa. and is focused on developing treatments for seizure disorders. The firm explained that its leading drug candidate ganaxolone is "a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects." The drug is currently being developed in both IV and oral dose formulations in order to deliver optimal therapeutic benefits to pediatric and adult patients in both acute and chronic care settings. The company is evaluating ganaxolone for use in several other indications including an ongoing Phase 3 trial in refractory status epilepticus. The company is dedicated to working closely with patients, physicians and the epilepsy community to provide much needed treatment options to children and adult patients afflicted with seizure disorders.
Marinus Pharmaceuticals began the day with a market cap of around $315.8 million with approximately 36.8 million shares outstanding and a short interest of about 3.4%. MRNS shares opened unchanged Friday at $8.59 (+$0.00, +0.00%) from Thursday's $8.59 closing price. The stock traded between $8.50 and $12.37 per share and closed for trading at $10.00 (+$1.41, +16.41%).
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