Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.
“We are pleased that ganaxolone has received Rare Pediatric Disease Designation from the FDA and are enthusiastic that the timing of this news aligns with our continued expectation to report top line data this quarter from our pivotal Phase 3 Marigold Study evaluating oral ganaxolone in patients with CDD,” said Scott Braunstein, M.D., Chief Executive Officer at Marinus. “This designation for ganaxolone for CDD underscores the significant unmet medical need for children and young adults with this serious and rare genetic disease, which causes early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Our goal is to advance a pipeline of programs that can treat rare seizure disorders, including CDD, tuberous sclerosis complex and PCDH19-related epilepsy, and bring much-needed medicines to patients as soon as possible.”
FDA grants RPD Designation for diseases that affect fewer than 200,000 people in the U.S. in which the serious or life-threatening manifestations are primarily in individuals 18 years of age and younger. If a new drug application (NDA) for ganaxolone in CDD is approved, Marinus may be eligible to receive a priority review voucher from the FDA, which can be redeemed for priority review in a subsequent marketing application. The program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
“The data that were used to support the Rare Pediatric Disease Designation comprised an analysis of existing literature as well as unpublished data specific to the CDD population, documenting that the serious or life-threatening manifestations of CDD primarily affect children,” said Joseph Hulihan, M.D., Chief Medical Officer at Marinus. "We plan to publish these new data that will describe important clinical and health economic outcomes related to how seizure burden in children with CDD may affect future developmental milestones."
The current program for RPD Designation will expire after September 30, 2020 unless Congress renews the program; however, a drug designated for a rare pediatric disease on that date can still receive a voucher if the drug is submitted and approved by the September 30, 2022 deadline.
In advance of topline data from that study, Marinus has begun preparations for an Expanded Access Program (EAP) in CDD that will allow the company, on positive data, to offer ganaxolone to patients who were unable to participate in the Phase 3 study. The global, double blind, placebo-controlled Phase 3 Marigold Study evaluating the use of oral ganaxolone in children and young adults has enrolled 101 patients between the ages of 2 and 21 with a confirmed disease related CDKL5 gene variant.