Marinus Pharmaceuticals Receives Positive Response from FDA on Sufficiency of One Phase 3 Clinical Trial for Filing of New Drug Application (NDA) for the Use of Ganaxolone in CDKL5 Deficiency Disorder (CDD)
RADNOR, Pa.--(BUSINESS WIRE) January 13, 2021 -- Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it has received a positive response from the U.S. Food and Drug Administration (FDA) that the efficacy and safety data resulting from the company’s pivotal Phase 3 Marigold Study on the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD) appear sufficient to support the filing of a New Drug Application (NDA). Adequacy of these data to support an approval of ganaxolone for the proposed indication will be a matter of future FDA review.
CDD is a rare, genetic epilepsy with refractory seizures. Marinus provided the data to the FDA in a briefing document meant to support a Type C meeting, and the FDA provided written preliminary comments in response. Based on this feedback, Marinus is targeting a pre-NDA meeting by the end of Q1 to support submission.
“We are pleased with the FDA’s assessment of the sufficiency of the efficacy and safety data from one Phase 3 clinical trial to support an NDA filing,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus. “We remain on track to submit the NDA for use of ganaxolone in CDD for FDA review by mid-2021 and look forward to continued advancement of ganaxolone as the first potential FDA approved treatment specifically indicated in patients diagnosed with CDD.”
In September, Marinus reported positive topline data from the Phase 3 Marigold Study, the first double-blind placebo-controlled trial to provide evidence of efficacy in CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients.
In the Phase 3 Marigold trial, patients treated with ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4.0% reduction for those receiving the placebo, achieving the trial’s primary endpoint (p=0.002). In this trial, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.
“We see continued progress in our other development programs,” continued Dr. Braunstein. “Marinus continues to initiate new sites to participate in the Phase 3 clinical trial of IV ganaxolone for the treatment of Refractory Status Epilepticus (RSE) or RAISE trial. While the COVID-19 pandemic is lengthening the time for new trial sites to open for enrollment, we do not anticipate this having major implications on the ultimate timing of trial results.”
“The Phase 2 Violet Study, evaluating both three times a day dosing of ganaxolone and the role of allopregnanolone sulfate as a biomarker in PCDH19-related epilepsy remains on track. In March, we plan to provide an update on this program,” said Dr. Braunstein. “Additionally, we expect to report top line data from our Phase 2 Tuberous Sclerosis Complex trial by late Q2.”
About CDKL5 Deficiency Disorder
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. Most children affected by CDD cannot walk, talk, or feed themselves. Currently, there are no therapies approved specifically for CDD.
Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Unlike benzodiazepines, ganaxolone exhibits antiseizure, antidepressant and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. More than 1,600 study participants, both adults and children, have received ganaxolone at therapeutically relevant dose levels and treatment regimens for up to four years.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders. Ganaxolone is a positive allosteric modulator of GABAA receptors that acts on a well-characterized target in the brain known to have anti-seizure, antidepressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus recently completed the first ever Phase 3 pivotal trial in children with CDKL5 deficiency disorder and is conducting a Phase 2 trial in tuberous sclerosis complex, as well as a Phase 2 biomarker-driven proof-of-concept trial in PCDH19-related epilepsy. The company is initiating a Phase 3 trial in status epilepticus.
Source: Marinus Pharmaceuticals
Posted: January 2021