This section highlights FDA-related milestones and regulatory updates for drugs developed by Marinus Pharmaceuticals (MRNS).
Over the past two years, Marinus Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ganaxolone, Ganaxolone, and Ganaxolone. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ganaxolone - FDA Regulatory Timeline and Events
ganaxolone is a drug developed by Marinus Pharmaceuticals for the following indication: For the Adjunctive Treatment of Epileptic Seizures Associated with CDKL5 Deficiency Disorder.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ganaxolone
- Announced Date:
- October 9, 2024
- Indication:
- For the Adjunctive Treatment of Epileptic Seizures Associated with CDKL5 Deficiency Disorder
Announcement
Marinus Pharmaceuticals, Inc announced that additional data from the Phase 3 RAISE trial in refractory status epilepticus and retrospective claims-based data from patients with status epilepticus will be presented at the upcoming Neurocritical Care Society (NCS) 22nd Annual Meeting, being held October 14-17 in San Diego, California.
AI Summary
Marinus Pharmaceuticals, Inc. announced that new information from the Phase 3 RAISE trial in refractory status epilepticus and data from a retrospective claims-based analysis on status epilepticus management will be presented at the Neurocritical Care Society's 22nd Annual Meeting. These presentations will take place during the event scheduled for October 14-17, 2024 in San Diego, California. The Phase 3 trial data, focusing on the efficacy and safety of intravenous ganaxolone in treating refractory status epilepticus, will be delivered in a podium presentation on October 17, 2024, at 9:35 a.m. PT. Additionally, a poster presentation will discuss challenges in the management of status epilepticus, including a high rate of interhospital transfers, during the session on October 16, 2024, at 2:45 p.m. PT.
Read Announcement- Drug:
- ganaxolone
- Announced Date:
- July 18, 2024
- Indication:
- For the Adjunctive Treatment of Epileptic Seizures Associated with CDKL5 Deficiency Disorder
Announcement
Marinus Pharmaceuticals, Inc announced that the China National Medical Products Administration (NMPA) has approved ganaxolone oral suspension for the treatment of epileptic seizures in patients two years of age and older with CDKL5 deficiency disorder (CDD).
AI Summary
Marinus Pharmaceuticals, Inc. announced that China’s National Medical Products Administration (NMPA) has approved its ganaxolone oral suspension for the treatment of epileptic seizures in patients aged two years and older with CDKL5 deficiency disorder (CDD). CDD is a severe neurodevelopmental condition that previously lacked any approved treatment options in China. The approval was based on data from a Phase 3 clinical trial, where patients treated with ganaxolone experienced a notable reduction in seizure frequency compared to those on placebo. This achievement marks a significant advancement for patients and families affected by CDD, providing a new therapeutic option to manage the disorder’s unpredictable and challenging symptoms. The development underscores Marinus’ commitment, alongside its partner Tenacia Biotechnology, to bring innovative therapies to market and address unmet needs in rare and severe seizure disorders.
Read Announcement- Drug:
- ganaxolone
- Announced Date:
- April 15, 2024
- Indication:
- For the Adjunctive Treatment of Epileptic Seizures Associated with CDKL5 Deficiency Disorder
Announcement
Marinus Pharmaceuticals announced that an independent Data Monitoring Committee (DMC) has recommended continuing the pivotal Phase 3 RAISE trial evaluating intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE) following an interim analysis.
AI Summary
Marinus Pharmaceuticals announced that an independent Data Monitoring Committee (DMC) has recommended continuing the pivotal Phase 3 RAISE trial. This trial is studying intravenous (IV) ganaxolone for treating refractory status epilepticus (RSE), a serious condition causing prolonged seizures. The DMC’s recommendation came after an interim analysis, as the trial did not meet predefined early stopping criteria. Marinus has now completed enrollment of roughly 100 patients, and the topline results are expected in summer 2024. Once the complete data set is unblinded and analyzed, the company will decide whether to move forward with the development of IV ganaxolone. This decision marks an important step in evaluating the potential of ganaxolone in providing a new treatment option for RSE, with further development plans to be determined based on the final results from the RAISE trial.
Read Announcement
Ganaxolone (RAISE) - FDA Regulatory Timeline and Events
Ganaxolone (RAISE) is a drug developed by Marinus Pharmaceuticals for the following indication: Refractory status epilepticus (RSE).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Ganaxolone (RAISE)
- Announced Date:
- June 17, 2024
- Estimated Event Date Range:
- October 1, 2024 - December 31, 2024
- Target Action Date:
- Q4 2024
- Indication:
- Refractory status epilepticus (RSE)
Announcement
Marinus announced that it will continue to prioritize the ZTALMY® franchise and expansion opportunities in highly refractory, chronic epilepsies with Phase 3 TrustTSC topline data expected in the first half of Q4 2024
AI Summary
Marinus recently shared topline results from its Phase 3 RAISE trial, which evaluated IV ganaxolone for refractory status epilepticus. While the trial successfully met the first co-primary endpoint by showing a rapid cessation of seizures, it did not achieve statistical significance on the second endpoint, which looked at avoiding progression to IV anesthesia. Despite these mixed results, Marinus is staying optimistic and announced that it will continue to prioritize the ZTALMY® franchise. The company is also focusing on expanding opportunities in highly refractory, chronic epilepsies. Additionally, Marinus expects to release Phase 3 TrustTSC topline data in the first half of Q4 2024, which could provide further insight into its treatment strategy. These efforts come alongside cost reduction initiatives that are projected to extend the company’s cash runway into the second quarter of 2025.
Read Announcement- Drug:
- Ganaxolone (RAISE)
- Announced Date:
- June 17, 2024
- Indication:
- Refractory status epilepticus (RSE)
Announcement
Marinus Pharmaceuticals, Inc announced topline results from the Phase 3 double-blind, randomized, placebo-controlled RAISE trial (NCT04391569) evaluating the safety and efficacy of intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE).
AI Summary
Marinus Pharmaceuticals, Inc. announced topline results from the Phase 3 RAISE trial (NCT04391569), a double-blind, randomized, placebo-controlled study evaluating intravenous (IV) ganaxolone for treating refractory status epilepticus (RSE). The trial enrolled 96 patients who had not responded to at least two antiseizure medications. The study met its first co-primary endpoint: 80% of patients treated with IV ganaxolone experienced cessation of status epilepticus within 30 minutes compared to 13% in the placebo group, demonstrating a rapid antiseizure effect. However, the second co-primary endpoint—preventing progression to IV anesthesia for 36 hours—did not show a statistically significant difference between the ganaxolone and placebo arms. Despite this, additional EEG analyses indicated a durable reduction in seizure activity. Marinus intends to further analyze the data and discuss potential next steps with the FDA as it continues to develop innovative treatments for severe seizure disorders.
Read Announcement
Ganaxolone (TSC) - FDA Regulatory Timeline and Events
Ganaxolone (TSC) is a drug developed by Marinus Pharmaceuticals for the following indication: Tuberous Sclerosis Complex.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Ganaxolone (TSC)
- Announced Date:
- September 20, 2024
- Estimated Event Date Range:
- October 1, 2024 - December 31, 2024
- Target Action Date:
- Q4 2024
- Indication:
- Tuberous Sclerosis Complex
Announcement
Marinus Pharmaceuticals, Inc. announced that the Phase 3 TrustTSC trial topline data readout anticipated in the first half of the fourth quarter of 2024.
AI Summary
Marinus Pharmaceuticals, Inc. announced significant progress with its global Phase 3 TrustTSC trial for tuberous sclerosis complex (TSC). The trial, which has successfully completed enrollment, showed a low double-blind discontinuation rate of 6.2% and a high retention rate, with 93% of patients continuing into the open-label extension. These strong enrollment and retention numbers support the trial’s design to detect a meaningful reduction in seizure frequency among TSC patients.
The company anticipates a topline data readout from the TrustTSC trial in the first half of the fourth quarter of 2024. In parallel, Marinus is preparing to submit a supplemental New Drug Application (NDA) to the U.S. FDA, targeting an April 2025 filing. These milestones highlight the company’s ongoing commitment to addressing the unmet need in TSC patients facing refractory seizures.
Read Announcement- Drug:
- Ganaxolone (TSC)
- Announced Date:
- September 20, 2024
- Indication:
- Tuberous Sclerosis Complex
Announcement
Marinus Pharmaceuticals, Inc today is hosting an Investor and Analyst Day and will present an in-depth review of the Company's development program for ZTALMY® (ganaxolone) oral suspension CV in tuberous sclerosis complex (TSC)
AI Summary
Marinus Pharmaceuticals, Inc. is hosting an Investor and Analyst Day to present a detailed review of their ongoing development program for ZTALMY® (ganaxolone) oral suspension CV in the treatment of tuberous sclerosis complex (TSC). The event will focus on how this therapy is being advanced to help TSC patients who suffer from refractory seizures. Company leaders will discuss the clinical progress from the global Phase 3 TrustTSC trial, where early indicators have shown promising reductions in seizure frequency and a very low discontinuation rate. They will also share insights on patient outcomes from prior Phase 2 studies and update attendees on upcoming regulatory plans, including a targeted New Drug Application filing in April 2025. This session aims to provide investors and analysts a clear overview of the clinical strategy and the potential of ZTALMY to address a significant unmet need for TSC patients.
Read Announcement
