Marinus Pharmaceuticals announces NMPA approval of Ganaxolone for CDKL5 deficiency disorder

Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a leader in developing innovative therapeutics for seizure disorders, has achieved a significant milestone with the approval of its ganaxolone oral suspension by the China National Medical Products Administration (NMPA). This approval marks a crucial development in the treatment of epileptic seizures in patients aged two years and older who are diagnosed with CDKL5 deficiency disorder (CDD), a rare genetic condition.

In November 2022, Marinus Pharmaceuticals entered into a strategic collaboration with Tenacia Biotechnology, granting the latter rights to develop and commercialize ganaxolone in Mainland China, Hong Kong, Macau, and Taiwan. This partnership is structured around royalties and milestone payments linked to net sales and regulatory achievements, aiming to introduce this novel treatment to a significant patient population in these regions.

Dr. Xiaoxiang Chen, CEO of Tenacia Biotechnology, emphasized the urgent need for innovative treatments for CDD, noting the absence of approved therapies for this severe condition in China prior to ganaxolone. “We are poised to introduce the first and only treatment option for CDD patients in China, addressing a critical care gap as diagnosis rates continue to climb,” said Dr. Chen.

The NMPA’s approval was bolstered by data from the Phase 3 Marigold trial conducted by Marinus. This double-blind, placebo-controlled study involved 101 patients and demonstrated that ganaxolone significantly reduced major motor seizure frequency by 30.7% over 28 days compared to a 6.9% reduction with placebo, meeting the trial’s primary endpoint. Further, a long-term open-label extension study showed a median 49.6% reduction in seizure frequency among patients treated with ganaxolone for at least 12 months.

Kimberly McCormick, PharmD., Chief Regulatory and Quality Assurance Officer at Marinus, highlighted the broader implications of the approval. “This approval not only advances treatment options for patients with CDD but also reaffirms our commitment to addressing the complex needs of those living with rare genetic epilepsies globally,” she stated.

Ganaxolone, marketed under the brand name ZTALMY, is a neuroactive steroid that modulates GABAA receptors in the brain, a mechanism well-documented for its anti-seizure effects. This prescription medication has now secured regulatory endorsements in the United States, European Union, and China, underscoring its global therapeutic potential for patients with CDKL5 deficiency disorder.

The approval of ganaxolone in China is not just a win for Marinus Pharmaceuticals but represents a significant advancement in global healthcare, providing a new lifeline to patients suffering from debilitating seizures associated with CDD. It sets a precedent for future treatments and collaborations that could transform care standards for rare genetic disorders worldwide.