Ganaxolone Approved in China for CDKL5 Deficiency Disorder Seizures

RADNOR, Pa.--Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company focused on developing innovative treatments for seizure disorders, has announced that China's National Medical Products Administration (NMPA) has approved ganaxolone oral suspension for treating epileptic seizures in patients aged two and older with CDKL5 deficiency disorder (CDD).

In November 2022, Marinus partnered with Tenacia Biotechnology, granting Tenacia the rights to develop and commercialize ganaxolone in Mainland China, Hong Kong, Macau, and Taiwan, in return for royalties and other payments tied to net sales and achieving commercial and regulatory milestones.

Dr. Xiaoxiang Chen, the Chief Executive Officer of Tenacia Biotechnology, highlighted the severity and progressive nature of CDD, noting that before ganaxolone, there were no approved treatments for this condition in China. Dr. Chen emphasized the urgent need for innovative therapies for patients and families dealing with CDD, especially as diagnosis rates continue to climb. He expressed anticipation in delivering the first and only treatment option for these patients in China.

The approval was based on findings from Marinus' Phase 3 Marigold trial in CDD, a double-blind placebo-controlled study involving 101 patients. The results showed that patients treated with ganaxolone experienced a median reduction of 30.7% in 28-day major motor seizure frequency, compared to a 6.9% reduction for the placebo group, meeting the trial’s primary endpoint (p=0.0036). Additionally, patients in an open-label extension study who were treated with ganaxolone for at least 12 months (n=48) showed a median 49.6% reduction in major motor seizure frequency. Ganaxolone was generally well-tolerated, with the most common adverse event being somnolence, consistent with previous clinical trials.

Kimberly McCormick, PharmD., Chief Regulatory and Quality Assurance Officer of Marinus, underscored the significant impact of CDD on patients and their families, noting the unpredictability and increased healthcare needs and costs associated with the disorder. McCormick stated that the approval in China is a crucial step for patients living with CDD and reaffirms Marinus' commitment to offering innovative treatments for rare genetic epilepsies worldwide. She also expressed gratitude for the cooperation and support from Tenacia and Chinese regulatory authorities in bringing this important treatment to CDD patients.

Ganaxolone has received regulatory approval in the United States, the European Union, and China for suitable CDD patients. As part of its collaboration agreements, Marinus supplies ganaxolone for all global markets.

ZTALMY (ganaxolone) oral suspension is a neuroactive steroid GABAA receptor modulator targeting a well-defined area in the brain known for its anti-seizure effects. This prescription medication has been approved by the U.S. Food and Drug Administration and the European Commission for patients with CDKL5 deficiency disorder.

Marinus Pharmaceuticals is a commercial-stage pharmaceutical company dedicated to developing innovative therapeutics for seizure disorders. The company introduced the FDA-approved prescription medication ZTALMY (ganaxolone) oral suspension CV in the U.S. in 2022.

Tenacia Biotechnology, based in China and supported by Bain Capital, is a clinical-stage biopharmaceutical company developing and commercializing innovative treatments for nervous system disorders. Tenacia is led by experts with extensive experience in life sciences, CNS, global and China-specific drug development and commercialization, and a track record of successful company formation. The company is advancing a portfolio of innovative, best-in-class therapies.

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