Immedica plans new phase 3 study of IV ganaxolone for refractory status epilepticus in 2027

Immedica Pharma AB will advance intravenous ganaxolone into a new phase 3 study for refractory status epilepticus and plans to initiate the study in 2027, the company said on October 26, 2026. The decision follows a review of clinical data and interactions with the U.S. Food and Drug Administration, news.cision.com reported.

Immedica's Phase 3 Plans for IV Ganaxolone

Immedica is preparing the study design, manufacturing of clinical trial material and clinical site planning. The company said the FDA interactions provided a clear regulatory path forward for the program.

Refractory status epilepticus is a life-threatening neurological emergency in which ongoing seizure activity does not respond to at least two different antiseizure medications. Patients who continue to experience seizures after first- and second-line treatment may require intravenous anesthetics and medically induced coma. There is currently no approved therapy specifically for the condition.

"Advancing IV ganaxolone into a new phase 3 study is an important step for Immedica and reflects our commitment to developing treatments for people affected by severe conditions with high unmet medical need," said Anders Edvell, CEO of Immedica.

Dr Aatif M. Husain, Professor of Neurology and Division Chief, Epilepsy, Sleep, and Clinical Neurophysiology at Duke University School of Medicine, said IV ganaxolone works through a mechanism distinct from currently used therapies. Immedica acquired the global rights to ganaxolone through its acquisition of Marinus Pharmaceuticals in 2025.

Immedica's Rare Disease Portfolio

Immedica is a Stockholm-based pharmaceutical company that commercializes medicines for rare diseases and specialty care across more than 50 countries, according to akta.pro company records. The same records show the FDA granted accelerated approval to Loargys (pegzilarginase) in February 2026 for Arginase 1 Deficiency in patients aged 2 and older, based on reduction of plasma arginine, with confirmatory studies required post-approval.

Immedica completed the $65 million acquisition of Neurocrine Group Limited (Diurnal Group) in January 2026, acquiring global rights to Alkindi and ex-U.S. rights to Efmody and establishing rare endocrinology as a new therapeutic area.

Earlier Immedica Regulatory Milestones

Japan's Ministry of Health, Labor and Welfare granted Orphan Drug Designation to pegzilarginase for arginase 1 deficiency in August 2026, making the drug eligible for a priority review process, news.cision.com reported. Immedica plans to submit a Japanese New Drug Application.

In June 2026, Immedica announced that the Emirates Drug Establishment granted marketing authorization for Ztalmy (ganaxolone) to treat seizures associated with CDKL5 deficiency disorder in the UAE, news.cision.com reported.

Immedica published its 2025 Business Review and Annual & Sustainability Report in April 2026, citing pro forma net sales of EUR 134.5 million, news.cision.com reported.

What Comes Next for the Ganaxolone Program

Immedica plans to initiate the new phase 3 study in 2027. The company is finalizing the study design, manufacturing clinical trial material and planning clinical sites.