Marinus Launches Ztalmy for Rare Seizure Disorder

Marinus Pharmaceuticals has launched Ztalmy (ganaxolone) oral suspension, which was approved in March 2022 to treat patients two years of age and older, usually girls, with seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD), a rare, genetic form of epilepsy.

CDD is a serious and rare genetic disorder characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment. It’s caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. The gene produces a protein that is important for normal brain development and function. Many children diagnosed with CDD also experience scoliosis, visual impairment, sensory problems, gastrointestinal difficulties, and sleeping disorders.

Ztalmy, a neuroactive steroid that acts as a positive allosteric modulator of the GABAA receptor, is the first FDA-approved treatment specifically for CDD. Data from the phase 3 Marigold double-blind placebo-controlled trial of 101 patients with CDD, published in The Lancet Neurology in which ZTALMY significantly reduced the frequency of monthly major motor seizures by a median of 30.7% compared with 6.9% for placebo.

Related: FDA Approves First Therapy for Rare, Genetic Seizure Disorder

Marinus has established Ztalmy One, a patient support program that facilitates access to treatment and provides ongoing prescription drug support and education throughout the treatment journey. This includes prescription benefit and prior authorization support, medication delivery, financial support programs for patients with no insurance, limited insurance or a gap in coverage and a co-pay savings program that helps commercially insured eligible patients pay as low as $0 per prescription.

Additionally, the company has selected Orsini Specialty Pharmacy as the exclusive specialty pharmacy partner. Orsini is focused exclusively on rare diseases and gene therapies. olutions include medication adherence programs, data analytics, customized manufacturer programs, and nationwide nursing coverage for convenient in-home infusion services. Headquartered in Elk Grove Village, Ill., Orsini Specialty Pharmacy is accredited by the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC, NABP, and ACHC’s Distinction in Rare Diseases and Orphan Drugs.