Marinus launches drug for seizures linked to CDKL5 deficiency disorder
Marinus Pharmaceuticals Inc. announced the Unites States commercial launch of Ztalmy oral suspension for the treatment of seizures associated with CDKL5 deficiency disorder in patients aged 2 years and older.
According to a company press release, Ztalmy (ganaxolone, Marinus) is the first and only FDA-approved treatment specifically indicated for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients as young as 2 years.
The drug — a sugar-free, cherry-flavored oral suspension administered three times per day — is a neuroactive steroid anticonvulsant that acts as a positive allosteric modulator of GABAA receptors in the central nervous system.
“Ztalmy represents an important first for both Marinus and the CDD community, and we are proud to make it available for patients by physician prescription,” Christy Shafer, chief commercial officer at Marinus, said in the release. “We look forward to engaging with health care providers and supporting patient access to Ztalmy as we raise awareness about CDD and this important new treatment option.”
Ztalmy was approved by the FDA in March 2022 based on data from the phase 3 Marigold double-blind, placebo-controlled trial of 101 patients with CDD, in which the therapeutic significantly reduced frequency of monthly major motor seizures by a median of 30.7% compared with 6.9% for placebo, the company stated. Ztalmy also demonstrated safety, efficacy and tolerability in the trial, with the most common adverse reactions including somnolence, pyrexia, salivary hypersecretion and seasonal allergy.