Immedica presents data at AES related to recent FDA approval of updated ZTALMY® (ganaxolone) label, highlighting the new 28-day titration schedule
Chicago, IL, December 10, 2025 – At the American Epilepsy Society (AES) 2025 Annual Meeting this week, Immedica Pharma US Inc. presented data that supports the recent United States Food and Drug Administration (FDA) update to the ZTALMY® (ganaxolone) label. ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two years of age and older.
“Revising the ZTALMY titration schedule has the potential to improve ZTALMY tolerability during the up-titration period with respect to the somnolence-related adverse events,” said Dan Camardo, President of Immedica Pharma US Inc. “This label update reflects our commitment to taking a patient-centered approach to rare diseases.”
The updates to the approved ZTALMY label include a modified titration schedule which is characterized by a lower initial dose of ZTALMY, graded dose progression, and extending the up-titration time from 21 to 28 days. This revised titration schedule targets the same maintenance dose of 1800 mg/day for patients weighing more than 28 kg or 63 mg/kg/day for patients weighing 28 kg or less. The titration schedule was also revised for patients with severe hepatic impairment.
“This update may benefit future ZTALMY patients by enabling them to initiate therapy and successfully up titrate over 28 days, resulting in a potentially lower risk of somnolence-related adverse events,” stated Mary Kay Koenig, MD, Professor & Associate Vice-Chair for Clinical Research at UTHealth Houston. “We’ve learned that modifying the titration schedule does not impact the final ZTALMY exposure levels that have demonstrated effectiveness in CDD patients.”
Data supporting these changes emerged following a post-hoc analysis of the Marigold clinical trial (NCT03572933) of ZTALMY in CDD which found that somnolence-related adverse events (including somnolence, sedation, hypersomnia, and lethargy) were reported during the up-titration period of ZTALMY treatment (Data on file. Immedica Pharma AB). To address this, the ZTALMY titration schedule was modified and studied in a separate clinical trial (TrustTSC: NCT04285346) in patients with Tuberous Sclerosis Complex (TSC). While the primary endpoint was not met in this study, somnolence-related adverse events were lowered with the modified titration schedule (Data on file. Immedica Pharma AB).
Key findings supporting this label update were presented at the AES 2025 Annual Meeting in Atlanta on December 8th, 2025.
ZTALMY was approved by the FDA in March 2022 based on the Marigold Phase 3 clinical trial data.
Please see the INDICATION AND SAFETY SUMMARY WITH WARNINGS below, and the full prescribing information for ZTALMY® (ganaxolone) oral suspension CV.
About ZTALMY® (ganaxolone)
ZTALMY® is the first and only FDA-approved treatment indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. ZTALMY, a neuroactive steroid anticonvulsant that acts as a positive allosteric modulator of GABAA receptors in the central nervous system, is a sugar-free cherry-flavored oral suspension taken three times daily.
About CDKL5 Deficiency Disorder
CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a pathogenic variant in the cyclin‑dependent kinase‑like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment.
About the Marigold Trial
ZTALMY was approved by the U.S. Food and Drug Administration in March 2022 based on 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 (p=0.0036). In this trial, ZTALMY demonstrated efficacy, safety and tolerability with the most common adverse reactions (incidence ≥5% and at least twice the rate of placebo) in the ZTALMY group being somnolence, pyrexia, salivary hypersecretion and seasonal allergy.
About the TrustTSC Trial
TrustTSC (NCT05323734) was a global Phase 3 randomized, double-blind, placebo-controlled clinical trial of adjunctive oral ganaxolone treatment in children and adults with TSC-related epilepsy. The trial consisted of a four-week baseline period, followed by a 16-week double-blind treatment phase, which included a 28-day titration period followed by a 12-week maintenance period. Participants then had the opportunity to enter the ongoing long-term open-label extension. The primary endpoint was percent change in 28-day TSC-associated seizure frequency. To be eligible, participants were required to have inadequate seizure control and have been treated with at least two prior antiseizure medications. (Data on file. Immedica Pharma AB.)
About Immedica Pharma US Inc.
Immedica Pharma US Inc. is an affiliate of Immedica Pharma AB, a pharmaceutical company headquartered in Stockholm, Sweden. Immedica is focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology and specialty care. Immedica was founded in 2018 and employs today around 160 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo.
For more information visit www.immedicaus.com | https://www.ztalmyhcp.com/
Immedica contact:
Linda Holmström
VP, Head of HR & Communication
linda.holmstrom@immedica.com