Marinus Pharmaceuticals

Patients must satisfy the following criteria to be enrolled in the expanded access protocol:
Main Inclusion Criteria:
1. Molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required.
2. Male or female patients aged ≥ 2 years.
3. Informed Consent signed by the patient, patient’s parent(s) or LAR indicating that they understand the purpose of and procedures required for expanded access and are willing to participate in the expanded access protocol.
4. Age-appropriate assent, if indicated.
5. In the opinion of the Investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.
6. Able and willing to take investigational product (oral suspension) with food 3 times daily (TID). Ganaxolone must be administered with food.

Exclusion Criteria:
1. Pregnant or breastfeeding.
2. Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging).
3. Known allergic reaction or sensitivity to GNX or excipients.
4. Previously or actively participating in a clinical trial of GNX.
5. Participating in any study involving administration of an investigational agent, excluding devices.