Prior to the end of March 2023, ganaxolone had received six separate Orphan Drug designations from the FDA; more recently, as shared in a news release from commercial-stage pharmaceutical company Marinus Pharmaceuticals, Inc. (“Marinus”), it earned its seventh for the treatment of Lennox-Gastaut syndrome (LGS). Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare conditions. “Rare” refers to any condition affecting fewer than 200,000 people nationwide. This designation comes with a number of benefits such as increased regulatory communication, fee waivers and tax credits, and 7 years of market exclusivity upon drug approval. 

So what exactly is ganaxolone? According to Marinus, ganaxolone is:

a GABAA receptor modulator that acts by regulating brain activity. This process can include inhibiting the abnormal electrical discharges that cause seizures and status epilepticus or restoring balance in disrupted neuronal activity in other CNS disorders.

Marinus is developing both an intravenous (IV) and oral formulation of ganaxolone for LGS. The company hopes to evaluate ganaxolone for LGS in a study later this year. Outside of LGS, ganaxolone has already shown promise and efficacy in CDKL5 deficiency disorder (CDKL5 or CDD) and tuberous sclerosis complex (TSC). 

About Lennox-Gastaut Syndrome (LGS) 

Lennox-Gastaut syndrome is a severe form of epilepsy characterized by frequent or recurrent seizures that begin in childhood. There are a number of potential LGS causes: premature birth, low birth weight, brain injury during pregnancy or birth, brain infections, and lack of oxygen at birth. For some cases, the exact cause is unknown. 

In most individuals with LGS, seizures begin between ages 2-6. Children with LGS experience multiple types of seizures, such as:

• Tonic seizures that often occur during sleep and are characterized by muscle stiffening
• Atypical absence seizures which may cause a partial loss of consciousness and may be characterized by blank stares, nods, and blinking
• Atonic seizures characterized by a sudden loss of muscle tone 
• Status epilepticus which refers to prolonged periods of seizure activity

Unfortunately, these seizures often respond poorly to treatment and can be difficult to manage. Additional characteristics of LGS include injuries from falls, behavioral issues, and learning and developmental delays. Treatments include anti-epileptic medications, special diets, nerve stimulator placement surgery, and a corpus callosotomy in extremely severe cases. However, as shared above, LGS can be difficult to manage. Advocating for increased research and treatment development has the potential to change lives.