According to a fairly recent news release from biopharmaceutical company Epygenix Therapeutics, Inc. (“Epygenix”), the company’s therapy, EPX-200, received Orphan Drug designation from the FDA. Altogether, the therapy is designed for patients with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood epilepsy.

EPX-200

As described by Epygenix, EPX-200 is an FDA-approved weight management drug. The drug works by modulating serotonin (5HT) signaling pathways. Outside of weight management, Epygenix has been evaluating EPX-200 as a potential treatment for patients with LGS and Dravet Syndrome, another rare epileptic encephalopathy, using zebrafish models. After a small number of human patients with Dravet syndrome were treated, researchers determined that EPX-200 has anti-epileptic properties.

So why is receiving Orphan Drug status so important? The answer to this question comes in parts. First, LGS can be difficult to treat and manage using the current standards of care. Thus, new treatment options are crucial to improving patient outcomes and better managing seizures. Since the Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare conditions (affecting under 200,000 Americans), it makes sense that the status was granted to a drug intended to fulfill unmet needs within this patient population. Next, Epygenix benefits from the status, earning tax credits, fee waivers, increased FDA regulatory assistance, and 7 years of market exclusivity (if/once approved).

Lennox-Gastaut Syndrome (LGS)

Lennox-Gastaut syndrome (LGS) is a rare epileptic encephalopathy that first appears in childhood, and is characterized by tonic, atonic, and atypical absence seizures, as well as some intellectual disability. An estimated 5% of childhood epilepsy results from LGS. However, doctors are not exactly sure what causes LGS, especially as causes vary from patient to patient. Some known causes include low birth weight or premature birth, lack of oxygen or severe brain injury to child during birth, severe brain infections, or brain development issues while the fetus is still developing. Symptoms and seizures usually manifest before age 4. These include:

• Absence seizures – prolonged epileptic activity involving blank stares, rapid blinking, and head nodding
• Atonic seizures – characterized by sudden muscle tone loss, causing collapse or falls
• Tonic seizures – characterized by stiffened muscles, an upward eye gaze, and dilated pupils. Often happens during sleep.
• Intellectual and developmental delays
• Diffuse slow spikes and waves on EEG
• Loss of consciousness
• Difficulty concentrating
• Sudden muscle jerks or rhythmic muscle contractions
• Injuries from falls
• Behavior issues and/or psychotic episodes (hyperactivity, aggression, hypersexuality)