In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A “rare” condition is one affecting under 200,000 Americans. Once a therapy receives this status, the drug developer also receives benefits such as fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval. In a news release from early February 2022, specialty ketamine pharmaceuticals company PharmaTher Holdings Ltd. (“PharmaTher”) shared that it had received Orphan Drug designation for ketamine, designed to treat patients with status epilepticus (SE).
Developing Unique Ketamine Solutions
To begin, let’s first get a basic understanding of what ketamine is. Initially, ketamine was used as an anesthetic in animals. It was approved for human use in 1970. Ketamine has hallucinogenic properties, but may also be used for pain relief and, more recently, treating mental health issues such as depression or anxiety. Learn more about ketamine.
Currently, PharmaTher has numerous ketamine therapies in its pipeline, including treatments for depression, complex regional pain syndrome (CRPS), Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The therapies may be delivered via a microneedle (MN) patch or intravenously. Since an estimated 35-45% of those experiencing status epilepticus do not respond to benzodiazepines, the current standard-of-care, ketamine could significantly improve patient outcomes and fill an unmet need.
About Status Epilepticus (SE)
Status epilepticus (SE) should be considered a medical emergency. This rare and potentially fatal condition is characterized by a non-stop seizure, or recurring seizures, lasting five or more minutes. During this time, patients do not regain consciousness. Without intervention, SE may cause permanent damage and can be fatal. For example, an estimated 150,000 SE incidences occur in the U.S. each year, with up to ⅓ of cases becoming fatal. In children, an infection with a fever is a leading cause of SE. For adults, a stroke, poorly managed epilepsy, imbalanced blood sugar, alcohol withdrawal, kidney or liver failure, or genetic conditions may cause SE. Symptoms associated with SE include:
- Recurring seizures without full consciousness OR a continuous seizure which lasts for 5+ minutes
- Muscle spasms
- Falling
- Confusion
- Loss of consciousness
- Clenched teeth
- Irregular breathing
- Uncommon noises (such as grunting)
- Inability to speak
- Loss of bladder or bowel control
- “Far-off” look
