In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. A rare disease is one affecting fewer than 200,000 people nationwide. Orphan Drug designation, designed to incentivize drug developers to create therapies for rare conditions, also comes with benefits such as fee waivers, tax credits, and seven years of market exclusivity upon approval. According to EMPR, serdexmethylphenidate (KP1077) was recently granted Orphan Drug designation for idiopathic hypersomnia (IH).
Developed by pharmaceutical company KemPharm, serdexmethylphenidate is described as:
a prodrug of an undisclosed parent drug that [KemPharm] believes has significant potential for treating IH while also addressing unmet needs with currently available therapies that are employed off-label in this patient population.
KemPharm has previously evaluated the safety and tolerability of serdexmethylphenidate in a Phase 1 study. 15 volunteers enrolled. The findings of the study were positive; KemPharm is now planning an upcoming clinical trial to evaluate the therapy for patients with IH. Additionally, at some point next year, the company hopes to evaluate serdexmethylphenidate as a potential treatment for narcolepsy.
What is Idiopathic Hypersomnia (IH)?
Unlike sleep disorders such as narcolepsy and cataplexy, the cause of idiopathic hypersomnia is unknown (idiopathic). This uncommon chronic neurological sleep disorder is characterized by excessive daytime sleepiness. For those living with IH, neither long naps or a full night’s sleep offer relief. Typically, IH manifests when people are teenagers or young adults, though it can arise at other ages. It may worsen at the start of a woman’s menstrual cycle. Symptoms and characteristics can include:
- Unrefreshing sleep even for long periods of time (10+ hours)
- Immediate and random need for sleep
- Headache
- Restlessness
- Low energy during the day
- Cold hands and feet
- Anxiety
- Irritation
- Sleep drunkenness (extreme difficulty waking up, dizziness, grogginess, disorientation)
- Appetite loss
