Jazz Pharmaceuticals announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration on December 20, 2017 seeking marketing approval for solriamfetol (JZP-110), an investigational medicine for the treatment of excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea (OSA).
"Excessive sleepiness due to narcolepsy or OSA can affect a person's ability to function at work or at home, and many patients unfortunately might suffer for years before their condition is properly addressed," said Karen Smith, M.D., Ph.D., executive vice president, research and development and chief medical officer at Jazz Pharmaceuticals. "Jazz is committed to addressing unmet needs in this area by improving awareness and accurate diagnosis of these conditions and by delivering meaningful treatment options."
The solriamfetol Phase 3 clinical program includes one study evaluating excessive sleepiness in adult patients with narcolepsy (TONES 2), two studies evaluating excessive sleepiness in adult patients with OSA (TONES 3 and TONES 4), and an open-label, long-term safety and maintenance of efficacy study (TONES 5) in the treatment of excessive sleepiness in patients with narcolepsy or OSA.
OSA is a prevalent disease (as high as 14% in men and 5% in women) with excessive sleepiness being a major presenting complaint in many cases. Excessive sleepiness in OSA is associated with impairments in cognitive function, safety, productivity, interpersonal relationships, and overall quality of life. Positive Airway Pressure (PAP) therapy, with its most common form being Continuous Positive Airway Pressure (CPAP), has been shown to be an effective therapy for sleep-related airway obstruction, with frequent improvement in excessive sleepiness in many patients; however, not all patients tolerate CPAP therapy and among those who tolerate CPAP, usage is highly variable. It is estimated that excessive sleepiness persists in 13%–65% of people utilizing CPAP for OSA.
Narcolepsy is a debilitating neurological disorder characterized by excessive sleepiness, and the inability to regulate sleep-wake cycles normally. It affects an estimated one in 2,000 people in the United States, with symptoms typically appearing in early adulthood. It is estimated that more than 50% of patients with narcolepsy have not been diagnosed. Studies have shown it may take 10 years or more for people with narcolepsy to receive a correct diagnosis. Excessive sleepiness is the primary symptom of narcolepsy and is present in all people with the disorder. Excessive sleepiness is characterized by the inability to stay awake and alert during the day resulting in unplanned lapses into sleep or drowsiness.
Solriamfetol (JZP-110) is a selective dopamine and norepinephrine reuptake inhibitor (DNRI) in development for treatment of excessive sleepiness in adult patients with narcolepsy, OSA, and Parkinson's disease. In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma. Jazz Pharmaceuticals has worldwide development, manufacturing, and commercialization rights to solriamfetol, excluding certain jurisdictions in Asia. SK Biopharmaceuticals, the discoverer of the compound (also known as SKL-N05), maintains rights in Korea, Japan, China, Taiwan, Singapore, Indonesia, India, Philippines, Thailand, Malaysia, Vietnam, and Hong Kong. Solriamfetol has orphan drug designation in the United States for narcolepsy.