Zevra Therapeutics Announces FDA Acceptance of IND Application for KP1077 in Narcolepsy
Zevra plans to expand KP1077 clinical program to address multiple rare sleep disorders with trials initiating in 2023
“The FDA’s acceptance of KP1077's IND for narcolepsy is an important step forward in the advancement of this novel therapy, creating a meaningful opportunity to expand our development efforts into this high-need indication,” said
Zevra filed the IND for KP1077 for the treatment of narcolepsy in
Narcolepsy is a chronic debilitating central disorder of hypersomnolence. The primary symptom of narcolepsy is excessive daytime sleepiness characterized by daily episodes of an irrepressible need to sleep or daytime lapses into sleep. People with narcolepsy have abnormal rapid eye movement (REM) sleep which can cause disrupted nighttime sleep, sleep paralysis and sleep-related hallucinations around sleep-wake transitions. Narcolepsy has severe personal, social, and economic consequences. People with narcolepsy experience substantial impairment of their mental and physical wellbeing, and depression and anxiety are common. Cognitive dysfunction, such as difficulty with focus, concentration and memory (also referred to as ‘brain fog’) is often reported. All of which contribute to overall disease burden and poor quality of life in people with narcolepsy.
Narcolepsy is categorized as narcolepsy type 1 (NT1) and type 2 (NT2). NT1 is considered a distinct entity characterized by loss of hypocretin neurons. When narcolepsy presents without cataplexy and CSF hypocretin-1 concentration that do not meet the type 1 criterion it is categorized as NT2.
The worldwide prevalence for both types of narcolepsy together has been estimated to be approximately 25 to 50 per 100,000 people. Epidemiological studies using well-defined criteria for estimating the prevalence of narcolepsy (both NT1 and NT2) in
To learn more about Zevra Therapeutics, visit zevra.com.
Zevra Therapeutics is a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. With unique, data-driven clinical, regulatory, and commercialization strategies, the Company is overcoming complex drug development challenges to bring much-needed therapies to patients.
Arimoclomol, Zevra’s orally-delivered, first-in-class investigational product candidate for the treatment of Niemann-Pick disease type C (“NPC”), has been granted orphan drug designation, Fast Track designation, Breakthrough Therapy designation and rare pediatric disease designation for NPC by the
KP1077 is Zevra’s lead clinical candidate being developed to treat idiopathic hypersomnia (“IH”) and narcolepsy. KP1077 is comprised solely of serdexmethylphenidate (“SDX”), Zevra’s proprietary prodrug of d-methylphenidate (“d-MPH”). The FDA has granted KP1077 orphan drug designation for the treatment of IH, and the
Early access programs are made available by
Caution Concerning Forward Looking Statements
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, and which can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue," "could," "intend," "target," "predict," or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include statements regarding: the promise and potential impact of our preclinical or clinical trial data, including without limitation the initiation, timing, expansion or results of any clinical trials or readouts, the timing or results of any Investigational New Drug (“IND”) applications and New Drug Application (“NDA”) submissions for KP1077 or any other product candidates for any specific disease indication or at any dosage, and our strategic and product development objectives. These forward-looking statements are based on information currently available to Zevra and its current plans or expectations and are subject to a number of known and unknown uncertainties, risks and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s (formerly KemPharm’s) Annual Report on Form 10-K for the year ended
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