Zevra Therapeutics Reiterates Commitment to Rare Disease Community as New Corporate Council Member of the National Organization for Rare Disorders (NORD)
A rare disease is a condition that impacts fewer than 200,000 people in
“Zevra’s dedication to the rare disease community is critical as we all work to bring much-needed therapies and relief to people affected by a rare condition without treatment options," said
"Rare Disease Day is both a reminder of the strides the rare disease community has made over the last four decades with the support of NORD's leadership, as well as a call to action to continue making meaningful progress in rare disease therapies," said
Visit Zevra’s new corporate website at www.zevra.com to learn more.
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 Neimann-Pick disease type C ("NPC"), has been granted orphan drug designation, Fast Track designation and rare pediatric disease designation for the treatment of 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, including without limitation 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 our efforts to bring accessible treatments to patients with Niemann-Pick disease type C disease and idiopathic hypersomnia, 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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