Eiger BioPharmaceuticals, Inc. today announced that the European Medicines Agency (EMA) has granted Orphan Medicinal Product status to ubenimex for the treatment of pulmonary arterial hypertension (PAH).
"We are very pleased with the EMA Committee of Orphan Medicinal Products (COMP) designation of orphan status for ubenimex in PAH," said Joanne Quan, MD, Chief Medical Officer at Eiger. "We will soon begin enrolling the LIBERTY study, a Phase 2, randomized, double-blind, placebo-controlled, multi-center study of ubenimex in PAH patients."
Ubenimex is a well-characterized, oral, small-molecule, dual-inhibitor of aminopeptidase and leukotriene A4 hydrolase (LTA4H), the enzyme responsible for catalyzing the committed step in the formation of the pro-inflammatory mediator, LTB4. Ubenimex is approved in Japan as an adjunct to chemotherapy agents to extend survival and to maintain remission after treatment for acute non-lymphocytic leukemia in adults. Ubenimex has been used for over 25 years in Japan and remains commercially available through Nippon Kayaku under the brand name, Bestatin™. Ubenimex is not approved for any indication in the US or Europe. Ubenimex received orphan drug designation for PAH in the US in November 2015.
Pulmonary Arterial Hypertension is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. PAH begins when tiny arteries in the lungs, called pulmonary arterioles, become narrowed, blocked or destroyed. This makes it harder for blood to flow through the lungs, and raises pressure within the lungs' arteries. As the pressure builds, the heart's lower right chamber (right ventricle) must work harder to pump blood through the lungs, eventually causing the heart muscle to weaken and eventually fail. PAH is a progressive, life-threatening illness.
About Orphan Medicinal Product Designation
Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union. Orphan medicinal product designation qualifies the sponsor of the drug candidate for various development incentives, which may include fee waivers for regulatory procedures or a 10-year market exclusivity period following approval. Orphan medicinal product designation applies specifically to the active moiety and the indication for which it is granted, and is not applicable to other indications for that moiety.
Eiger is a clinical-stage biopharmaceutical company committed to bringing to market products for the treatment of rare diseases. The Company has built a diverse, clinical-stage portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology is clear and an effective therapy is urgently needed.
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Investors: Jim Shaffer, Eiger Bio, Inc., 919-345-4256, firstname.lastname@example.org
Source : prnewswire.com