Arno Therapeutics, Inc. (OTCBB: ARNI) announced today that its investigational compound known as AR-42 has been granted orphan-drug designation by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products for the treatment of neurofibromatosis type 2 (NF2). NF2 is a rare genetic disorder characterized by the growth of noncancerous tumors in the brain and spinal cord, juvenile cataracts and neurofibromas of the skin. The disease is caused by mutations in a gene which plays a role in preventing cells from dividing uncontrollably.
“The EMA's orphan-drug designation represents another important step in our development program for AR-42 that will help us pursue its clinical development for the treatment in this rare and debilitating disease.”
Orphan-drug designation is granted in the European Union for medicines to treat life-threatening or debilitating conditions affecting no more than 5 in 10,000 people. It qualifies the developer for inspection-fee reductions, protocol assistance, a centralized application procedure and 10 years of marketing exclusivity upon the drug's approval.
Glenn Mattes, President and Chief Executive Officer of Arno, stated: "The EMA's orphan-drug designation represents another important step in our development program for AR-42 that will help us pursue its clinical development for the treatment in this rare and debilitating disease."
AR-42 is a novel, orally available, broad-spectrum deacetylation inhibitor of both histone and non-histone proteins which has demonstrated greater potency and activity in solid tumors and hematological malignancies when compared in preclinical studies to vorinostat (also known as "SAHA" or Zolinza®), the first of two marketed compounds in this class. AR-42 is currently being studied in a Phase I/IIa clinical trial in adult patients with relapsed or refractory hematologic malignancies and solid tumors. In addition to this orphan designation by the EMA, AR-42 been designated an orphan drug by the U.S. Food and Drug Administration for the treatment of meningioma and schwannoma of the central nervous system; it is currently under FDA review for U.S. orphan-drug designation for the treatment of neurofibromatosis type 2-associated central nervous system tumors and EMA review for European orphan-drug designation for the treatment of meningioma and schwannoma.
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