Viamet Pharmaceuticals announced Thursday that VT-1129, a potent and selective, oral, antifungal agent, has been granted orphan drug designation by the U.S. Food & Drug Administration (FDA) for the treatment of cryptococcal meningitis, a
life-threatening invasive fungal infection of the lining of the brain and spinal cord. Viamet expects to submit an investigational new drug (IND) application for VT-1129 to the FDA during the first half of 2015.
Under the Orphan Drug Act, the FDA may designate a product as an orphan drug if it is a drug intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 individuals in the United States. Generally, if a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the product is entitled to a period of marketing exclusivity, which precludes the FDA from approving another marketing application for the same indication for that drug during that time period. The applicable period is seven years in the United States.
VT-1129 has been shown in preclinical studies to be a potent and selective, orally available inhibitor of fungal CYP51. VT-1129 blocks the production of ergosterol, an essential component of the fungal cell membrane, which is critical to fungal proliferation and survival. Additionally, VT-1129 has demonstrated substantial potency against Cryptococcus species and high concentrations within the central nervous system in preclinical studies. As a result of these advantageous properties, we selected VT-1129 for further preclinical development targeting the treatment of cryptococcal meningitis, a life-threatening fungal infection of the lining of the brain and the spinal cord. This infection occurs most often in immunocompromised patients, including those with HIV, transplant recipients and oncology patients. A study published in February 2013 in the scientific journal PLOS ONE estimated that there are 3,400 hospitalizations associated with cryptococcal meningitis annually in the United States.