June 1, 2007 — The US Food and Drug Administration (FDA) has granted orphan drug designation for aminolevulinic acid HCl in combination with photodynamic therapy for the treatment of esophageal dysplasia, a cyclosporine A drug for the treatment of vernal keratoconjunctivitis, and a peptidomimetic analog of hexarelin for the diagnosis of growth hormone deficiency in adults.
Orphan Drug Aminolevulinic Acid With Photodynamic Therapy ( Levulan Photodynamic Therapy) for Esophageal Dysplasia
On March 20, the FDA granted orphan drug designation to aminolevulinic acid HCl in combination with photodynamic therapy ( Levulan Photodynamic Therapy; DUSA Pharmaceuticals, Inc) for the treatment of esophageal dysplasia.
Esophageal dysplasia occurs in some patients with Barrett's esophagus, a condition in which the normal cells of the esophageal mucosa are replaced by columnar cells. Dysplasia is the degeneration of the abnormal mucosa, a premalignant phase for which treatment options are currently limited. Degeneration to high-grade dysplasia places patients at high risk for esophageal cancer.
Levulan is a formulation of aminolevulinic acid, a compound that is found naturally in the human body. When taken systemically, the drug is converted into a photosensitizer by rapidly growing cells, such as those found in esophageal dysplasia. When illuminated endoscopically by DUSA's light delivery system, a photochemical process occurs that selectively destroys the targeted cells.
Orphan Drug Cyclosporine A ( Vekacia) for Vernal Keratoconjunctivitis
On May 4, the FDA granted orphan drug designation to cyclosporine A ( Vekacia; Novagali Pharma) for the treatment of vernal keratoconjunctivitis (VKC), a rare and severe form of chronic allergic conjunctivitis characterized by hard, elevated bumps on the upper eyelid, mucous discharge, intense itching, and a debilitating sensitivity to light.
VKC occurs most often in children and young adults. There are currently no therapies approved for the treatment of VKC in the United States or Europe.
Orphan Drug Peptidomimetic Analog of Hexarelin for Growth Hormone Deficiency Diagnosis
On May 14, the FDA granted orphan drug designation to peptidomimetic analog of hexarelin (ARD-07; Ardana) for the diagnosis of growth hormone deficiency in adults.
Adult growth hormone deficiency is a syndrome associated with symptoms of increased body fat, decreased muscle and bone mass, and reduced strength, endurance, and vitality. It most commonly results from damage to the pituitary gland, which produces growth hormone.
Peptidomimetic analog of hexarelin is a growth hormone secretagogue, an agent that stimulates pituitary secretion of growth hormone in a dose-dependent manner when taken orally.
Medscape Medical News © 2007
Cite this: Jill Taylor. New FDA Orphan Drugs: Levulan, Vekacia, Peptidomimetic Analog of Hexarelin - Medscape - Jun 01, 2007.