FDA Accepts IND for Optina (Danazol) for Treatment of Diabetic Macular Edema
Clinical trial expected in first quarter, 2013 (Jan. 22)The FDA has accepted an investigational new drug (IND) application for Optina (danazol, Ampio Pharmaceuticals) for the treatment of diabetic macular edema (DME). A clinical trial is expected to begin in the first quarter of 2013. The FDA granted the drug 505(b)(2) status in July, 2012. Drugs designated under this pathway can be approved based on the results of a single study.
The planned multicenter trial will evaluate the safety and efficacy of 12 weeks of treatment with oral Optina compared with placebo in adult patients with DME. The patients will be randomized to receive one of two dosages of Optina (0.5 mg per BMI or 1.0 mg per BMI per day) or placebo. After the patients have completed 4 weeks of initial treatment, an interim analysis will determine the best dose of Optina. Following the 12-week active treatment period, there will be a further 4-week washout period. The primary endpoint is improvement in visual acuity, defined by responder status, compared with placebo. Following treatment and washout, the patients will be assessed for vision regression, and a 12-week open-label extension study will be offered to evaluate the duration of the effect of the optimal dose. A total of 450 patients are expected to enroll.
Optina is a drug based on a low dose of danazol mdash; a weak androgen, low-molecular-weight, lipophilic steroid. In vitro data suggest that danazol has a biphasic effect on endothelial cells: at low doses, the drug decreases vascular leakage, while at higher concentrations an increase in vascular permeability is observed.
Source: Ampio Pharmaceticals; January 22, 2013.