Reata Pharmaceuticals, a US-based developer of breakthrough medicines, has secured orphan drug designation from the FDA Office of Orphan Products Development (OOPD) for bardoxolone methyl to treat pulmonary arterial hypertension (PAH).
PAH is a life-threatening disease involving endothelial dysfunction, pulmonary vasoconstriction, vascular remodeling, pulmonary fibrosis, and right ventricular hypertrophy.
In addition, PAH involves skeletal muscle dysfunction that contributes to the exercise intolerance observed in these patients.
In preclinical trials, bardoxolone methyl has showed potent antioxidant, anti-inflammatory, and bioenergetic properties, which may lead to improved exercise tolerance in patients.
Currently, the company is evaluating the safety, tolerability and efficacy of bardoxolone methyl in the LARIAT study, a Phase II dose-ranging trial in PAH patients.
Reata chief medical officer Dr Colin Meyer said: "Pulmonary arterial hypertension is a devastating disease for patients and significantly decreases their quality of life and lifespan.
"The FDA’s decision to grant orphan designation is an important step in the development of bardoxolone methyl to potentially treat this population with high unmet need.
"We believe the novel mechanism of activating Nrf2 has profound bioenergetic effects which have the potential to meaningfully impact the course of disease."
The primary efficacy endpoint of the multi-center, double-blind, randomized, dose-ranging, placebo-controlled LARIAT trial is a six minute walk test.