Reata Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration for its bardoxolone methyl to treat pulmonary arterial hypertension (PAH).
PAH is a life-threatening disease, which involves endothelial dysfunction, pulmonary vasoconstriction, vascular remodeling, pulmonary fibrosis, and right ventricular hypertrophy.
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Reata Pharmaceuticals 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.”
Currently, bardoxolone methyl is being assessed in the LARIAT study, a Phase II dose-ranging study designed to examine the safety, tolerability and efficacy of bardoxolone in patients with PAH.
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By GlobalDataLARIAT is a multi-centre, double-blind, randomised, dose-ranging, placebo-controlled study and its primary efficacy endpoint is a six-minute walk test.
In addition, PAH causes skeletal muscle dysfunction that leads to the exercise intolerance in patients.
Bardoxolone methyl showed antioxidant, anti-inflammatory and bioenergetic properties in preclinical studies, which may help to improve exercise tolerance in patients.
Reata’s lead drugs are known as antioxidant inflammation modulators (AIMs), which are potent activators of the transcription factor Nrf2 and potent inhibitors of the transcription factor NF-?B.
Image: Micrograph showing a plexiform lesion of the lung, as seen in irreversible pulmonary hypertension. Photo: courtesy of Bulent Celasun, MD.
