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news.Reata Pharmaceuticals, a clinical-stage biopharmaceutical company, announced the US Food and Drug Administration (FDA) has granted orphan drug designation to bardoxolone methyl (bardoxolone) for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
Image: The US FDA’s Center for Drug Evaluation and Research. Photo: courtesy of The U.S. Food and Drug Administration.
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ADPKD is the most common inherited form of kidney disease affecting approximately 140,000 patients in the United States. It is characterized by the development of pathologic fluid-filled cysts throughout the kidneys, which leads to organ enlargement and chronic kidney disease (CKD). Despite standard of care treatment, approximately 50% of these patients will progress to end-stage kidney disease and require dialysis or a kidney transplant by 60 years of age.
“Obtaining orphan drug designation for bardoxolone for the treatment of ADPKD is an important milestone for Reata. This is the third orphan drug designation obtained for bardoxolone in the United States for the treatment of diseases characterized by mitochondrial dysfunction and inflammation, and it is the second designation for the treatment of patients with rare forms of CKD,” said Warren Huff, Reata’s Chief Executive Officer and President.
“We believe that, if approved, bardoxolone may prove to be a meaningful new treatment option for patients with ADPKD.”
Last year, Reata released positive data from the ADPKD cohort of the PHOENIX Phase 2 study of bardoxolone in four rare forms of CKD. After 12 weeks of bardoxolone treatment, we observed a mean increase from baseline in estimated glomerular filtration rate (eGFR) of 9.3 mL/min/1.73 m2 (n=31; p<0.0001).
Notably, 96% of patients who reached Week 12 demonstrated an improvement in eGFR. The observed improvement represents a recovery of approximately two years of average eGFR loss. Based on these encouraging results, Reata launched FALCON, an international, double-blind, placebo-controlled, parallel group, Phase 3 study of bardoxolone in approximately 300 patients with ADPKD.
The primary efficacy endpoint, which may support accelerated approval under Subpart H, is the change from baseline in eGFR compared to placebo after 48 weeks of treatment followed by a 4-week drug withdrawal period.
Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the United States and provides specific incentives for the development of therapies intended for the treatment, diagnosis, or prevention of rare diseases. Such designation will provide Reata with certain development incentives, including tax credits for clinical testing, exemption from a prescription drug user fee, and seven years of market exclusivity.
Source: Company Press Release
