ADPKD Drug News

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Although its drug for use in patients with autosomal dominant polycystic kidney disease (ADPKD) is still pending FDA approval, Reata (Irving, TX) recently won orphan drug designation for the candidate drug, bardoxolone methyl, according to Zacks Equity Research.

In early June, the company started its phase 3 clinical trial, called the FALCON study, for patients with ADPKD, which is caused by mutations in the PKD1 and PKD2 genes, and often leads to end stage kidney disease.

This is the second orphan drug designation for treating patients with rare forms of kidney disease with bardoxolone. It is the third designation for using bardoxolone to treat a kidney disease characterized by cellular mitochondrial dysfunction.

According to the FDA, orphan drug status is based on factors including the pathogenesis of the disease or condition, course of the disease or condition, prognosis of the disease or condition, and resistance to treatment.

According to Zack’s Equity Research, the designation also entitles Reata to certain tax credits related to clinical trial expense exemption from the FDA user fee, and eligibility for seven years of exclusive marketing rights in the United States.

Recently a set of findings relevant to ADPKD patients using a different medication was published in Gastroenterology. In a phase 3 trial over 120 weeks (about 2.5 years) treatment with Somatuline Depot (lanreotide [Ipsen, Paris]) was effective at reducing organ volume. The drug lowered the growth of liver and combined liver and kidney volume in patients with polycystic liver disease. According to the official website for the drug, lanreotide is “the first and only FDA-approved treatment for adults both to slow the growth of gastrointestinal and pancreatic neuroendocrine tumors (GEP NETs) that have spread or cannot be removed by surgery . . . and to treat carcinoid syndrome to reduce the need for the use of short-acting somatostatin medicine.”

The study was launched to follow patients over a longer time period because previous studies with somatostatin analogues were “underpowered and of too short duration to reach a definitive conclusion about renal and hepatic protective efficacy for the drug candidate,” according to

van Aerts RMM. Lanreotide reduces liver growth in patients with autosomal dominant polycystic liver and kidney disease. Gastroenterol 2019; DOI: 10.1053j.gastrol2019.04.018.