FDA Grants Orphan Drug Designation to Allena Pharmaceuticals’ Investigational Therapy for the Treatment of Primary Hyperoxaluria

Saturday, July 15, 2017

Allena Pharmaceuticals, Inc., a specialty biopharmaceutical company dedicated to bringing first in class, specific, non-absorbed, oral protein therapeutics to patients with serious renal, urologic and orphan diseases, announced today that the US Food and Drug Administration (FDA) has granted orphan drug designation to Allena’s investigational product ALLN-177, an oral formulation of oxalate decarboxylase, for the treatment of primary hyperoxaluria (PH).

Allena’s lead compound ALLN-177, is being developed to treat patients with severe hyperoxaluria, or patients with markedly elevated urinary oxalate excretion. PH, a type of severe hyperoxaluria, is a rare genetic disorder caused by endogenous overproduction of oxalate by the liver that can result in kidney stone disease, kidney damage, and kidney failure, which may lead to death. PH has three main types, PH1, PH2 and PH3, and is estimated to affect approximately 1 in 58,000 based on population analysis.1 The most severe and most common type among genetically characterized patients is PH1. These patients typically develop recurrent kidney stones with progressive nephrocalcinosis and end stage renal disease by 20-30 years of age.1 There are no FDA approved therapies for PH, and the most severe patients may be treated with liver and/or kidney transplant.

ALLN-177 is a first-in-class therapeutic being developed to treat patients with severe hyperoxaluria using an oral, non-absorbed enzyme that works in the gastrointestinal (GI) tract, where it can degrade both dietary and endogenously produced oxalate. GI elimination of oxalate may help alleviate the chronic systemic oxalate burden on PH patients.

“Primary Hyperoxaluria is a devastating disease for patients and their families. We desperately need better therapeutic options to treat this disease,” said Craig B. Langman, M.D., the Issac A. Abt M.D. Professor of Kidney Diseases at Feinberg School of Medicine, Northwestern University and Head, Kidney Diseases at Lurie Children’s Hospital Chicago.

The preclinical data supporting the FDA orphan designation demonstrated oxalate decarboxylase significantly reduced urinary and plasma oxalate in multiple animal models including a rodent disease model of PH and a porcine model with urine oxalate in the range seen in PH.

The Orphan Drug Designation Program is administered by the FDA's Office of Orphan Products Development, which grants orphan status to drugs intended to treat rare diseases that affect fewer than 200,000 people in the U.S. The program provides incentives for sponsors and has enabled the development and marketing of more than 400 products for rare diseases since 1983.

“This is an important regulatory designation to advance the development of ALLN-177 for patients who could benefit from novel therapeutic options that directly address excess oxalate,” said Louis Brenner, M.D., President and Chief Operating Officer of Allena Pharmaceuticals. “We are committed to the development of ALLN-177 for the treatment of patients with severe hyperoxaluria disorders.”

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