Earlier today, Allena Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its compound, ALLN-177, for the treatment of primary hyperoxaluria (PH).
ALLN-177 is in development for the treatment of patients with severe hyperoxaluria, or patients with distinctly high urinary oxalate excretion.
PH is a rare type of hyperoxaluria, ultimately affects the kidneys as a result of a buildup of oxalate, a substance which is normally filtered through the kidneys and excreted through the urine. An accretion of oxalate deposits into the kidneys and urinary tract, and groups together with calcium to form kidney and bladder stones.
There are three types of PH – PH1, PH2, and PH3 – and among them, 1 in every 58,000 people are affected. There are currently no approved therapies for any of the subtypes, and in the most severe version (PH1), patients may require a liver and/or kidney transplant.
“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 in a press release
ALLN-177 is a novel therapeutic in development that intends to use an oral, non-absorbed enzyme as the mechanism of action. The enzyme operates in the gastrointestinal (GI) tract, where it can reduce dietary and endogenously-produced oxalate. Elimination of oxalate in the GI is proposed to assist in alleviating the continuing systemic oxalate burden on patients.
“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.”
A Phase 2b, multi-center, randomized, double blind, placebo-controlled, crossover study
was completed in January to evaluate the effect of ALLN-177 in reducing urinary oxalate in patients with hyperoxaluria and kidney stones. Study results have yet to be released.
The preclinical data that was the impetus behind the new FDA designation, though, established oxalate decarboxylase significantly reduced urinary and plasma oxalate in an array of animal models, including one rodent disease model of PH, and a porcine model with urine oxalate like that seen in PH patients.
For more information on FDA applications, designations and approvals, follow Rare Disease Report