Allena Pharmaceuticals, Inc., a specialty biopharmaceutical company focused on developing and commercialising innovative, non-systemic, oral protein therapeutics to treat metabolic and orphan diseases, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to Allena’s investigational therapy oxalate decarboxylase for the treatment of paediatric hyperoxaluria.
Allena’s lead compound ALLN-177, an oral formulation of oxalate decarboxylase, is being developed to treat patients with disorders of oxalate metabolism. Hyperoxaluria is a metabolic disorder characterized by excess excretion of oxalate in the urine. Both primary and secondary pediatric hyperoxaluria can present as early as infancy. Paediatric hyperoxaluria has been associated with significant complications including kidney stones, nephrocalcinosis and kidney failure (end stage renal disease or ESRD).
Presently there are no effective treatments for hyperoxaluria in children. The granting of this designation highlights the increasing recognition that kidney stones and other complications of hyperoxaluria are a paediatric problem.
The Orphan Drug Designation Program is administered by the FDA’s Office of Orphan Products Development, which grants orphan status to drugs which are intended to treat rare diseases that affect fewer than 200,000 people in the US, or diseases that affect more than 200,000 people in the US in circumstances where there is not expectation of recovering the costs of developing and marketing a therapeutic drug.
ALLN-177 is an orally-administered, recombinant oxalate-degrading enzyme in development for the chronic management of hyperoxaluria and kidney stones (nephrolithiasis). ALLN-177 targets oxalate in the gastrointestinal tract in an effort to reduce the burden of both dietary and endogenously produced oxalate. ALLN-177 has the potential to decrease the oxalate available systemically for deposition as calcium oxalate crystals or stones in the kidneys, as well as reduce the incidence of calcium oxalate related complications. Effective management of hyperoxaluria could reduce long-term kidney complications, as well as the number of interventions required for the management of kidney stones.
ALLN-177 is currently being tested in two clinical trials in adult patients. A phase 2b, multicenter, randomized, double-blind, placebo-controlled, crossover study is evaluating multiple doses of ALLN-177 in recurrent calcium oxalate kidney stone formers with hyperoxaluria. An additional phase 2 Multicenter, randomized, double-blind, placebo-controlled study is evaluating the safety, tolerability and efficacy of 28 days of treatment with ALLN-177 for reducing urinary oxalate excretion in patients with secondary hyperoxaluria.