OxThera receives positive decision from EMA on Paediatric Investigational Plan (PIP) for treatment of primary hyperoxaluria with Oxalobacter formigenes
STOCKHOLM, SWEDEN – August 23, 2019. OxThera AB, a privately-held biopharmaceutical company dedicated to improving the lives of people with Primary Hyperoxaluria (PH), today announced that, based on a positive opinion from the Paediatric Committee (PDCO), the EMA has agreed to the proposed PIP for Oxalobacter formigenes in the treatment of primary hyperoxaluria.
“We are pleased to announce this important regulatory milestone. The approval of the Paediatric Investigational Plan provides us with a clear development path for Oxabact in the treatment of paediatric patients with primary hyperoxaluria”, said Matthew Gantz, CEO of OxThera.
Primary Hyperoxaluria is a rare autosomal recessive disorder leading to markedly elevated levels of endogenous oxalate in plasma and urine. High levels of oxalate cause kidney damage, primarily driven by crystallization of oxalate in tissues and in the kidney and can cause kidney failure and premature death.
A pivotal phase 3 study, evaluating the effect of Oxabact in PH patients is currently ongoing. This double-blind, placebo-controlled trial is being conducted at ten clinical sites in US and Europe. The study is planned to be fully recruited before end of the year. The Phase 3 study is monitoring change in plasma oxalate and change in estimated glomerular filtration rate (eGFR) over 12 months, and will, together with the follow-up study, evaluate the clinical benefit from treatment with Oxabact®.
Data from a 24-month interim analysis of the Company’s ongoing open-label phase 2 extension study in PH-patients on a stable dialysis regimen, has previously shown a consistent and long-lasting reduction of plasma oxalate together with a clinical improvement in cardiac function.
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Matthew Gantz, CEO
Oxabact is a bi-modal enteric biotherapy containing a lyophilized formulation of Oxalobacter formigenes, a non-pathogenic, oxalate-degrading commensal bacterium. Oxabact is administered orally as a coated capsule. By promoting active and passive secretion of oxalate from the plasma into the gut, Oxabact potentials elimination of oxalate via the gut, lowering the oxalate burden in the kidneys.
OxThera AB is a Swedish biotech company developing a new treatment for primary hyperoxaluria - a rare genetic and devastating disease with fatal outcomes. Currently pharmaceutical treatment is not available and median age of death is 30. A phase 3 study of Oxthera's drug candidate Oxabact® is ongoing, and an application for registration is expected to be submitted in the second half of 2021. Oxabact® has received orphan drug status in the US and the EU.