OxThera Receives Rare Pediatric Disease Designation from U.S. FDA for Oxabact Treatment of Primary Hyperoxaluria
Stockholm – June 26 2020. OxThera AB, a privately-held biopharmaceutical company dedicated to improving the lives of people with Primary Hyperoxaluria (PH), today announced that the U.S. Food and Drug Administration (“FDA”) has granted a Rare Pediatric Disease Designation for Oxabact OC5.
The FDA stated, that based on information provided by OxThera, there is sufficient documentation to demonstrate that nephrocalcinosis, nephrolithiasis, growth failure, and oxalate retinopathy are serious manifestations of PH that primarily affect children. Therefore, Oxabact is eligible for and has been granted a Rare Pediatric Disease Designation for treatment of PH. A “rare pediatric disease” is defined by FDA as a serious or life-threatening disease in which the manifestations primarily affect individuals aged from birth to 18 years and which affects fewer than 200,000 people in the U.S. Subject to FDA approval of Oxabact for the treatment of PH, OxThera would be eligible to receive a voucher that may be redeemed to receive priority review for a subsequent marketing application for a different product candidate, or which could be sold or transferred.
“The FDA’s Rare Pediatric Disease designation for Oxabact for the treatment of PH underlines the agency’s recognition that PH is a serious and potentially fatal condition,” said Matthew Gantz, CEO of OxThera. “Our development program for Oxabact for the treatment of patients with all types of PH is designed to address the unmet need among patients with PH. Our fully enrolled pivotal ePHex study (OC5-DB-02) is on its way to deliver top line results mid 2021, as we continue to work toward our goal of providing a treatment for patients with PH.”
Primary hyperoxaluria is a rare autosomal recessive disorder leading to markedly elevated levels of oxalate in plasma and urine. High levels of oxalate cause kidney damage, driven by the harmful effects of calcium-oxalate crystallization in kidneys and other tissues. If left untreated, the disease can cause kidney and cardiac failure and premature death.
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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 promotes elimination of oxalate, lowering the oxalate burden in the kidneys. Oxabact is a registered trademark of OxThera Intellectual Property AB.
OxThera AB is a Swedish biopharmaceutical company developing a new treatment for primary hyperoxaluria (PH) - a rare genetic and devastating disease with fatal outcomes. Currently pharmaceutical treatment is not available and median age of death is 30, if not treated. A pivotal study of Oxthera's investigational drug candidate Oxabact is ongoing in patients with PH Type 1, 2 and 3 with maintained renal function, and top line data is expected mid- 2021. Oxabact has received orphan drug designation in the US and the EU for the treatment of PH.