OxThera AB announced that its product Oxabact has been granted an Orphan Drug Designation in the European Union for treatment of short bowel syndrome (SBS).
SBS is a highly disabling malabsorptive condition. SBS is associated with significant morbidity and mortality, reduced quality of life and high healthcare costs.
"We are happy to announce that Oxabact is now also recognised as a potential treatment for short bowel syndrome. OxThera believe that Oxabact would be an excellent add-on therapy in SBS patients and would help the underlying gastritis and malabsorption in the gut, as well as subsequently reducing plasma oxalate and preventing kidney disease," said Elisabeth Lindner, chief executive officer, of OxThera.
EMA/Committee on Orphan Medicinal Products (COMP) considered that OxThera had provided data from preclinical models and preliminary clinical data, suggesting favourable effects of Oxabact in SBS. The Committee considered that this may translate into clinically relevant benefit for patients affected by the condition.
Oxabact is an oral product composed of highly concentrated live bacteria (Oxalobacter formigenes). OxThera is currently pursuing a complete clinical development programme in the EU and in the US for the treatment of patients suffering from Primary Hyperoxaluria.
Oxabact previously holds orphan drug designations in the EU and the US for the treatment of Primary Hyperoxaluria.
Short bowel syndrome is an intestinal failure and is characterised by diarrhoea, nutrient malabsorption, bowel dilation and dysmobility. The mean prevalence is estimated at about 0.39 in 10,000 in the European population, translating to almost 20,000 patients. Approximately 15,000 people in the US have Short Bowel Syndrome.
Secondary Hyperoxaluria results from excess intake, malabsorption or malsecretion of oxalate. The disorder may cause recurrent kidney stones. An estimated four million people in the US suffer from kidney stone disease.
Primary Hyperoxaluria is a rare inborn autosomal genetic disorder leading to markedly elevated levels of endogenous oxalate in plasma and urine. High levels of urinary oxalate cause kidney damage, including recurrent kidney stone formation and/or calcification of the kidney.