A company currently embroiled in a lawsuit from a health insurance company got some better news Thursday as it reported positive data from a Phase III study of a drug in a rare digestive system disease.
Staines-upon-Thames, U.K.-based Mallinckrodt said top-line data from the CONFIRM study of terlipressin among 300 patients with hepatorenal syndrome type 1, or HRS-1, met its primary endpoint of disease reversal, showing statistically significant superiority for the drug over placebo. The company plans to apply for Food and Drug Administration approval of the drug early next year.
The HRS-1 reversal endpoint encompassed three components: renal function improvement, avoidance of dialysis and short-term survival. Verified HRS reversal and percentage of patients who were viable for inclusion in the primary endpoint analysis were co-primary endpoints, according to the study’s ClinicalTrials.gov page.
HRS-1 is a rare and life-threatening disease that causes complications of liver disease that can lead to kidney failure. Currently, the disease – which affects an estimated 30,000-40,000 patients each year – has no approved drug therapies in the U.S. or Canada. The study took place at 64 U.S. and Canadian sites.
“The study met nearly all of the pre-specified secondary endpoints,” lead investigator and Virginia Commonwealth University professor of medicine Dr. Arun Sanyal said in a statement. “HRS-1 is a life-threatening disease that is extremely difficult to treat.”
According to the study page, secondary endpoints included incidence of participants with HRS reversal; durability of HRS reversal; incidence of HRS reversal in the systemic inflammatory response syndrome subgroup; and incidence of verified HRS reversal without recurrence after 30 days.
The news comes the week after health insurance company Humana filed a lawsuit in a federal court in California against Mallinckrodt, alleging that the drugmaker engaged in a price-gouging scheme for the injectable drug H.P. Acthar Gel, a drug on the market since 1952. Mallinckrodt has rejected the claims in the lawsuit, calling them “completely without merit.”
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