The drug improves kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function.
The FDA approved injectable terlipressin (Terlivaz; Mallinckrodt plc) for hepatorenal syndrome (HRS) with rapid reduction in kidney function, making terlipressin the first and only FDA-approved treatment for adults with HRS and rapidly reduced kidney function.
In the United States, between 30,000 and 40,000 adults have HRS with rapid reduction in kidney function; it is a condition that occurs in patients with advanced liver disease. Left untreated, the median survival time is 2 weeks, and more than 80% of patients die within 3 months.
"Diagnosing and treating HRS can be challenging, and every minute counts when managing patients who have it,” said Steven Romano, MD, executive vice president and chief scientific officer at Mallinckrodt, in a press release.
The phase 3 CONFIRM trial evaluated the safety and efficacy of terlipression for HRS type 1 patients in the United States and Canada. The largest prospective study using terlipressin in HRS involving rapid reduction in kidney function, the trial met the primary endpoint of renal function improvement (Verified HRS Reversal) without using dialysis and short-term survival.
During the study, an improvement in renal function was measured using consecutive serum creatinine (SCr) values. Patients needed to have 2 SCR values that were less than 1.5 mg/dL and 2 hours apart by day 14 or at discharge. Efficacy was measured as being alive without dialysis for 10 days following the SCr-based goal of Verified HRS Reversal.
HRS involving rapid reduction in kidney function is an acute and life-threatening disease that can be broken down into 2 types, with the first type being more severe. It can incite acute renal failure and usually hospitalizes the patient. The second type of HRS involving rapid reduction in kidney function is a chronic disease that worsens to kidney failure over the coming weeks or months.
Among patients who reported adverse events, 19.5% experienced abdominal pain and 16% nausea. Additionally, 15.5% of patients reported respiratory failure, 13% diarrhea, and 12.5% reported dyspnea.
While terlipressin has been approved outside the US for more than 30 years, it is now being recommended by the American Association for the Study of Liver Diseases guidance and the American College of Gastroenterology guidelines.
“[Terlipressin] gives US physicians the first FDA-approved option for treating HRS patients with rapid reduction in kidney function that may help them improve kidney function and lessen the associated need for renal replacement therapy, such as dialysis," Romano said in the press release.
Terlipressin will launch within the coming weeks, according to Siggi Olafsson, president and chief executive officer of Mallinckrodt, in the press release.
"The FDA approval of Terlivaz is a significant milestone for Mallinckrodt as it brings an important treatment option to these critically ill patients requiring hospitalization and to US physicians who historically have had limited treatment interventions,” Olafsson added in the press release.
Mallinckrodt Pharmaceuticals. Mallinckrodt Receives U.S. FDA Approval for Terlivaz® (terlipressin) for injection for the Treatment of Hepatorenal Syndrome (HRS). Mallinckrodt website. September 14, 2022. Accessed on September 15, 2022. https://www.mallinckrodt.com/about/news-and-media/news-detail/?id=29401