April 22, 2020: “Mallinckrodt plc, a global biopharmaceutical company announced that the U.S.FDA has accepted for review the company’s New Drug Application (NDA) for terlipressin, an investigational agent being evaluated for the treatment of hepatorenal syndrome type 1 (HRS-1).
On March 17, 2020 the company announced the completion of its rolling submission of the NDA for terlipressin. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target date of September 12, 2020.
HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis. The condition has a median survival time of approximately two weeks and greater than 80 percent mortality within three months if left untreated. At present, there are no approved drug therapies for HRS-1 in the U.S., and it is estimated to affect between 30,000 and 40,000 patients in the U.S. annually.
“We are pleased that we received filing acceptance of our NDA submission. This is an important milestone in our clinical development program for terlipressin to address a critically high unmet need for patients with HRS-1 and their physicians, who historically have had limited treatment options,” said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. “We look forward to working with the agency during their review of our regulatory package.”
In 2005, terlipressin was granted Fast Track designation by the FDA, which provides for expedited review to facilitate development of drugs intended to treat serious or life-threatening conditions and fill an unmet medical need.
In 2016, Mallinckrodt and the FDA reached agreement on the Phase 3 CONFIRM trial protocol design and data analysis under the agency’s special protocol assessment (SPA) process.
A SPA is an advance agreement with the FDA for the acceptability of the clinical design, endpoints and statistical data analyses for a Phase 3 trial before the start of the trial. The submission is a Class 2 resubmission. Mallinckrodt anticipates an FDA Advisory Committee meeting to discuss the submission later this year.
The terlipressin NDA is based, in part, on results from the Phase 3 CONFIRM trial, which was the largest-ever prospective study (n=300) conducted in patients with HRS-1, and the culmination of a sustained, 17-year effort to develop terlipressin for potential use in the U.S. and Canada.
Initial results were presented at The Liver Meeting® 2019, the annual meeting of the American Association for the Study of Liver Diseases (AASLD).
Terlipressin is a potent vasopressin analogue selective for V1 receptors being investigated for the treatment of HRS-1 in the U.S. and Canada. It is an investigational product in these countries as the safety and efficacy have not been established with, nor has approval been granted by, regulatory authorities in either country. Terlipressin is approved for use outside the U.S. and Canada.”
http://www.mallinckrodt.com/about/news-and-media/news-detail/?id=26706
