FDA: More data needed to approve terlipressin NDA for hepatorenal syndrome type 1
Although the FDA accepted a new drug application for terlipressin to treat hepatorenal syndrome type 1, the agency has issued a complete response letter to Mallinckrodt plc stating that more data are required before the NDA can be approved.
More specifically, according to a company press release, the complete response letter indicated the FDA seeks more information regarding a positive risk-benefit profile of the drug. Therefore, the FDA will not approve the NDA in its “current form.”
The release further noted that terlipressin is approved in many other countries outside the United States and has been “a standard of care for decades in the treatment of patients with [hepatorenal syndrome type 1] HRS-1.”
Granted fast track designation in 2005, the Cardiovascular and Renal Drugs Advisory Committee of the FDA more recently voted to recommend approval of terlipressin to treat adults with HRS-1 in July of 2020. According to the release, this recommendation was based in part on positive results from Mallinckrodt’s long-term CONFIRM study, which were presented at the 2019 Liver Meeting (the annual meeting of the American Association for the Study of Liver Diseases).
“While we are disappointed that the FDA issued a complete response letter for terlipressin, we remain confident in the strength of the data from our phase 3 CONFIRM study, which is the largest clinical trial ever conducted in this rare condition,” Steven Romano, MD, executive vice president and chief scientific officer at Mallinckrodt, said in the release. “HRS-1 is a complex disease that affects a critically ill patient population with no approved treatment in the U.S. at present. We are surprised by and disagree with the FDA’s decision and remain committed to pursuing all available options as we continue working with the FDA toward approval of terlipressin in order to help address this difficult and life-threatening syndrome.”