Lexicon Pharmaceuticals announced today that the FDA accepted its New Drug Application application for telotristat etiprate.
According to the company website, “The FDA has granted a Priority Review of the NDA filing and set a Prescription Drug User Fee Act (“PDUFA”) target action date of November 30, 2016.”
“The granting of priority review by the FDA underscores the need for improving the lives of the patients and caregivers who live with carcinoid syndrome on a daily basis,” said Lexicon President and Chief Executive Officer, Lonnel Coats. “If approved, telotristat etiprate would be the only approved therapy for patients who are no longer able to control their carcinoid syndrome with the current standard of care alone.”
Carcinoid syndrome is a rare disease affecting thousands of cancer patients with metastatic neuroendocrine tumors (mNETs) that have spread to the liver and other organs from the gastrointestinal tract. The condition is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives, as well as facial flushing, abdominal pain, fatigue and, over time, heart valve damage.
Telotristat etiprate is the first investigational drug in clinical studies to target tryptophan hydroxylase, an enzyme that triggers the excess serotonin production within mNET cells that leads to carcinoid syndrome.”
The current standard of care for carcinoid syndrome is somatostatin analogue therapy given by injectables which reduces the release of serotonin outside of tumor cells.
Telotristat etiprate works in a different way, at the source to reduce serotonin production within the tumor cells. By specifically inhibiting serotonin production, telotristat etiprate seeks to control this important driver of carcinoid syndrome and, in turn, provide patients with more control over their disease.
In August, as the NANETS meeting in Austin, the phase III TELESTAR study was reported. In this study of patients with carcinoid syndrome, telotristat etiprate decreased mean daily bowel movements by 35% among participants (n=135) who received 500 mg of the drug three times a day and 29% among those who took 250 mg three times a day, compared with 17% for individuals who received a placebo. The results, measured from baseline to 12 weeks, were statistically significant
(P <.001). Click here to read more at OncLive.
Telotristat etiprate has received Fast Track and Orphan Drug designation from the U.S. Food and Drug Administration.