Lexicon Pharmaceuticals has submitted a New Drug Application to the FDA seeking approval for the marketing and sale of telotristat etiprate, an oral drug for the treatment of carcinoid syndrome. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing. Lexicon has requested a Priority Review by the FDA as part of the NDA filing.
"The filing of the NDA for telotristat etiprate brings us one step closer to the possibility of bringing this innovative new investigational treatment to the market to improve the lives of the community of patients and caregivers who live with carcinoid syndrome on a daily basis," said Lexicon President and Chief Executive Officer Lonnel Coats. "We look forward to working closely with the FDA during the review process."
The NDA filing is supported by the results from TELESTAR, a pivotal phase III clinical trial of telotristat etiprate in patients with carcinoid syndrome, and TELECAST, a phase III companion study to TELESTAR. Results from TELESTAR demonstrated a statistically significant reduction from baseline compared to placebo in the average number of daily bowel movements over the 12-week study period (p<0.001), meeting the study's primary endpoint. Top-line results from TELECAST demonstrated a statistically significant reduction in urinary 5-hydroxyindoleacetic acid (5-HIAA, the main metabolite of serotonin) at week 12 (p<0.001) as compared to placebo, meeting that study's primary endpoint. The proportions of patients with treatment-emergent adverse events, serious adverse events and discontinuation due to adverse events were generally similar between the telotristat etiprate and placebo arms in both studies.
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.
Discovered using Lexicon's unique approach to gene science, 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. While existing treatments for carcinoid syndrome work to reduce the release of serotonin outside tumor cells, telotristat etiprate works 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.
Telotristat etiprate has received Fast Track and Orphan Drug designation from the FDA.
Lexicon retains rights to market telotristat etiprate in the U.S. and Japan, and is building the in-house commercial infrastructure to serve the U.S. market. Lexicon has a license and collaboration agreement with Ipsen to commercialize telotristat etiprate in Europe and other countries outside the U.S. and Japan.