FDA Approved Lexicon Pharma’s Xermelo® Against Carcinoid Syndrome Diarrhea

FDA Approved Lexicon Pharma’s Xermelo® Against Carcinoid Syndrome Diarrhea

First-In-Class: Tryptophan Hydroxylase Inhibitor; Orphan Drug XERMELO®  Now Available to Treat Rare and Debilitating Condition Characterized by Frequent, Severe Diarrhea

Source: FDA News and Lexpharma News

March 9, 2017

The U.S. Food and Drug Administration today approved Xermelo (telotristat ethyl) tablets in combination with somatostatin analog (SSA) therapy for the treatment of adults with carcinoid syndrome diarrhea that SSA therapy alone has inadequately controlled.

Carcinoid syndrome is a cluster of symptoms sometimes seen in people with carcinoid tumors. These tumors are rare, and often slow-growing. Most carcinoid tumors are found in the gastrointestinal tract. Carcinoid syndrome occurs in less than 10 percent of patients with carcinoid tumors, usually after the tumor has spread to the liver. The tumors in these patients release excess amounts of the hormone serotonin, resulting in diarrhea. Complications of uncontrolled diarrhea include weight loss, malnutrition, dehydration, and electrolyte imbalance.

“This approval will provide patients whose carcinoid syndrome diarrhea is not adequately controlled with another treatment option,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research.

Xermelo, in a regimen with SSA therapy, is approved in tablet form to be taken orally three times daily with food. Xermelo inhibits the production of serotonin by carcinoid tumors and reduces the frequency of carcinoid syndrome diarrhea.

The safety and efficacy of Xermelo were established in a 12-week, double-blind, placebo-controlled trial in 90 adult participants with well-differentiated metastatic neuroendocrine tumors and carcinoid syndrome diarrhea. These patients were having between four to 12 daily bowel movements despite the use of SSA at a stable dose for at least three months. Participants remained on their SSA treatment, and were randomized to add placebo or treatment with Xermelo three times daily. Those receiving Xermelo added on to their SSA treatment experienced a greater reduction in average bowel movement frequency than those on SSA and placebo. Specifically, 33 percent of participants randomized to add Xermelo on to SSA experienced an average reduction of two bowel movements per day compared to 4 percent of patients randomized to add placebo on to SSA.

The most common side effects of Xermelo include nausea, headache, increased levels of the liver enzyme gamma-glutamyl transferase, depression, accumulation of fluid causing swelling (peripheral edema), flatulence, decreased appetite and fever. Xermelo may cause constipation, and the risk of developing constipation may be increased in patients whose bowel movement frequency is less than four bowel movements per day. Patients treated with a higher than recommended dosage of Xermelo developed severe constipation in clinical trials. One patient required hospitalization and two other patients developed complications of either intestinal perforation or intestinal obstruction. Patients should be monitored for severe constipation. If a patient experiences severe constipation or severe, persistent or worsening abdominal pain, they should discontinue Xermelo and contact their healthcare provider.

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Xermelo is manufactured by Woodlands, Texas-based Lexicon Pharmaceuticals, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

About XERMELO™

Discovered using Lexicon’s unique approach to gene science, XERMELO is the first and only approved oral therapy for carcinoid syndrome diarrhea. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within mNET cells. 

Lexicon has built the in-house capability and infrastructure to launch and market XERMELO in the U.S., where it retains all commercialization rights.  Lexicon also retains rights to market telotristat ethyl in Japan.  Lexicon has established a license and collaboration agreement with Ipsen to commercialize telotristat ethyl in Europe and other countries outside of U.S. and Japan.  For more information about XERMELO, please visit www.xermelo.com. 

XERMELO™ Important Safety Information 

  • Warnings and Precautions: XERMELO may cause constipation which can be serious. Monitor for signs and symptoms of constipation and/or severe, persistent, or worsening abdominal pain in patients taking XERMELO. Discontinue XERMELO if severe constipation or severe persistent or worsening abdominal pain develops.
  • Adverse Reactions: The most common adverse reactions (≥5%) include nausea, headache, increased GGT, depression, peripheral edema, flatulence, decreased appetite, and pyrexia.
  • Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. 

For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.

Lexicon Conference Call 

Lexicon management will hold a conference call to discuss the approval of XERMELO at 5:00 pm Eastern Time today, February 28, 2017.  The dial-in number for the conference call is 888-645-5785 (within the US/Canada) or 970-300-1531 (international).  The conference ID for all callers is 78166536.  Investors can access a live webcast of the call at www.lexpharma.com.  An archived version of the webcast will be available on the website through March 31, 2017.

About Lexicon

Lexicon is a fully integrated biopharmaceutical company that is applying a unique approach to gene science based on Nobel Prize-winning technology to discover and develop precise medicines for patients with serious, chronic conditions. Through its Genome5000™ program, Lexicon scientists have studied the role and function of nearly 5,000 genes over the last 20 years and have identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to safely and effectively treat disease. In addition to XERMELO, Lexicon has a pipeline of promising drug candidates in clinical and pre-clinical development in diabetes and metabolism and neuropathic pain. For additional information please visit www.lexpharma.com.

In an effort to ensure all appropriate patients have fast access to affordable treatment, Lexicon offers LexCares—a comprehensive program designed to guide patients through each step of their treatment journey. Through LexCares, patients will have access to financial assistance programs that may reduce or eliminate out-of-pocket costs associated with their XERMELO prescription. For additional information about the LexCares program, visit www.xermelo.com/lexcares. 

Safe Harbor Statement

This press release contains “forward‑looking statements,” including statements relating to the safety and efficacy and the therapeutic and commercial potential of XERMELO (telotristat ethyl) 250 mg. In addition, this press release also contains forward looking statements relating to Lexicon’s growth and future operating results, discovery and development of products, strategic alliances and intellectual property, as well as other matters that are not historical facts or information. All forward‑looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the degree of market acceptance of XERMELO, the availability of coverage and reimbursement for XERMELO, Lexicon’s dependence on third parties for manufacturing and distribution of XERMELO, Lexicon’s compliance with applicable legal and regulatory requirements and other factors relating to the commercialization of XERMELO. Other risks include Lexicon’s ability to meet its capital requirements, successfully conduct preclinical and clinical development and obtain necessary regulatory approvals of its other potential drug candidates, achieve its operational objectives, obtain patent protection for its discoveries and establish strategic alliances, as well as additional factors relating to manufacturing, intellectual property rights, and the therapeutic or commercial value of its drug candidates. Any of these risks, uncertainties and other factors may cause Lexicon’s actual results to be materially different from any future results expressed or implied by such forward‑looking statements. Information identifying such important factors is contained under “Risk Factors” in Lexicon’s annual report on Form 10‑K for the year ended December 31, 2015, as filed with the Securities and Exchange Commission. Lexicon undertakes no obligation to update or revise any such forward‑looking statements, whether as a result of new information, future events or otherwise.

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