FDA approves Lexicon Pharmaceuticals’ Xermelo for carcinoid syndrome diarrhea

Lexicon Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for its oral drug Xermelo (telotristat ethyl) 250mg, to be used in combination with somatostatin analog (SSA) therapy to treat carcinoid syndrome diarrhea in patients with metastatic neuroendocrine tumors (mNETs). This groundbreaking development offers a new hope for patients suffering from this debilitating condition, who will soon have access to the prescription medication in select pharmacies across the US starting next Monday.

Understanding Carcinoid Syndrome Diarrhea

Carcinoid syndrome diarrhea is a challenging and rare condition, often occurring secondary to mNETs. Patients afflicted with this syndrome experience frequent, severe diarrhea, which significantly disrupts their daily lives and ability to maintain normal routines. In addition to diarrhea, symptoms include facial flushing, abdominal pain, fatigue, and over time, damage to heart valves.

Groundbreaking Treatment for a Debilitating Condition

Xermelo’s approval marks a significant advancement in the treatment landscape for carcinoid syndrome diarrhea. Previously, patients with symptoms inadequately controlled by SSA therapy had limited options. “The approval of Xermelo represents a shift in the treatment paradigm of carcinoid syndrome diarrhea for cancer patients,” stated Lonnel Coats, President and CEO of Lexicon Pharmaceuticals. Coats emphasized the company’s commitment to addressing the needs of this patient population, highlighting the drug’s role in enabling patients to lead more predictable lives with fewer incidents of severe diarrhea.

How Xermelo Works

Xermelo targets the excess production of serotonin, a monoamine neurotransmitter that, when overproduced, can cause diarrhea and other side effects. Specifically, Xermelo inhibits the tryptophan hydroxylase enzyme, responsible for this overproduction in mNET cells. This novel approach has demonstrated efficacy in reducing the frequency and severity of diarrhea symptoms in affected patients, significantly improving their quality of life.

Matthew H. Kulke, M.D., TELESTAR primary investigator and director of the Program in Neuroendocrine and Carcinoid Tumors at Dana Farber Cancer Institute, elaborated on the drug’s mechanism: “Inhibition of tumoral serotonin production represents a novel approach for patients with this condition. Studies have shown that Xermelo can reduce the debilitating effects of carcinoid syndrome diarrhea and has a favorable efficacy and safety profile in patients who currently have limited treatment options.”

Global Reach and Collaborative Efforts

While Lexicon retains the rights to market Xermelo in the US and Japan, it has entered into a licensing and collaboration agreement with Ipsen to distribute the drug in Europe and other international markets. This strategic partnership aims to make Xermelo available to a broader audience, maximizing its potential impact on carcinoid syndrome patients worldwide.