- Tuesday was Rare Disease Day 2017, a particularly apt time for the Food and Drug Administration to approve Lexicon Pharmaceuticals' drug for carcinoid syndrome symptoms.
- The syndrome occurs when carcinoid tumors, a type of neuroendocrine cancer that has powerful effects on hormones, spread throughout the body. One common byproduct of this, and the one Lexicon's drug Xermelo (telotristat ethyl) aims to treat, is severe diarrhea.
- The FDA approved Xermelo in combination with an analog of somatostatin, a chemical that inhibits secretion in the gastrointestinal system. A 28-day regimen of the Lexicon's drug will carry a wholesale acquisition cost of $5,164, which the company said is on-par with the price of the leading carcinoid syndrome diarrhea treatment: Novartis' Sandostatin LAR (octreotide acetate).
Even after losing patent exclusivity, Sandostatin LAR garnered $1.6 billion for Novartis in 2016. Lexicon has predicted the product, which boasts an orphan drug designation and the title of first orally-administered medication for carcinoid syndrome diarrhea, will achieve peak annual sales of $350 million.
"As we've looked at the market, we've been out there for a while, and we understand the energy around this now-approved innovation," Lexicon's CEO Lonnel Coats said during a Feb. 28 investor call. "We feel pretty confident about the forecast we've given, so we're going to stand by that at this moment."
Lexicon has already had "good conversations" with healthcare insurers and isn't particularly concerned about coverage for its drug. A bigger challenge will be informing patients about Xermelo and getting physicians to prescribe it.
"The biggest barrier I think we'll be fighting will not be as much with the payers as it will be of trying to change habit of what physicians may be used to doing, and, given that they have had no new technology in so long, you see a lot of polypharmacy out in the marketplace," Coats said in the call. "The rate limiting step for us is how quickly can we begin to change and mold people's habits about how best to use Xermelo."
According to the American Society of Clinical Oncology and the National Organization for Rare Disorders, about 12,000 people are given a carcinoid tumor diagnosis each year, with about 10% of them developing carcinoid syndrome.