http://www.raredr.com/news/fda-approves-xermelo
FDA Approves Xermelo (telotristat ethyl) for Carcinoid Syndrome

James Radke

The FDA has approved Xermelo (telotristat ethyl) for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy.
 
Carcinoid syndrome is a rare and debilitating condition that affects people with metastatic neuroendocrine tumors. It is characterized by frequent and debilitating diarrhea, as well as by facial flushing, abdominal pain, fatigue and possibly heart valve damage.
 
Xermelo targets the overproduction of serotonin inside mNET cells, providing a new treatment option for patients suffering from carcinoid syndrome diarrhea.
 
According to the developers of the drug, Lexicon Pharmaceutics, the treatment is now available by prescription and will be in select specialty pharmacies beginning March 6, 2017.

Pivotal Study

The Phase 3 trial was a global double-blind study which enrolled 135 patients from 12 countries with carcinoid syndrome whose symptoms were not adequately controlled on somatostatin analog therapy (SSA), the current standard of care.
 
The subjects who added telotristat ethyl to SSA therapy at both doses of 250 mg and 500 mg experienced a statistically significant reduction from baseline compared to placebo in the average number of daily bowel movements over the 12-week study period (P < .001).
 
BM frequency reduction ≥ 30% from baseline for ≥ 50% of the double-blind treatment period, were observed in 20%, 44%, and 42% of patients given placebo, telotristat ethyl 250 mg, and telotristat ethyl 500 mg, respectively.
 
Along with the reduced number of bowel movements, telotristat ethyl also showed a significant reduction in the levels of urinary 5-hydroxyindole acetic acid (5-HIAA), from baseline to week 12 with a reduction of 40 mg/24 hours (250 mg arm) and 58 mg/24 hours (500 mg arm) versus an increase of 11 mg/24 hours in the placebo arm (P < .001).  
 
Majority of the patients enrolled (85%) continued to participate in the study and to received 500mg of telotristat ethyl in a 36-week open-label extension.
 
Treatment with telotristat ethyl was well tolerated throughout the trial.

About Carcinoid Syndrome

In the video clips below from 2015, Pablo Lapuerta, M.D., chief medical officer at Lexicon Pharmaceuticals discussed the pathophysiology of carcinoid syndrome as well as the use of telotristat to treat this rare condition.
 

 


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