Telotristat Ethyl is an extremely significant introduction to the treatment of Carcinoid Syndrome as it would be the first addition to the standard of care in more than 16 years and the first time an oral syndrome treatment has been developed. The drug was previously known as Telotristat Etiprate but was changed to Ethyl in Oct 2016. Etiprate was previously a truncation of ethyl hippurate.
Who is the drug for?
The drug may be of benefit to those whose carcinoid syndrome is not adequately controlled by somatostatin analogues (Octreotide/Lanreotide). It doesn’t replace somatostatin analogues – it is an additional treatment.
When is it due in service?
The US FDA’s decision to approve the drug is anticipated by 28 February 2017 (previously 30 November 2016). The European Medicines Agency has also accepted the filing for Telotristat Ethyl as an adjunct (i.e. in addition) to somatostatin analogue therapy for the long-term treatment of carcinoid syndrome.
In addition to the European submission, Ipsen will pursue a worldwide regulatory plan for marketing authorisation submissions in the territories in which it operates. Once approved, Ipsen will be distributing the drug in all countries less USA and Japan where Lexicon retains the rights. The approval estimate outside USA is not yet clear but my best guess is that the EMA approval should be later in 2017 after the US FDA approval and then subject to local regulations for achess to treatment and reimbursement. Outside USA and Europe will be constrained by national approval timelines.
How does it work?
In the simplest of terms, the drug is an inhibitor of the enzyme tryptophan hydroxylase (TPH). TPH is the rate-limiting enzyme in serotonin synthesis which converts tryptophan (an essential amino acid which comes from diet) to 5-hydroxytryptophan, which is subsequently converted to serotonin, one of the main causes of carcinoid syndrome effects including carcinoid heart disease. The trial data indicates that Telotristat ethyl significantly reduced the frequency of bowel movements. Furthermore it was also associated with “significantly reduced levels of urinary 5-HIAA“, a marker for systemic serotonin levels, which are typically elevated in severe carcinoid syndrome.
Resources for your perusal:
- You can read more about the trial data in a summary by Dr Matthew Kulke (Dana Farber) by CLICKING HERE (latest review from 2017 ASCO).
- There is also an excellent summary in video form by Dr Lowell Anthony (University of Kentucky) by CLICKING HERE. (“any reduction in diarrhea is meaningful“).
- The 2016 ENETS conference also indicated a knock on effect of reducing the risk of, and perhaps even halting Carcinoid Heart Disease. You can read about this by CLICKING HERE.
- An excellent technical description of the drug can be found by CLICKING HERE.
- The detailed output from the trial (results) can be found by CLICKING HERE.
- Great 2016 article from ASCO (American Society of Clinical Oncologists) can be found by CLICKING HERE.
I know some patients who participated in the trial and they seem to be positive about the drug – if anyone can make a comment, that might be helpful to others who are thinking this new drug might be useful for them when it becomes available.
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