[Ip-health] FDA approves ivosidenib/Tibsovo, a $870 per day leukemia drug from Agios

James Love james.love at keionline.org
Mon Jul 23 08:59:55 PDT 2018

This is a ​Stat article about a ​
$870 PER
​ DAY​
​drug for  Acute Myeloid Leukemia
.   The average duration of therapy is four month
, according to the article but for some patients,
​apparently ​
​, since that is an average​
​ The drug targets an
 IDH1 mutation. ​

​The FDA press release said that just
174 patient
​s were the single arm trial used to evaluate the efficacy of the
treatment. That would make the trial costs pretty small.  Stat puts the US
patient population at 700 to 1,100 persons, based upon the initial label.
  If a 1,000 patents received the drug and paid $100,000 per treatment on
average, that would be $100 million, per year, I assume, from the US
market, for this indication.

​ClinicalTrials.Gov actually lists six trials for the drug,  Five have
start dates, including 3 in 2017 and 2 in 2018.
So this was a pretty quick turn around.

Only two the of trails are listed as finished.  One Trial has a start date
of July 31, 208, which is next week, and 2 of the trials are still

The company Agios Pharmaceuticals, received 2 NIH grants  "t
o screen for chemical inhibitors that are specific for the mutant form of
isocitrate dehydrogenase 1 (IDH1)".
​  The FDA Orange Book does not yet have a listing for the drugs or the
patents asserted. ​


The drug, ivosidenib
​[Brand name Tibsovo]​
— developed by Cambridge-based Agios Pharmaceuticals— is a once-daily pill
that might replace chemotherapy for some people with refractory or
relapsing AML, a type of blood cancer that forms in the bone marrow.


The efficacy of Tibsovo was studied in a single-arm trial of 174 adult
patients with relapsed or refractory AML with an
IDH1 mutation. The trial measured the percentage of patients with no
evidence of disease and full recovery of blood counts after treatment
(complete remission or CR), as well as patients with no evidence of disease
and partial recovery of blood counts after treatment (complete remission
with partial hematologic recovery or CRh). With a median follow-up of 8.3
months, 32.8 percent of patients experienced a CR orCRh that lasted a
median 8.2 months. Of the 110 patients who required transfusions of blood
or platelets due to AML at the start of the study, 37 percent went at least
56 days without requiring a transfusion after treatment with Tibsovo.

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