Generic Name: Avatrombopag
Brand Name: Doptelet
Manufacturer: AkaRx Inc
General Information & indications:
The U.S. Food and Drug Administration on
21st May, 2018 approved DOPTELET (AVATROMBOPAG) tablets to
treat low blood platelet count (Thrombocytopenia) in adults with chronic
liver disease who are scheduled to undergo a medical or dental procedure.
This is the first drug approved by the FDA for this use.
Patients with chronic liver
disease who have low platelet counts and require a procedure are at increased
risk of bleeding. “Doptelet” was demonstrated to safely increase the platelet
count. This drug may decrease or eliminate the need for platelet transfusions,
which are associated with risk of infection and other adverse reactions.
Platelets (thrombocytes) are
colorless cells produced in the bone marrow that help form blood clots in the
vascular system and prevent bleeding. Thrombocytopenia is a condition in which
there is a lower-than-normal number of circulating platelets in the blood. When
patients have moderately to severely reduced platelet counts, serious or
life-threatening bleeding can occur, especially during invasive procedures.
Patients with significant thrombocytopenia typically receive platelet
transfusions immediately prior to a procedure to increase the platelet count.
Clinical trials
information:
The safety and efficacy of Doptelet was studied in two trials
(ADAPT-1 and ADAPT-2) involving 435 patients with chronic liver disease and
severe thrombocytopenia who were scheduled to undergo a procedure that would
typically require platelet transfusion. The trials investigated two dose levels
of Doptelet administered orally over five days as compared to placebo (no
treatment). The trial results showed that for both dose levels of Doptelet, a
higher proportion of patients had increased platelet counts and did not require
platelet transfusion or any rescue therapy on the day of the procedure and up
to seven days following the procedure as compared to those treated with
placebo.
Tablets: 20 mg avatrombopag as round, biconvex, yellow,
film-coated tablets debossed with “AVA” on one side and “20” on the other side.
Mechanism of action:
Avatrombopag
is an orally bioavailable, small molecule TPO receptor agonist that stimulates
proliferation and differentiation of megakaryocytes from bone marrow progenitor
cells resulting in an increased production of platelets. Avatrombopag does not
compete with TPO for binding to the TPO receptor and has an additive effect with
TPO on platelet production.
Adverse effects:
The most common side effects reported by clinical trial
participants who received Doptelet were fever, stomach (abdominal) pain,
nausea, headache, fatigue and swelling in the hands or feet (edema). People
with chronic liver disease and people with certain blood clotting conditions
may have an increased risk of developing blood clots when taking Doptelet.
Type of approval:
This product was granted Priority
Review, under which the FDA’s goal is to take action on an
application within six months where the agency determines that the drug, if
approved, would significantly improve the safety or effectiveness of treating,
diagnosing or preventing a serious condition.
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