Doptelet (avatrombopag)

One of 4 TPO receptor agonist products for ITP, alongside eltrombopag (Promacta and its generics), Alvaiz and the weekly injection Nplate. It is taken with food rather than apart from it, which some patients find easier than eltrombopag's calcium timing rule.

Platelets are made by large cells in the bone marrow called megakaryocytes, and the body's signal to make more of them is a hormone called thrombopoietin (TPO). Avatrombopag attaches to the TPO receptor on those cells and switches on the same signal, so the marrow makes more platelets. In ITP the immune system destroys platelets faster than normal; avatrombopag does not stop that attack, it raises production enough to keep the count at a safer level, usually 50,000 per microliter or more.

Doptelet has no boxed warning and no contraindications. Its main warning is blood clots: TPO receptor agonists have been linked to clots in veins and arteries, and in the adult ITP studies 7% of patients on Doptelet (9 of 128) had a clot event. People with a history of clots or an inherited clotting condition such as Factor V Leiden need extra caution. The most common side effects in adults with ITP, against placebo, were headache (31% against 14%), tiredness (28% against 9%), bruising (26% against 18%), nosebleed (19% against 18%) and upper respiratory infection (15% against 5%). In children the most common were viral infections (20% against 5%), runny or stuffy nose (20% against 10%), cough (17% against 0%) and fever (17% against 0%). Animal studies suggest it may harm an unborn baby, and the patient information says not to breastfeed during treatment and for at least 2 weeks after the last dose.

Brand-only oral TPO receptor agonist; the FDA lists no generic avatrombopag. Cost to patients depends mainly on insurance and copay programs.

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Approved for adults with chronic ITP and children 1 year and older with persistent or chronic ITP, in each case after an insufficient response to a previous treatment. Doptelet should not be used to bring platelets to a normal level.

Source: Doptelet Copay Assistance Program (Sobi)

Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.

In the adult Phase 3 trial (NCT01438840), 49 adults with chronic ITP and platelet counts under 30,000 were randomized 2 to 1 to Doptelet or placebo for 6 months. Patients on Doptelet had a median of 12.4 weeks with platelets at 50,000 or higher without rescue treatment, against 0 weeks on placebo, and 66% reached that level by day 8 against none on placebo. In the pediatric trial (NCT04516967), 75 children aged 1 to 17 whose ITP had not responded well enough to earlier treatment were randomized 3 to 1. After 12 weeks, 27.8% on Doptelet had a durable platelet response against none on placebo; most had already tried another TPO receptor agonist.

The FDA first approved Doptelet on May 21, 2018 for adults with chronic liver disease and low platelets before a procedure. Approval for adults with chronic ITP followed on June 26, 2019. On July 24, 2025, the FDA approved its use in children 1 year and older with persistent or chronic ITP, along with Doptelet Sprinkle, a granule form for children under 6. The drug is made by AkaRx, a Sobi company.

Steroids and IVIG are usually tried first. When they are not enough, options include the TPO receptor agonists Nplate (romiplostim), Promacta and generic eltrombopag, Alvaiz and Doptelet, which raise platelet production; Tavalisse (fostamatinib) and Wayrilz (rilzabrutinib), which slow platelet destruction; and rituximab or spleen removal. Trials test FcRn blockers and other approaches.