Generic Eltrombopag Is Now Available for Severe Aplastic Anemia and Immune Thrombocytopenia

Severe Aplastic Anemia
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Generic, AB-rated formulations of eltrombopag (Promacta) as an oral suspension and a tablet are now commercially available for the treatment of select patients with severe aplastic anemia and immune thrombocytopenia.1,2

Specifically, the generic oral suspension and tablet formulations of eltrombopag are approved in the following indications:

• For patients with severe aplastic anemia who are insufficiently responsive to immunosuppressive therapy
• For patients 1 year of age and older with persistent or chronic immune thrombocytopenia that is insufficiently responsive to other treatments and who have an increased risk of bleeding
• For the treatment of thrombocytopenia in patients with chronic hepatitis C whose clinical condition prevents or limits initiation/maintenance of interferon-based therapy

Eltrombopag was initially approved by the FDA in November 2008 for the immune thrombocytopenia indication.3 In August 2014, the regulatory agency approved eltrombopag for the treatment for patients with severe aplastic anemiafollowing an insufficient response to immunosuppressive therapy; the label was expanded in November 2018, allowing for eltrombopag to be administered in combination with standard immunosuppressive therapy in adult and pediatric patients 2 years of age and older with newly diagnosed severe aplastic anemia.4,5

Additionally, through a copayer program with Camber Pharmaceuticals—the developer of the generic formulations of eltrombopag—some patients could be eligible to receive the generic agent for no out-of-pocket cost.1

“For years, many patients have struggled to afford this life-saving therapy,” Kon Ostaficiuk, president of Camber Pharmaceuticals, stated in a news release. “We are excited to offer this important medication with a copay assistance program.”

The generic version of eltrombopag is available as an oral suspension in cartons featuring 30 unit-dose packets at 12.5 mg and 25 mg. It is also available as a tablet at doses of 12.5 mg, 25 mg, 50 mg, and 75 mg.

Prior Clinical Data for Eltrombopag in Persistent or Chronic Immune Thrombocytopenia

The initial approval of eltrombopag for adult patients with persistent or chronic immune thrombocytopenia was supported by data from the phase 2 Study TRA100773B/Study TRA100773A (NCT00102739) and phase 3 RAISE (NCT00370331) trial.6 The pediatric indication was based on findings from the phase 2 PETIT (NCT00908037) and phase 3 PETIT2 (NCT01520909) trials.

Study TRA100773B/Study TRA100773A included patients who had completed at least 1 prior line of therapy for immune thrombocytopenia and had a platelet count less than 30 x 109/L. In TRA100773B, patients treated with eltrombopag at 50 mg per day (n = 73) achieved a platelet count response rate of 59% compared with 16% for patients treated with placebo (n = 37). In Study TRA100773A, these respective rates were 70% for eltrombopag (n = 27) and 11% for placebo (n = 27).

Findings from RAISE showed that in patients treated with eltrombopag (n = 135), the mean time with a platelet count of at least 50 x 109/L was 11.3 weeks compared with 2.4 weeks for patients treated with placebo (n = 62). The respective rates of patients who required salvage therapy were 18% and 40%.

In the PETIT trial, patients with relapsed/refractory immune thrombocytopenia following at least 1 line of therapy who had a platelet count less than 30 x 109/L experienced a platelet count response rate of 62% when treated with eltrombopag (n = 45) vs 32% when treated with placebo (n = 22).

The same patient population was evaluated in PETIT2, and those treated with eltrombopag (n = 63) achieved a platelet count response rate of 41% compared with 3% for those given placebo (n = 29).

Data for Eltrombopag in Severe Aplastic Anemia

Eltrombopag was approved for use in patients with refractory severe aplastic anemia based on data from the phase 2 Study ETB115AUS28T (NCT00922883). The trial included patients with severe aplastic anemia who had an insufficient response to at least 1 prior immunosuppressive therapy and a platelet count of no more than 30 x 109/L.

Patients treated with eltrombopag during the single-arm, single-center, open-label trial (n = 43) achieved a hematologic response rate of 40% (95% CI, 25%-56%). The median duration of response was not reached (NR; 95% CI, 3.0-NR).

Eltrombopag Safety Information

The most common adverse effects across all indications for eltrombopag that were reported in at least 20% of patients in any indication included anemia, nausea, pyrexia, increased alanine aminotransferase levels, cough, fatigue, headache, and diarrhea.

The prescribing information for the agent includes warnings and precautions regarding hepatotoxicity; an increased risk of death or progression of myelodysplastic syndromes to acute myeloid leukemia; and thrombotic/thromboembolic complications. For hepatotoxicity, it is recommended that liver function be monitored before and during treatment. Regarding thrombotic/thromboembolic complications, patients with chronic liver disease have experienced portal vein thrombosis during treatment with eltrombopag; it is recommended that platelet counts are regularly monitored. The prescribing information also warns that the combination of eltrombopag, interferon, and ribavirin may increase the risk of hepatic decompensation in patients with chronic hepatitis C.

References

1. Camber Pharmaceuticals Linkedin. Camber Pharmaceuticals is proud to announce the launch of Eltrombopag, the first AB-rated generic alternative. Posted May 14, 2025. Accessed May 14, 2025. https://www.linkedin.com/posts/camber-pharmaceuticals_camber-pharmaceuticals-is-proud-to-announce-activity-7328407491480809473-ug0C
2. Eltrombopag tablets and oral suspension. Camber Pharmaceuticals. Accessed May 14, 2025. https://www.cambereltrombopag.com
3. Dmytrijuk A, Robie-Suh K, Rieves D, Pazdur R. Eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura. Oncology (Williston Park). 2009;23(13):1171-1177.
4. GSK's Promacta (eltrombopag) receives FDA approval of an additional indication. News release. GSK. August 26, 2014. Accessed May 14, 2025. https://www.gsk.com/en-gb/media/press-releases/gsks-promacta-eltrombopag-receives-fda-approval-of-an-additional-indication/
5. FDA approves Novartis drug Promacta for first-line SAA and grants breakthrough therapy designation for additional new indication. News release. Novartis. November 16, 2018. Accessed May 14, 2025. https://www.novartis.com/us-en/news/media-releases/fda-approves-novartis-drug-promacta-first-line-saa-and-grants-breakthrough-therapy-designation-additional-new-indication
6. Eltrombopag for oral suspension. Prescribing information. Camber Pharmaceuticals. September 2024. Accessed May 14, 2025. https://www.camberpharma.com/wp-content/uploads/2025/04/2103428-Eltrombopag-OS-Leaflet-500-x-750-mm.pdf