FDA/CDC

Adjunctive Promacta approved for first-line SAA


 

The Food and Drug Administration has approved use of eltrombopag (Promacta) for patients severe aplastic anemia (SAA), Novartis announced Nov. 16.

The agency’s move means that eltrombopag, a synthetic thrombopoietin-receptor agonist, is now approved for use in combination with standard immunosuppressive therapy as first-line treatment for adults, and pediatric patients aged 2 and older with SAA. The drug received breakthrough therapy designation and priority review for this indication, the company said in a press release.

In addition, eltrombopag is FDA-approved for SAA patients who have had an insufficient response to immunosuppressive therapy, those with chronic immune thrombocytopenia who have had an insufficient response to other treatments, and those with thrombocytopenia and with chronic hepatitis C infection.

The expanded approval is based on results of a phase 1-2 trial (NCT01623167), which were published last year (N Eng J Med. 2017 Apr 20;376[16]:1540-50). The trial included 153 previously untreated SAA patients aged 2 and older. The patients received eltrombopag in combination with horse antithymocyte globulin and cyclosporine.

The starting dose of eltrombopag in the trial was 150 mg once daily for patients aged 12 and older (75 mg for East and Southeast Asians), 75 mg once daily for patients aged 6 to 11 (37.5 mg for East and Southeast Asians), and 2.5 mg/kg once daily for patients aged 2 to 5 (1.25 mg/kg for East and Southeast Asians).

Patients were divided into three cohorts with different dosing schedules. The recommended schedule from the third cohort (n = 92) was eltrombopag from day 1 to month 6, plus horse antithymocyte globulin and cyclosporine. All patients in this cohort were eligible to receive a low dose of cyclosporine for an additional 18 months if they achieved a hematologic response at 6 months.

Among the patients treated at the recommended dosing schedule, the 6-month overall response rate was 79%, and the complete response rate was 44%. The median duration of both overall and complete response was 24.3 months.

The most common adverse events in these patients were increases in ALT (29%), AST (17%), and blood bilirubin (17%). Also, rash (8%), and skin discoloration, including hyperpigmentation (5%), were cited as adverse events.

Updated results from the trial are available in the prescribing information for eltrombopag. In most countries outside of the United States, the drug is marketed as Revolade.

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