FDA approved eltrombopag (Promacta for oral suspension, Novartis) for the treatment of thrombocytopenia in pediatric patients 1 year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. August 24, 2015. More Information: http://www.fda.gov/Drugs/
Eltrombopag / Promacta
On August 24, 2015, the U. S. Food and Drug Administration approved eltrombopag (Promacta for oral suspension, Novartis) for the treatment of thrombocytopenia in pediatric patients 1 year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
The approval was based on two randomized, double-blinded, placebo-controlled trials enrolling pediatric patients with chronic ITP. The PETIT trial (PEdiatric patients with Thrombocytopenia from ITP) was a phase 2 trial with the primary efficacy endpoint of the proportion of subjects achieving platelet counts greater than or equal to 50 x109/L at least once between days 8 and 43 of the randomized period of the study. Patients were stratified by age cohort (12-17 years, 6-11 years, and 1-5 years), and 67 patients were randomized (2:1) to eltrombopag or placebo for seven weeks. Eltrombopag dose was titrated to a target platelet count of 50-200 x109/L. The percentage of responders was statistically significantly higher in patients treated with eltrombopag compared to placebo (62.2% vs. 31.8%, p-value = 0.011).
The PETIT2 trial was a phase 3 trial with the primary efficacy endpoint of the proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts greater than or equal to 50 x109/L for at least 6 out of 8 weeks, between weeks 5-12 of the randomized period. In the randomized period, 92 pediatric patients with chronic ITP were randomized (2:1) to eltrombopag or placebo for 13 weeks. Eltrombopag dose was titrated to a target platelet count of 50-200 x109/L. More patients in the eltrombopag group met the primary endpoint than in the placebo group (41.3% vs. 3.4%, p-value < 0.001).
Safety data was evaluated in 107 patients who were randomized to eltrombopag during both trials. The most common adverse reactions that occurred more frequently in patients treated with eltrombopag were upper respiratory tract infection, nasopharyngitis, cough, diarrhea, pyrexia, rhinitis, abdominal pain, oropharyngeal pain, toothache, ALT increased, rash, AST increased, and rhinorrhea.
Serious adverse reactions were reported in 8% of patients during the randomized part of both trials with no serious adverse event occurring in more than one patient (1%). An elevation of ALT greater than or equal to 3x upper limit of normal occurred in 5% of patients in the eltrombopag group, and of those 2% had increases in ALT greater than or equal to 5x upper limit of normal. No deaths or thromboembolic events occurred during either study.
The recommended dose and schedule for pediatric patients 6 years and older is 50 mg daily or 25 mg daily of the tablet formulation for patients with East Asian ancestry. The recommended dose for all patients age 1 to 5 years is 25 mg daily of the powder for oral suspension formulation.
Full prescribing information, including clinical trial information, safety, dosing, drug-drug interactions and contraindications is available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/207027s000lbl.pdf
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online athttp://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088)
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