The U.S. Food and Drug Administration (FDA) has approved ibrutinib (Imbruvica) for pediatric patients ≥ 1 year of age with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy.
cGVHD is a rare complication that may occur after a stem cell or bone marrow transplant. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of cGVHD is unknown but it likely results from a complex immune-mediated interaction between the donor and recipient cells.
The approval was largely based on the iMAGINE study (NCT03790332), an open-label, multi-center, single-arm trial of ibrutinib for pediatric and young adult patients ( 1 – 22 years old; N=47) with moderate or severe cGVHD who required additional therapy after failure of 1 or more lines of systemic therapy.
The median age of patients was 13 years with a range of 1 to 19 years.
The primary outcome measure was overall response rate (ORR) through Week 25 . At the end of the 25 week period, the ORR was 60%. Further, the median duration of response was 5.3 months and median time from first response to death or new systemic therapies for cGVHD was 14.8 months.
The most common adverse reactions were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.
The recommended dosage of ibrutinib is age dependent. For patients 12 years of age and older with cGVHD, the recommended dose is 420 mg orally once daily, and for patients 1 to 11 years of age with cGVHD is 240 mg/m2 orally once daily (up to a dose of 420 mg), until cGVHD progression, recurrence of an underlying malignancy, or unacceptable toxicity.
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