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. 2022 Jun 13;28(12):2488-2492.
doi: 10.1158/1078-0432.CCR-21-4176.

FDA Approval Summary: Belumosudil for Adult and Pediatric Patients 12 Years and Older with Chronic GvHD after Two or More Prior Lines of Systemic Therapy

Donna Przepiorka  1 , Robert Q Le  1 , Alexei Ionan  1 , Ruo-Jing Li  1 , Ying-Hong Wang  1 , Ramadevi Gudi  1 , Soumya Mitra  1 , Jonathon Vallejo  1 , Olanrewaju O Okusanya  1 , Lian Ma  1 , Yuching Yang  1 , Paresma Patel  1 , Djelila Mezaache  1 , Rakhi Shah  1 , Anamitro Banerjee  1 , Sherita McLamore  1 , Adam N Maung  2 , Kirsten B Goldberg  3 , Richard Pazdur  1   3 , Marc R Theoret  1   3 , R Angelo De Claro  1
Affiliations

FDA Approval Summary: Belumosudil for Adult and Pediatric Patients 12 Years and Older with Chronic GvHD after Two or More Prior Lines of Systemic Therapy

Donna Przepiorka et al. Clin Cancer Res. .

Abstract

On July 16, 2021, the FDA approved belumosudil, a kinase inhibitor, for adult and pediatric patients 12 years and older with chronic GvHD (cGvHD) after failure of at least two prior lines of systemic therapy. Approval was based on the results of Study KD025-213, which included 65 patients with cGvHD treated with belumosudil 200 mg daily in an open-label, single-arm cohort. Efficacy was determined by the overall response rate (ORR) through Cycle 7 Day 1, which included complete response (CR) or partial response (PR) according to the 2014 NIH consensus criteria, and durability of response. The ORR through Cycle 7 Day 1 was 75% [95% confidence interval (CI), 63-85]; 6% of patients achieved a CR, and 69% achieved a PR. The median duration of response was 1.9 months (95% CI, 1.2-2.9), and 62% (95% CI, 46-74) of responding patients remained alive without new systemic therapy for at least 12 months from response. The common adverse reactions were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma-glutamyl transferase increased, lymphocytes decreased, and hypertension. Additional study is warranted to confirm safety with long-term use.

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Conflict of interest statement

Disclosure of Potential Conflicts of Interest: The authors report no financial interests or relationships with the commercial sponsors of any products discussed in this report.

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