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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.

Authors
  • Przepiorka, Donna1
  • Le, Robert Q1
  • Ionan, Alexei1
  • Li, Ruo-Jing1
  • Wang, Ying-Hong1
  • Gudi, Ramadevi1
  • Mitra, Soumya1
  • Vallejo, Jonathon1
  • Okusanya, Olanrewaju O1
  • Ma, Lian1
  • Yang, Yuching1
  • Patel, Paresma1
  • Mezaache, Djelila1
  • Shah, Rakhi1
  • Banerjee, Anamitro1
  • McLamore, Sherita1
  • Maung, Adam N2
  • Goldberg, Kirsten B3
  • Pazdur, Richard1, 3
  • Theoret, Marc R1, 3
  • And 1 more
  • 1 Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
  • 2 Oncology Center of Excellence Summer Scholars Program, U.S. Food and Drug Administration, Silver Spring, Maryland.
  • 3 Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, Maryland.
Type
Published Article
Journal
Clinical Cancer Research
Publisher
American Association for Cancer Research
Publication Date
Jun 13, 2022
Volume
28
Issue
12
Pages
2488–2492
Identifiers
DOI: 10.1158/1078-0432.CCR-21-4176
PMID: 35135839
Source
Medline
Language
English
License
Unknown

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. ©2022 American Association for Cancer Research.

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