Health Canada has approved Imbruvica (ibrutinib) for the treatment of patients with steroid dependent or refractory chronic graft-versus-host disease (cGVHD), a life-threatening condition that affects some patients who undergo stem cell transplant.
Following a priority review, the agency approved Imbruvica as an oral, once-daily treatment. The decision follows the approval of Imbruvica in the U.S. for the same indication.
Imbruvica, a Bruton’s tyrosine kinase (BTK) inhibitor, was developed by Cilag GmbH International, a member of the Janssen Pharmaceutical Companies, in collaboration with Pharmacyclics, an AbbVie company.
The drug already was approved for the treatment of certain leukemias and lymphomas. This is the sixth approval for Imbruvica in Canada.
cGVHD is a serious complication that develops in 30 to 70 percent of patients who receive stem cell transplants from a partially matched donor (a sibling, for example). It happens when the donor’s immune cells react against the patient’s healthy cells and tissues.
Currently, cGVHD is managed with corticosteroids and immunosuppressants. But these treatments weaken the immune system, making a patient more susceptible to infections and tumor progression.
“Symptoms related to cGVHD can have a significant impact on a patient’s quality of life, and for most they come after an already long and difficult battle with a life-threatening disease such as leukemia or lymphoma,” Andrew Daly, MD, director of the Alberta Blood and Marrow Transplant Program, said in a press release. Daly also is clinical associate professor at the Cumming School of Medicine, University of Calgary.
“Physicians have had a real challenge finding options with compelling clinical data to treat cGVHD safely and effectively. This approval provides a much-needed new approach for patients who fail initial therapy, as data shows treatment with Imbruvica resulted in improved patient outcomes,” Daly added.
Health Canada’s decision was supported by data from the PCYC-1129 Phase 1b/2 trial (NCT02195869). The study included 42 patients with cGVHD who failed to respond to first-line corticosteroid therapy. Participants received once-daily 420 mg of Imbruvica orally for up to 18 months.
About 67 percent of patients responded to the treatment, including 21 percent complete responses and 45 percent partial responses. Responses were observed across affected organs, including skin, mouth, gastrointestinal tract, and liver.
The benefits of Imbruvica led to a reduction of the median steroid dose used over the course of the study. Five patients completely stopped steroid therapy.
“Lymphoma and leukemia patients are among those who may be able to get a stem cell transplant and achieve a potential cure. The procedure also comes with a high risk of cGVHD, which can have a severe impact on a patient’s ability to work and go about their day-to-day life,” said Robin Markowitz, CEO of Lymphoma Canada.
“To date, many patients have had to rely on immunosuppressants and high-dose steroids to treat their cGVHD symptoms, and these come with significant issues. It is great news that patients finally have an approved treatment option,” she concluded.