Wayrilz wins approval in Japan for treating hard-to-manage ITP
OK for Sanofi’s twice-daily pill follows similar approvals in US, Europe
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Regulatory authorities in Japan have approved Sanofi’s oral therapy Wayrilz (rilzabrutinib) for treating people with immune thrombocytopenia (ITP) who haven’t responded to, or cannot tolerate, other therapies.
The marketing and manufacturing authorization from Japan’s Ministry of Health, Labor and Welfare follows similar approvals of Wayrilz in the U.S., the European Union, the United Arab Emirates, and the U.K.
“People living with immune thrombocytopenia who continue to experience symptoms, despite trying existing treatments, face a great unmet need,” Manuela Buxo, executive vice president, global head of specialty care at Sanofi, said in a company press release announcing this latest authorization.
Buxo called the approval in Japan a “milestone,” and said it “underscores Sanofi’s commitment to bringing innovative treatment options to rare disease patients around the world.”
ITP is a rare autoimmune disease in which the immune system attacks and destroys platelets, which are cell fragments that normally help blood to clot. The resulting low platelet levels can lead to symptoms such as unusually easy and prolonged bleeding.
Wayrilz found to ease symptoms, improve life quality in clinical trials
The newly approved treatment was found in clinical trials to ease symptoms and reduce bleeding risk in people with ITP. It works by blocking the activity of Bruton’s tyrosine kinase (BTK), an enzyme that plays a key role in the activation of immune cells that drive platelet destruction in ITP.
“Wayrilz … offers new hope to patients who have not responded to prior therapy,” Buxo said, noting the therapy “has a differentiated mechanism of action that enables multi-immune modulation to address the underlying pathophysiology [processes] of ITP.”
Regulatory approvals of Wayrilz across the globe have been based predominantly on data from a Phase 3 clinical trial called LUNA 3 (NCT04562766), which tested the twice-daily oral therapy against a placebo in more than 200 people with ITP. The study’s participants didn’t respond to, or couldn’t tolerate, standard treatments. The initial placebo-controlled part of the study lasted as long as six months, after which all participants were treated with Wayrilz for several more months.
Results from LUNA 3 showed Wayrilz was significantly better than the placebo at boosting platelet levels. The therapy also outperformed the placebo on the Immune Thrombocytopenia Patient Assessment Questionnaire, a clinical tool that’s used to measure the severity of ITP symptoms and the impact of the disease on patients’ day-to-day lives. Average scores on this questionnaire improved by more than 10 points in patients given Wayrilz, compared with less than three points for those on the placebo. Sanofi noted that the quality of life analyses were not designed to detect statistical significance.
[Wayrilz] is expected to become a new therapeutic option that may help patients with disease management, including not only improvement in platelet counts but also considering patients’ quality of life.
“For ITP patients, achieving platelet counts close to normal and living without worrying about bleeding is extremely important,” said Hisashi Kato, MD, PhD, of The University of Osaka Hospital in Japan. “Additionally, in recent years, there has been growing interest in various other symptoms associated with ITP such as fatigue.”
According to Kato, the “novel” treatment “works through multi-immune modulation to help address the root cause of ITP.”
The most common side effects of Wayrilz, each reported in at least 10% of participants in LUNA 3, are diarrhea, nausea, headache, abdominal pain, and COVID-19.
Kato said that, with the approval in Japan, Wayrilz “is expected to become a new therapeutic option that may help patients with disease management, including not only improvement in platelet counts but also considering patients’ quality of life.”
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