Wayrilz recommended for approval in Europe to treat ITP
Final decision from European Commission expected in coming months
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An arm of the European Medicines Agency (EMA) is recommending that Wayrilz (rilzabrutinib) be approved to treat adults with immune thrombocytopenia (ITP) who have not responded well to other therapies.
The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), will now be reviewed by the European Commission, which has ultimate say over drug approvals in the European Union. The commission isn’t obligated to abide by the CHMP’s recommendations, but it almost always does.
Sanofi, Wayrilz’s developer, said a final decision from the commission is expected in the coming months.
“The CHMP’s recommendation of Wayrilz for approval is a positive step forward for people in the EU living with ITP who continue to experience symptoms that impact their daily living despite treatment with certain other therapies,” Brian Foard, executive vice president and head of specialty care at Sanofi, said in a company press release.
Wayrilz designed to inhibit key enzyme
ITP is an autoimmune disorder caused by antibodies that attack and destroy platelets, which are cell fragments that help blood clot. This results in low platelet levels, leading to symptoms like easy bleeding and bruising.
Wayrilz is an oral therapy designed to inhibit Bruton’s tyrosine kinase, an enzyme involved in the activation of immune cells that help drive ITP, including the B-cells that make platelet-attacking antibodies. By inhibiting these immune cells, the therapy aims to prevent the destruction of platelets and ultimately normalize clotting to control symptoms.
“Through multi-immune modulation, Wayrilz shows promise in addressing the root cause of ITP – complex immune system dysregulation,” Foard said.
The CHMP’s positive opinion comes just a few weeks after the U.S. Food and Drug Administration (FDA) approved Wayrilz for adults with persistent or chronic ITP who haven’t responded well to previous treatments. Both the CHMP’s opinion and the FDA’s approval were based on data from a Phase 3 clinical trial called LUNA 3 (NCT04562766), which tested Wayrilz against a placebo in more than 200 people with ITP who weren’t responding to, or couldn’t tolerate, available treatments.
The main goal of the LUNA 3 study was to evaluate Wayrilz’s effects on platelet levels. Specifically, the trial looked at the number of patients with a durable platelet response — defined as having platelet levels of at least 50 billion platelets per liter of blood on at least eight of the 12 weekly assessments in the last half of the six-month-long study. Results showed that none of the patients given the placebo had a durable platelet response, whereas 23% of patients given Wayrilz did. Secondary measures also consistently showed better outcomes with Wayrilz than with the placebo.
