Sanofi Gains European Authorization for Wayrilz in the Treatment of Immune Thrombocytopenia
The European Commission has approved Wayrilz (rilzabrutinib), a Bruton's tyrosine kinase inhibitor developed by Sanofi, for the treatment of immune thrombocytopenia in adult patients who are refractory to other treatments, according to the company. This marks the first BTK inhibitor authorized in the European Union for this indication.
European Market Authorization Based on Phase 3 LUNA 3 Study Results
The authorization for market release in the European Union is based on the results of the pivotal phase 3 LUNA 3 study, which assessed the efficacy and safety of the treatment in 202 adults with persistent or chronic immune thrombocytopenia, the company states. Patients received either 400 mg of Wayrilz orally twice a day or a placebo, during a double-blind treatment period of 12 to 24 weeks. According to results presented at the 66th annual meeting of the American Society of Hematology and published in the journal Blood, 64% of patients in the Wayrilz group achieved a platelet count response at 12 weeks, compared to 32% in the placebo group. The primary endpoint for the European Union focused on the proportion of participants achieving a platelet count of 50,000 per microliter or higher for at least eight of the last 12 weeks of the treatment period, in the absence of rescue treatment, the company specifies.
Improvement in Overall Quality of Life Reported
According to the study, patients treated with Wayrilz reported a 10.6-point improvement in overall quality of life, compared to a 2.3-point increase in the placebo group, measured by the immune thrombocytopenia patient assessment questionnaire. However, the results of this analysis are descriptive and the study was not designed to demonstrate statistical significance, the company emphasizes. The most common adverse effects, observed in at least 10% of patients, include diarrhea, nausea, headaches, abdominal pain, and COVID-19, according to the group. Wayrilz acts by inhibiting Bruton's tyrosine kinase, a protein expressed in B cells, macrophages, and other innate immune cells, allowing multi-immune modulation to treat the underlying pathology of immune thrombocytopenia, Sanofi states.
Wayrilz Already Approved in the US and UAE, Under Review in Japan and China
Wayrilz is already approved for immune thrombocytopenia in the United States and the United Arab Emirates, and is currently under regulatory review in Japan and China for this indication, the company reports. The treatment has received fast track and orphan drug designations in the United States for immune thrombocytopenia, as well as similar orphan drug designations in the European Union and Japan, the company specifies. In terms of other indications under study, the US Food and Drug Administration has granted Wayrilz orphan drug status for three additional rare diseases, namely warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease, according to Sanofi. These additional indications are currently under study and have not been approved by regulatory authorities, the group notes.
