FDA grants Breakthrough Therapy designation to Wayrilz (rilzabrutinib) for warm autoimmune hemolytic anemia, while Japan awards orphan drug status, marking parallel regulatory progress for the rare blood disorder
Written By: Nikita Jha, BPharm
Reviewed By: Pharmacally Editorial Team
The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to Wayrilz (rilzabrutinib) for the treatment of warm autoimmune hemolytic anemia (wAIHA), a rare and potentially life-threatening autoimmune blood disorder. At the same time, Japan’s Ministry of Health, Labour and Welfare has awarded orphan drug designation to rilzabrutinib for the same indication, highlighting parallel regulatory progress in the US and Japan.
Both regulatory decisions are supported by data from the ongoing LUMINA 2 phase 2b study (NCT05002777), which is evaluating the efficacy and safety of rilzabrutinib in patients with wAIHA. A confirmatory LUMINA 3 phase 3 trial (NCT07086976) is now underway, comparing rilzabrutinib with placebo in this patient population.
“These recognitions highlight the critical unmet need that persists for people living with wAIHA,” said Karin Knobe, Global Head of Development, Rare Diseases, noting that the designations reinforce the company’s focus on developing innovative therapies for rare diseases with limited treatment options.
Rilzabrutinib is already approved as Wayrilz in the US, the EU, and the United Arab Emirates for adults with immune thrombocytopenia (ITP) and is currently under regulatory review for ITP in Japan. Outside of these approved ITP uses, all other indications, including wAIHA, remain investigational.
The FDA has previously granted rilzabrutinib orphan drug designation for autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease, along with fast-track designation in ITP and IgG4-related disease. In the EU, the drug also holds orphan designation in ITP, autoimmune hemolytic anemia, and IgG4-related disease.
Rilzabrutinib is a novel, oral, reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor designed to restore immune balance by modulating key immune pathways involved in autoimmune and inflammatory diseases. If approved for wAIHA, it could become the first therapy to directly address the immune drivers of this rare condition, potentially changing the treatment landscape for affected patients.
wAIHA is caused by immune-mediated destruction of red blood cells, leading to anemia, fatigue, shortness of breath, and an increased risk of thromboembolic complications. Despite its serious nature, there are currently no approved therapies that directly target the underlying disease mechanism.
References
Press Release: Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic anemia, 09 February 2026, Press Release: Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic anemia
Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA) (LUMINA 2), ClinicalTrials.gov ID NCT05002777, https://clinicaltrials.gov/study/NCT05002777
A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia (LUMINA 3), ClinicalTrials.gov ID NCT07086976, https://clinicaltrials.gov/study/NCT07086976