Sanofi’s investigational therapy rilzabrutinib has received breakthrough therapy designation from the FDA for the treatment of warm autoimmune hemolytic anemia.
The designation is based on data from Sanofi’s ongoing Lumina 2 phase 2b trial, which is evaluating the safety and efficacy of rilzabrutinib in patients with warm autoimmune hemolytic anemia, according to a Feb. 9 company news release. A phase 3 study comparing rilzabrutinib with placebo is also underway.
Rilzabrutinib is the first and only investigational Bruton’s tyrosine kinase inhibitor to receive FDA breakthrough therapy designation for the condition, the release said.
Sanofi leaders said the designation highlights a critical unmet need for people with the rare autoimmune condition, which currently has no approved therapy targeting its underlying cause.
The therapy is approved in the U.S., the EU and the United Arab Emirates under the brand name Wayrilz for adults with immune thrombocytopenia.
