Sanofi's Rilzabrutinib Gets FDA's Rare-Disease Drug Designation

 Sanofi said that its Rilzabrutinib treatment was granted orphan drug designation, which is used in the U.S. by the Food and Drug Administration.

The designation is a status assigned to a medicine intended for use against a rare condition.

The designation is applied to the drug for the treatment of two rare immune system diseases with no approved medicines, warm autoimmune hemolytic anemia and IgG4-related disease, the French drug-maker said.

The FDA's designation comes as the drug is currently under review in the U.S., the EU, and China for its potential use in immune thrombocytopenia, a disease that can lead to bruising and bleeding.

The FDA is expected to unveil its regulatory decision on the use of the drug for immune thrombocytopenia on August 29.

Rilzabrutinib also received orphan drug designation for immune thrombocytopenia in the U.S., EU, and Japan.

https://www.morningstar.com/news/dow-jones/20250403897/sanofis-rilzabrutinib-gets-fdas-rare-disease-drug-designation