FDA grants orphan drug status to Sanofi’s Rilzabrutinib

Published 03/06/2025, 07:02
© Reuters.

Investing.com -- The Food and Drug Administration (FDA) has granted orphan drug designation to Rilzabrutinib, a drug developed by Sanofi (NASDAQ:SNY) SA (EPA:SAN) for the treatment of sickle cell disease, according to the company on Tuesday.

This marks the fourth time that Rilzabrutinib has received this status.

Orphan drug designation is granted to drugs that are intended for the treatment of rare diseases, offering benefits such as market exclusivity for a certain period, tax credits, and regulatory support.

Rilzabrutinib is currently under regulatory review in the United States, the European Union, and China for Immune Thrombocytopenia (ITP), a disorder that can lead to easy or excessive bruising and bleeding due to abnormally low levels of platelets, the cells that help blood clot.

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