Sanofi has received FDA orphan drug designation for rilzabrutinib, an investigational oral BTK inhibitor, as a potential treatment to reduce vaso-occlusive crises in sickle cell disease (SCD). The therapy is currently under regulatory review for ITP in the U.S., EU, and China, with an FDA decision expected by August 29, 2025. Rilzabrutinib represents a novel approach to SCD treatment by targeting inflammation through multi-immune modulation. Preclinical data presented at ASH 2024 demonstrated the therapy’s potential to reduce vaso-occlusion and inflammation in transgenic mouse models of SCD. The drug’s unique mechanism of action could address critical unmet needs for SCD patients who continue to experience painful crises despite available treatments.

OxyDial recognizes the importance of this development in expanding the treatment landscape for SCD. As research continues to validate rilzabrutinib’s clinical benefits, ensuring global accessibility will be essential for improving outcomes in affected communities worldwide.

Read more: https://www.globenewswire.com/news-release/2025/06/03/3092429/0/en/Press-Release-Rilzabrutinib-granted-orphan-drug-designation-in-the-US-for-sickle-cell-disease.html

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