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Sanofi’s Rilzabrutinib Gains Orphan Drug Status for Rare Diseases

Story Highlights
  • Sanofi’s rilzabrutinib received orphan drug designation from the FDA for two rare diseases.
  • Rilzabrutinib shows promise as a treatment for multiple immune-mediated diseases, including ITP.
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Sanofi’s Rilzabrutinib Gains Orphan Drug Status for Rare Diseases

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An announcement from Sanofi ( (SNY) ) is now available.

In April 2025, Sanofi announced that the US FDA granted orphan drug designation to rilzabrutinib for two rare diseases, warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), which currently have no approved treatments. This designation underscores Sanofi’s commitment to addressing unmet medical needs in rare diseases. Rilzabrutinib, an investigational BTK inhibitor, is also under regulatory review for immune thrombocytopenia (ITP) in the US, EU, and China, with a target FDA decision date set for August 29, 2025. The drug has shown promising results in clinical trials, indicating its potential as a first- and best-in-class treatment for multiple immune-mediated diseases.

More about Sanofi

Sanofi is an innovative global healthcare company focused on transforming the practice of medicine by developing potentially life-changing treatment options and life-saving vaccines. The company is committed to sustainability and social responsibility, providing healthcare solutions to millions worldwide. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY.

YTD Price Performance: 13.34%

Average Trading Volume: 2,611,473

Technical Sentiment Signal: Strong Sell

Current Market Cap: $135.2B

For detailed information about SNY stock, go to TipRanks’ Stock Analysis page.

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