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BioWorld - Saturday, June 20, 2026

Rare disease

Home » Topics » Disease categories and therapies » Rare disease
  • Fda icons and doctor
    June 11, 2026
    By Karen Carey

    Another FDA no for Camurus’ monthly acromegaly drug Oclaiz

    In a repeat move, the U.S. FDA issued yet again a complete response letter (CRL) to Camurus AB for its subcutaneous extended-release injection drug CAM-2029 (octreotide) to treat the rare chronic growth disorder acromegaly. The drug, which expects to be branded Oclaiz in the U.S. upon approval, is called Oczyesa in the EU and the U.K., where it received marketing authorization in 2025.
  • Nephrology renal kidney
    June 2, 2026
    By Karen Carey

    Travere expands rare kidney portfolio in $1B+ Everest deal

    Less than two months after winning FDA approval for a second indication for Filspari (sparsentan), Travere Therapeutics Inc. added to its rare kidney disease pipeline by exclusively licensing civorebrutinib from Everest Medicines Ltd. in a deal that could be worth more than $1.14 billion.
  • Blood cancer illustration
    May 28, 2026
    By Randy Osborne

    Elzonris this: FDA clears Abbvie’s Decnupaz in BPDCN

    Payoff for the November 2023 buyout of Immunogen Inc. came for Abbvie Inc. in the form of U.S. FDA clearance for the CD123-targeting antibody-drug conjugate Decnupaz (pivekimab sunirine) to treat adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra-rare, aggressive and quick-killing hematologic malignancy.
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