Sickle Cell Disease Market Snapshot: "The Time Has Come"
Three new drugs for sickle cell disease could be on the market in the US next year, representing a groundswell of advancement for the devastating blood disorder that has had limited options.
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Vertex and partner CRISPR Therapeutics hope CDX001 can offer a single-use curative regimen for sickle cell disease and beta thalassemia patients. A regulatory filing is possible within 18-24 months.
GlycoMimetics’ commercial and pipeline prospects are in doubt after Pfizer terminated their 2011 sickle cell drug development deal, following a failed late-stage study.
Oxbryta is the second new drug for sickle cell disease approved in just over one week, but it was approved with a broad label for sickle cell disease while Novartis' Adakveo was approved for pain crises.