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Myelofibrosis | FDA grants an orphan drug designation for PXS-5505

Jul 15, 2020

On July 14, 2020, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation for PXS-5505.

Myelofibrosis is a rare hematological disease where bone marrow is gradually replaced by fibrotic tissue eventually leading to bone marrow failure. Patients with myelofibrosis have limited therapeutic options and a poor prognosis. Lysyl oxidases (LOX) are involved in cross-linking extracellular matrix proteins and have been shown to be upregulated in patients with myelofibrosis.1  PXS-5505, a pan-LOX inhibitor, is currently under development for the treatment of myelofibrosis. It is an oral compound that has demonstrated a well-tolerated safety profile, as well as good pharmacokinetic and pharmacodynamic properties in a phase I study. Also, in vivo models have demonstrated significant reductions in fibrotic burden.2

Read here for more information on myelofibrosis.

  1. Tadmor T, Bejar J, Attias D, et al. The expression of lysyl‐oxidase gene family members in myeloproliferative neoplasms. Am J Hematol. 2013;88(5):355-358. DOI: 10.1002/ajh.23409
  2. Pharmaxis granted orphan drug designation for PXS‐5505 by FDA for treatment of myelofibrosis. Jul 14, 2020. Accessed Jul 14, 2020.