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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 vivomodels have demonstrated significant reductions in fibrotic burden. 2

Read herefor 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. https://www.proactiveinvestors.com.au/companies/news/924111/pharmaxis-granted-orphan-drug-designation-for-pxs5505-by-fda-for-treatment-of-myelofibrosis-924111.html.Published Jul 14, 2020. Accessed Jul 14, 2020.