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

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.