Orphan designation qualifies the sponsor of the drug for various development incentives such as reduced regulatory fees and extended periods of market exclusivity.

() has been granted an orphan drug designation for its pan LOX inhibitor PXS-5505 for the treatment of myelofibrosis by the US Food and Drug Administration (FDA).

PXS5505 is an oral drug that inhibits all lysyl oxidase family members (LOX, LOXL1, 2, 3 & 4).

The compound successfully cleared preclinical safety including 6month toxicity studies and has shown significant reductions in fibrosis in invivo models of myelofibrosis and other cancers.

PXS5505 has shown to be well tolerated in phase 1 single and multiple ascending dose studies in humans with an excellent pharmacokinetic and pharmacodynamic profile.

Orphan designation qualifies the sponsor of the drug for various development incentives such as reduced regulatory fees and extended periods of market exclusivity.

Pharmaxis chief executive officer Gary Phillips said: We are very pleased with the FDA orphan drug designation for PXS5505.

Pharmaxis believes that the current treatments for myelofibrosis can be augmented by a panLOX inhibitor and be disease modifying in a market with high unmet need and significant deal values for programs with clinical proof of concept.

We expect to file an investigational new drug (IND) application with the FDA shortly and will provide an update on the clinical trial plans at that time.

Myelofibrosis is a rare cancer in which normal bone marrow tissue is gradually replaced with a fibrous scarlike material.

Over time, this leads to progressive bone marrow failure preventing the production of adequate numbers of red cells, white cells and platelets.

Myelofibrosis has a poor prognosis and limited therapeutic options.

Apart from a small group of patients eligible for stem cell transplantation, current standard of care are JAK1/2 inhibitors which provide mainly symptomatic relief but carry a risk of worsening blood cell counts.

A recent publication reported that Pharmaxis panLOX inhibitor compounds significantly decreased the bone marrow fibrotic burden in two different models of primary myelofibrosis.

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Pharmaxis granted orphan drug designation for PXS5505 by FDA for treatment of myelofibrosis - Proactive Investors Australia