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news.PR Pharmaceuticals has been granted orphan drug designation by the FDA for its lead compound, PulmoLAR for the treatment of pulmonary arterial hypertension.
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The FDA granted orphan drug status upon review of an application containing data from both preclinical and in vitro studies demonstrating the multiple effects of 2-methoxyestradiol named 2ME or PulmoLAR, in addressing many of the pathological processes associated with pulmonary arterial hypertension (PAH).
In endothelial cell cultures, 2ME can significantly reduce endothelin-1 levels and increase prostacyclin production. Cell culture data have also been generated showing 2ME is a potent inhibitor of vascular smooth muscle and endothelial cell proliferation.
In vitro effects have been translated into significant beneficial effects in various animal models of cardiovascular and renal injury. In animal models of PAH, treatment with PulmoLAR has resulted in markedly reduced vascular remodeling and right ventricular hypertrophy, and reduction of pulmonary hypertension and inflammatory cell infiltration in lung tissues.
Acknowledging the benefits of orphan drug status, Dr Claude Piche, PRP’s vice-president of clinical development said: “In addition to recognizing the potential the drug offers for this indication, orphan drug designation offers benefits in terms of regulatory exclusivity, assistance with clinical development and a waiver of PDUFA filing fees.”