FDA Grants Reviva Pharmaceuticals' RP5063 Orphan Designation for IPF

The US Food and Drug Administration (FDA) granted Orphan Drug Designation for Reviva Pharmaceuticals’ clinical stage drug candidate, RP5063 for treatment of idiopathic pulmonary fibrosis (IPF).

The drug has shown robust efficacy in highly recognized translational animal models proven to emulate pulmonary fibrosis in humans, significantly improving survival rate and reducing inflammatory cytokines and fibrosis in the lungs of bleomycin-induced pulmonary fibrosis animal model.

The findings indicate the potential for a clinically meaningful improvement and stabilization of lung function.

“Obtaining the Orphan Drug Designation is a significant regulatory milestone as well as a notable achievement for Reviva, since it validates the significant therapeutic potential of RP5063 for the treatment of IPF,” Laxminarayan Bhat, PHD, found, president, chief executive officer, Reviva, said in a statement.

The new chemical entity involves a novel mechanism of multimodal modulation of serotonin and dopamine signaling pathways. A potent inhibitor of the 5-HT2A/2B/7 receptors, RP5063 weakens functional changes in a series of well recognized translational animal models proven to emulate pulmonary fibrosis and pulmonary arterial hypertension.

“IPF is a devastating disease with limited treatment options and no cure,” Bhat added. “RP5063, however, provides a novel mechanism of action that has the potential to impact multiple aspects of this disease and, combined with convenient delivery options, RP5063 affords hope to many patients suffering from this debilitating disease.”

In the US, IPF, a progressive, debilitating and fatal disease, affects approximately 150,000 people with 48,000 new cases diagnosed annually.

The FDA also recently granted Orphan Drug Designation to RP5063 for treatment of pulmonary arterial hypertension (PAH) and plans to launch phase 2 clinical trials for IPF and PAH in the near future.

With orphan designation, Reviva qualifies for various incentives like FDA assistance in clinical trial design, tax credits for clinical trial costs, an exemption from the FDA user fee and 7 years of market exclusivity in the US when market approval is granted.