FDA Grants Orphan Designation to Investigational Idiopathic Pulmonary Fibrosis Treatment

Officials with the 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.

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Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive disease characterized by fibrosis and worsening lung function that predominantly affects individuals aged 60 years and older. IPF remains difficult to diagnose and manage, however, because of its unknown etiology, varied clinical course, associated comorbidities, and nonspecific respiratory symptoms. In fact, data show that when compared with age-, gender-, race-, and region-matched controls, patients with IPF have a 126% increased risk of emergency room visits and a 134% higher risk of hospitalization (per person-years, respectively). Consequently, IPF-related hospitalizations and acute exacerbations are associated with a high economic burden, accentuating the need for treatments that slow disease progression and improve patients’ quality of life. Two agents, nintedanib and pirfenidone, were FDA-approved for the treatment of IPF in 2014 and received conditional recommendations for use in the updated American Thoracic Society IPF Clinical Practice Guidelines. However, use of these medications is expected to significantly increase cost of care, considering each treatment regimen exceeds $90,000 annually. To optimize pharmacoeconomic outcomes, the use of these medications requires innovative strategies to manage costs associated with drug utilization and clinical management. Specialty pharmacies have emerged and continue to play a significant role in managing drugs with high acquisition costs through medication dispensing, disease management programs, and negotiating discounts and rebates to optimize patient care. As a result, it is critical that specialty pharmacists understand evolving treatment paradigms, the clinical course and evolution of disease, as well as the safe and appropriate use of medications in the management of IPF.
Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive disease that is characterized by worsening dyspnea, declining lung function, nonspecific respiratory symptoms, a varied clinical course, as well as a host of comorbid conditions, making diagnosis and management challenging. Prior to the approval of pirfenidone and nintedanib in 2014, lung transplant and palliative care were the only options for management of IPF. With the addition of these 2 agents in the updated American Thoracic Society IPF Clinical Practice Guidelines with conditional recommendations, cost of care for IPF is expected to increase significantly. Pharmacists can play an important role for patients by knowing the nonpharmacologic and pharmacologic treatment options for patients with IPF, and providing education and guidance on the therapies to improve adherence.
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