The designation has been given by the US Food and Drug Administration to treat patients with idiopathic pulmonary fibrosis.
Bellerophon Therapeutics (NASDAQ:BLPH) has announced it has received orphan drug designation from the US Food and Drug Administration (FDA) for nitric oxide in treating patients with idiopathic pulmonary fibrosis (IPF).
As quoted in the press release:
IPF is a progressive, irreversible and fatal interstitial lung disease characterized by thickening and scarring of the air sacs in the lungs affecting approximately 100,000 people in the U.S. and reducing their life expectancy to between two and five years from diagnosis. IPF patients suffer from severe functional impairment that limits their ability to perform basic daily tasks, resulting in a significant deterioration in their quality of life. Pulsed nitric oxide, delivered via Bellerophon’s patented INOpulse® device and delivery algorithm, is the first therapy with the potential to improve a patient’s physical activity levels, cardiac output and oxygen saturation by treating both the ventilation perfusion mismatch driven by the fibrosis, as well as pulmonary hypertension, a common comorbidity in the IPF patient population.
Bellerophon is currently conducting a Phase 2/3 (iNO-PF) study of nitric oxide and its proprietary INOpulse system to treat patients with IPF, as well as other pulmonary fibrotic diseases.
“The receipt of orphan drug designation represents a significant milestone for our INOpulse clinical development program,” said Fabian Tenenbaum, Chief Executive Officer of Bellerophon. “We are encouraged by the Cohort 1 results of our iNO-PF study, which demonstrated that treatment with INOpulse provided clinically and statistically significant improvement in activity levels. We recently completed enrollment in Cohort 2, which is assessing a higher dose, as well as longer treatment duration, and expect to report top-line results before the end of the year. Moreover, we have FDA agreement on our pivotal Phase 3 Cohort, which we anticipate initiating in the first quarter of 2020. IPF is a debilitating life threatening disease and we believe that INOpulse is well-positioned to potentially become a first-in class therapy for this serious unmet medical need.”