FDA Grants Orphan Drug Status to Nitric Oxide as Potential IPF Treatment

Nitric oxide, a gas that induces the relaxation and widening of lung airways, has received orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of idiopathic pulmonary fibrosis (IPF).

The gas will be delivered in pulses through Bellerophon Therapeutics’ patented INOpulse device to act primarily on healthy regions of patients’ lungs.

Orphan drug status is given to investigative treatments for people with a rare disease, defined in the United States as disorders that affect fewer than 200,000 people. The designation comes with certain benefits, including financial incentives for therapy development and commercialization, U.S. market exclusivity for seven years following approval, FDA support for clinical studies, and fee exemptions and reductions.

“The receipt of orphan drug designation represents a significant milestone for our INOpulse clinical development program,” Fabian Tenenbaum, CEO of Bellerophon, said in a press release.

According to the company, this is the first therapy that holds the potential to improve physical activity, heart function, and blood oxygen distribution in people living with IPF and pulmonary hypertension (PH), a common complication seen in people with advanced pulmonary fibrosis (PF).

“The theory behind this delivery is that because inhaled nitric oxide is very short-acting and is deactivated quickly when it contacts blood, delivering it to well-ventilated parts of the lung will allow it to open up the blood vessels where good gas exchange is possible,” the company states on its website.

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The safety and efficacy of nitric oxide delivered through the INOpulse device is now being evaluated in the double-blind, placebo-controlled, Phase 2b iNO-PF trial (NCT03267108) in a group of IPF patients with or without PH.

Participants will be randomly assigned to one of three groups, in which they will receive either nitric oxide or a placebo administered at different doses, for an initial period of eight or 16 weeks. After completion of this early study, all patients will be treated with nitric oxide during an additional open-label treatment period of eight weeks.

The iNO-PF trial is currently recruiting participants. Find more about locations and contacts here.

“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,” Tenenbaum said. “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.”