United Therapeutics Corp. has scored a key regulatory designation that brings the Silver Spring biotech a step closer to moving beyond its core franchise.
The drugmaker said Wednesday the Food and Drug Administration has awarded orphan drug status to treprostinil, the active ingredient in several of its products, as a treatment for patients with idiopathic pulmonary fibrosis (IPF) — a progressive lung disease that scars the lungs and impedes breathing.
Armed with this designation, United Therapeutics (NASDAQ: UTHR) said it plans to kick off a late-stage clinical trial of Tyvaso, its product for pulmonary arterial hypertension containing an inhaled form of treprostinil, for patients with IPF.
The orphan drug status comes after the FDA cleared UT to begin a Phase 3 clinical trial of treprostinil in patients with IPF, the company said Wednesday. Enrollment for that study should begin in 2021. It would build upon a positive February trial that showed Tyvaso was effective in increasing the distance patients with interstitial lung disease could walk, paving the way for UT to break into pulmonary fibrosis and expand beyond its core PAH business.
The FDA grants orphan drug status to drugs that address rare diseases, or those that affect fewer than 200,000 people in the U.S. In the case of IPF, two approved drugs exist but there is no cure for the condition, which affects roughly 100,000 people in the U.S. with between 30,000 and 40,000 new cases diagnosed each year, according to the U.S. National Library of Medicine at the National Institutes of Health.
The designation brings support throughout the development and regulatory review process to help get it to patients faster. It brings tax incentives, research subsidies, grants, FDA fee waivers, extended patent protection and marketing rights to help cut development and commercialization costs.
The company also said it's “the first step” toward receiving what’s called orphan drug exclusivity after a regulatory approval, which gives the product seven years of market exclusivity for that indication. That’s critical for UT, as the company contends with generic competition for some of its other products.
Earning that designation “validates our drive to address orphan diseases, like IPF, with a significant unmet need,” United Therapeutics Chairman and CEO Martine Rothblatt said in a statement. The new trial "represents a significant move outside the pulmonary hypertension space, but based on data collected during the recent INCREASE study we’re confident that inhaled treprostinil can help address clinical gaps presented by existing therapies in IPF,” she added.
UT’s win comes after a strong third quarter in which the biotech said it had more patients than ever before using some of its PAH medicines, and reported double-digit growth year-over-year in the number of patients using two of them. It raked in $1.098 billion in revenue for first nine months of 2020, a 3% decrease from its $1.137 billion in revenue for the same period of 2019. It also had $570.1 million in operating expenses for the first three quarters of the year, a notable decrease from its $1.4 billion in 2019. As of September, UT counted $2.8 billion in total cash and cash equivalents and marketable investments.
The company’s stock closed up 3.92% at $135.08 per share Tuesday and was trading down 1.2% at 133.88 around 1 p.m. Wednesday.
