United Therapeutics ($UTHR) suffered a setback in its pursuit of commercializing an oral therapy for pulmonary arterial hypertension (PAH) yesterday, as the FDA rejected the company's new drug application with a dreaded complete response letter.
The FDA found multiple weaknesses in the biotech's data package in support of oral treprostinil for PAH, a narrowing of the arteries to the lungs that triggers high blood pressure. Regulators questioned the effectiveness of the drug in measures of 6-minute walk distance and improvement in time to clinical worsening in late-stage studies of the drug, according to the company.
United has sought an oral version of treprostinil, which it already offers in injected, intravenous and inhaled forms on the market, to bolster its offerings in the competitive market for PAH meds. Both Bayer and Actelion have trumpeted upbeat Phase III data for those companies' respective new PAH therapies, riociguat and macitentan, over the past week. And those two oral meds are poised to battle for market share with Gilead ($GILD) in the coming years. United gave no signs of quitting on its oral drug, but the remedies to the program won't come quickly.
"We will continue using our best efforts to gain approval of oral treprostinil, and we will focus on doing so within the next four years," said United Therapeutics CEO Martine Rothblatt, in a statement. "We will convene with our experts over the next several weeks to decide which of several paths forward to pursue."
The agency gave no indication that quick fixes to the United's NDA would improve matters, saying it wasn't sure whether adding a clinical study to bolster the case for oral treprostinil would change its views. And the company's share prince sank in after-hours trading Tuesday evening.
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