Biotechnology company United Therapeutics Corporation (Nasdaq:UTHR) said on Monday that the US Food and Drug Administration (FDA) has accepted its New Drug Application for ralinepag to treat pulmonary arterial hypertension (PAH), setting a Prescription Drug User Fee Act target action date of 24 June 2027.
If approved, ralinepag would be the first and only once-daily oral prostacyclin for PAH, according to the company.
The filing is supported by Phase 3 ADVANCE OUTCOMES data, in which ralinepag reduced the risk of clinical worsening by 55% versus placebo in predominantly pre-treated PAH patients. Ralinepag also reduced NT-proBNP levels, improved exercise capacity and increased the odds of clinical improvement by 47%.
The results were consistent across patient subgroups, including disease etiology, six-minute walk distance, WHO Functional Class, NT-proBNP levels and background therapies.
United Therapeutics said ralinepag's safety profile was consistent with known prostacyclin-related adverse events, with no new safety signals identified.
Ralinepag remains investigational and has not been approved by the FDA for any indication.
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