FDA Approves J&J’s Uptravi for Intravenous Use in Adult Patients with PAH

Rare Daily Staff

The U.S. Food and Drug Administration approved Johnson & Johnson division Janssen Pharmaceuticals’ Uptravi injection for intravenous use for the treatment of pulmonary arterial hypertension in adult patients with WHO functional class II–III, who are temporarily unable to take oral therapy.

Pulmonary Arterial Hypertension (PAH) is a specific form of pulmonary hypertension that causes the walls of the pulmonary arteries to become thick and stiff, narrowing the space for blood to flow, and causing an increased blood pressure to develop within the lungs. PAH is a rare, serious, progressive disease with a variety of etiologies and has a major impact on patients’ functioning as well as their physical, psychological and social wellbeing. There is currently no cure for PAH and it is often fatal.

Uptravi (selexipag) is a selective prostacyclin IP receptor agonist, discovered by Nippon Shinyaku and licensed to J&J division Actelion Pharmaceuticals outside Japan. It is licensed for the oral treatment of PAH in more than 60 countries.

Uptravi IV is a therapeutic option that will allow patients to avoid short-term treatment interruptions and stay on Uptravi therapy, as uninterrupted treatment is considered key for individuals with PAH. Uptravi tablets were first approved by the FDA in 2015 to delay disease progression and reduce the risk of hospitalization for PAH.

The FDA approval of the for Uptravi is based upon the findings from a prospective, multi-center, open-label single sequence cross-over phase 3 study in 20 patients that was designed to assess the safety, tolerability and pharmacokinetics of temporarily switching between Uptravi tablets and Uptravi IV. The results were published earlier this year in Respiratory Research and examined switching from a stable dose of Uptravi tablets to a corresponding dose of Uptravi IV and back to Uptravi tablets. The study found that the switch between tablets and IV was well tolerated with no unexpected safety findings.

“Today marks an important day for patients who rely on Uptravi, as this new intravenous formulation meets a current unmet need for these patients. As part of our commitment to investing in research and understanding the science around the potential of Uptravi, we’re inspired by this approval and are proud to be paving the way to advance treatment options and care for patients with PAH,” said Neil Davie, head of Global Therapeutics, Pulmonary Hypertension, Janssen.

Photo: Neil Davie, head of Global Therapeutics, Pulmonary Hypertension, Janssen