“The availability of Uptravi is a milestone for PAH patients in Germany, where until now, the options for treatments targeting the prostacyclin pathway have been limited, and were burdensome for the patients,” Michael Danzl, general manager of Actelion Germany, said in a press release. “With Uptravi, there is now an oral option in all three established treatment pathways.”
A highly selective oral prostacyclin receptor (IP) agonist, Uptravi was originally discovered and synthesized by Nippon Shinyaku. Activation of the IP receptor induces vasodilation, which decreases blood pressure and inhibits the growth and proliferation of vascular smooth muscle cells.
Uptravi is indicated for the treatment of idiopathic PAH, heritable PAH, PAH associated with connective tissue disease, PAH associated with congenital heart disease with repaired shunts, and PAH associated with drugs and toxins, in patients with WHO functional class II, III or IV symptoms. Its use is either as a combination therapy in patients for whom an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor are insufficient, or as single-agent for those who cannot use these therapies.
The EU label for Uptravi was based in part on the findings of the GRIPHON study, a long-term Phase 3 clinical trial that assessed the drug’s safety, efficacy and tolerability in 1,156 PAH patients with WHO Functional Class II-III symptoms. Patients received Uptravi combined with an ERA, a PDE-5 inhibitor, an ERA with a PDE-5 inhibitor, or were treated only Uptravi.
GRIPHON demonstrated that treatment with Uptravi reduced the risk of morbidity/mortality events by 40 percent.
“Uptravi offers an oral therapy that effectively targets the prostacyclin pathway and significantly delays the progression of pulmonary arterial hypertension,” said Professor H. Ardeschir Ghofrani, from University Hospital Giessen, Germany. “It is supported by robust outcome-based evidence in combination with an ERA, or a PDE-5 inhibitor, and even in combination with both an ERA and a PDE-5 inhibitor. This opens the way for more oral combination treatments early on in the progression of the disease, with proven long-term outcome benefits for more patients.”
Hans Dieter Kulla, president of pulmonale hypertonie e.v., added: “This new product launch is a major step in terms of achieving the best possible care for patients living with PAH in Germany. We hope Uptravi can further impact the long-term outcome for many patients, which ultimately also helps their families and caregivers.”