Actelion announced that one of its lead products, Uptravi (selexipag), designed for the treatment of pulmonary arterial hypertension (PAH), has become commercially available in Germany. The drug was granted market authorization by the European Commission on May 12, and has been commercially available for patients in Germany since June 15.
Selexipag received market authorization by the U.S. FDA in December 2015. The drug also received market authorization in Canada, New Zealand, Australia, and South Korea. The drug is still under regulatory review in countries including Japan, Switzerland, Taiwan, and Turkey.
Patients with the chronic life-threatening disorder PAH suffer from narrowed and blocked arteries that carry blood from the heart to the lungs. This results in build-up of pressure in the pulmonary artery in a way that can influence heart function and eventually lead to complications like heart failure. There is currently no cure for PAH, but several treatments are available to relieve symptoms and expand patient’s life expectancy.
Uptravi is one of the drugs approved for the long-term therapy of adult patients with PAH, indicated either as a monotherapy or in combination with other treatments. The drug was shown to be efficient in PAH patients, including PAH connective tissue disorders, idiopathic/heritable PAH, and corrected simple congenital heart disease-associated PAH.
In Europe, the Phase 3 GRIPHON study demonstrated that Uptravi was efficient, tolerable, and safe in PAH patients. It was also found to reduce morbidity/mortality incidents by 40 percent. Data from the trial was published in December 2015 issue of The New England Journal of Medicine.
The GRIPHON study included 1,156 patients with symptomatic PAH. The primary endpoint was to evaluate the safety of Uptravi for long-term use of up to 4.2 years. The other evaluated endpoint was safety, which found that Uptravi induced side effects in 3 percent or more of treated patients when compared to a placebo, particularly during the dose titration phase.
Typical recorded side effects were headaches, nausea or vomiting, diarrhea, jaw pain, anemia, myalgia, pain in extremity, flushing, reduced appetite, rash, and arthralgia. Also, about 1 percent of patients on Uptravi suffered from hyperthyroidism compared to none in the placebo group.
“Uptravi offers an oral therapy that effectively targets the prostacyclin pathway and significantly delays the progression of pulmonary arterial hypertension,” said Prof. H. Ardeschir Ghofrani of University Hospital Giessen in Germany, in a press release. “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.”
Michael Danzl, general manager of Actelion Germany, said 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. With Uptravi, there is now an oral option in all three established treatment pathways.”
Hans Dieter Kulla, president of Pulmonale Hypertonie e.v., said the new product launch is a major step when it comes to achieving optimal 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,” he said.