Scottish Agency Recommends Uptravi for PAH Treatment Through National Health System

Alice Melão, MSc avatar

by Alice Melão, MSc |

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Scottish patients with pulmonary arterial hypertension (PAH) can now be treated with Actelion‘s Uptravi (selexipag) through the country’s National Health System (NHS) after a positive recommendation from the Scottish Medicines Consortium (SMC).

However, the use of this therapy will be restricted to adult patients in WHO functional class 3 whose symptoms are not manageable or do not tolerate other available PAH therapies.

Uptravi was included among five approved therapies that SMC recommended be made available on NHS Scotland. The medication was endorsed through SMC’s Patient and Clinician Engagement (PACE) process, which is used when considering therapies for rare, life-threatening diseases.

The agency’s decision was supported by positive opinions provided by patient groups and clinicians at the PACE meeting. Their testimonies demonstrated that Uptravi — an oral treatment taken twice daily — could improve PAH patients’ quality of life, and offer them greater freedom in their regular daily routines without requiring the use of nebulizers.

“I am pleased the committee was able to accept these five medicines for use by NHS Scotland,” Alan MacDonald, MD, chairman of the SMC, said in a press release. “Selexipag can help reduce the burden of treatment for those with PAH.”

Uptravi is a selective activator of the prostacyclin receptor that helps vessels in the lungs to relax and widen, easing blood flow. It was found to be effective in reducing the symptoms of PAH in patients with idiopathic disease, as well as those with connective tissue disease-associated PAH.

Approved in Europe in May 2016, Uptravi can be used as a combination therapy with endothelin receptor antagonists and/or phosphodiesterase type 5 (PDE-5) inhibitors. Treatment should be started at a dose of 200 micrograms twice daily, every 12 hours, which can be increased on a weekly basis if necessary up to a maximum of 1,600 micrograms twice daily.

Uptravi’s marketing approval had a setback in February 2017 when five patients died in France after taking the therapy. A few months later, the European Medicines Agency concluded that physicians could continue to prescribe the medication after a thorough safety data review confirmed that Uptravi did not increase the risk of death, and that the death rate for the therapy was in line with other approved PAH therapies.

The other four therapies recommended by SMC include Spinraza (nusinersen) for type 1 spinal muscular atrophyBavencio (avelumab) for Merkel cell carcinomaStivarga (regorafenib) for advanced liver cancer; and Kyntheum (brodalumab) for severe plaque psoriasis.

A Conversation With Rare Disease Advocates