EMA branch favors approving Winrevair to treat PAH across EU

Therapy, approved in US for adults, works to increase exercise capacity

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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An advisory committee of the European Medicines Agency (EMA) is recommending that Winrevair (sotatercept) be approved for the treatment of pulmonary arterial hypertension (PAH) in the Europe Union.

Specifically, the Committee for Medicinal Products for Human Use (CHMP) issued an opinion recommending that Winrevair to be used in combination with other PAH therapies to improve exercise capacity among adult PAH patients in World Health Organization (WHO) functional class 2 or 3.

The European Commission will take the opinion into account when making its final decision, due in the coming months. Applicable across the European Union, Iceland, Liechtenstein, and Norway, the decision does not have to align with the CHMP’s recommendation, but it often does.

If approved, Winrevair will be the first medication in its class — called activin signaling inhibitors — to be approved for PAH in Europe, according to the treatment’s developer, Merck (known as MSD outside of North America).

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“This positive opinion marks the first step toward expanding access to our first-in-class activin signaling inhibitor therapy, Winrevair, for eligible adults with PAH in Europe,” Joerg Koglin, MD, PhD, senior vice president and head of general medicine, global clinical development, at Merck Research Laboratories, said in a company press release.

“We are pleased with the CHMP recommendation and look forward to the European Commission’s decision,” Koglin added.

Winrevair previously was granted Priority Medicines (PRIME) and orphan drug designations by the EMA for the treatment of PAH. Such designations are intended to speed the therapy’s development toward regulatory approval by offering financial incentives or regulatory support.

The injectable medication was approved in the U.S.  in March, where it is indicated to increase exercise capacity, improve WHO functional class, and reduce the risk of clinical worsening in adults with PAH.

As an activin signaling inhibitor, Winrevair works to block the activity of proteins implicated in the uncontrolled cell growth that causes blood vessels to narrow in PAH. It is administered as an under-the-skin (subcutaneous) injection once every three weeks, with the possibility for self-administration after proper training.

The CHMP recommendation is backed by data from the Phase 3 STELLAR clinical trial (NCT04576988), which showed that Winrevair on top of standard-of-care PAH therapy led to statistically significant and clinically meaningful improvements in exercise capacity relative to background therapy alone among adults with PAH.

Trials ongoing in at-risk adults with PAH and in pediatric patients

Its use also was associated with better heart health and a lower risk of death or events tied to PAH’s clinical worsening, and more treated patients had improvements in WHO functional class than those given a placebo.

“Based on the clinical benefits demonstrated in primary and secondary outcome measures in the Phase 3 STELLAR study, Winrevair has the potential to play a critical role in the treatment of appropriate adults with PAH,” said Marius Hoeper, MD, acting director of the department of respiratory medicine and infectious diseases at Hannover Medical School in Germany.

“WINREVAIR is the first activin signaling inhibitor therapy and is proposed to modulate the vascular proliferation underlying PAH,” Hoeper added. “It is very encouraging that physicians in Europe may soon have a novel treatment option available that targets a new PAH treatment pathway.”

Several global and ongoing clinical trials are continuing to evaluate Winrevair’s potential benefits in PAH.

These include the open-label SOTERIA study (NCT04796337), which is looking at Winrevair’s long-term safety and efficacy in adults with PAH who participated in previous clinical trials into the therapy.

Meanwhile, the ongoing Phase 3 ZENITH (NCT04896008) and HYPERION (NCT04811092) studies are looking at Winrevair’s effects in different adult PAH populations. Zenith, now fully enrolled, is evaluating PAH patients in WHO functional class 3 or 4 who are considered to be at a high risk of dying, while HYPERION is enrolling newly diagnosed adults (PAH diagnosis within the last year) in WHO functional class 2 or 3 thought to be at an intermediate-to-high risk of disease progression.

Finally, the MOONBEAM Phase 2 trial (NCT05587712) is assessing Winrevair’s safety and pharmacological properties in children and adolescents, ages 1-17, with PAH. It is recruiting up to 42 eligible patients at sites worldwide.

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