EU CHMP Recommends Approval of Merck’s Winrevair to Treat PAH

EU CHMP Recommends Approval of Merck’s Winrevair to Treat PAH

By, Admin 1 July 2024

Overview

Merck, known as MSD outside of the United States and Canada, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the approval of Winrevair (sotatercept), in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH in adult patients with World Health Organization (WHO) Functional Class (FC) II to III, to improve exercise capacity. Winrevair was previously granted Priority Medicines (PRIME) and orphan designation by the EMA for the treatment of PAH. The European Commission (EC) will now review the CHMP recommendation, and the EC’s decision on the marketing authorization application of Winrevair in the EU, Iceland, Liechtenstein and Norway is expected in the third quarter of 2024.

Merck Research Laboratories on PAH

“PAH is a progressive and debilitating rare disease,” said Dr. Joerg Koglin, senior vice president and head of general medicine, global clinical development, Merck Research Laboratories. “There is still a significant need for new therapies for patients. This positive opinion marks the first step toward expanding access to our first-in-class activin signalling inhibitor therapy, Winrevair, for eligible adults with PAH in Europe. We are pleased with the CHMP recommendation and look forward to the European Commission’s decision.”

Winrevair: Administration

Winrevair is administered once every 3 weeks as a single injection under the skin and may be administered by patients or caregivers with guidance, training and follow-up from a healthcare provider.

Phase 3 STELLAR Trial

  • The CHMP recommendation is based on data from the Phase 3 STELLAR trial of Winrevair on top of background PAH therapy compared to background therapy alone. 
  • Winrevair demonstrated a statistically significant and clinically meaningful improvement in 6-minute walk distance, the study’s primary endpoint, and on multiple important secondary outcome measures, including reducing the risk of death from any cause or PAH clinical worsening events. 
  • These results were published in The New England Journal of Medicine.

From Hannover Medical School

  • “Winrevair is the first activin signalling inhibitor therapy and is proposed to modulate the vascular proliferation underlying PAH,” said Dr. Marius Hoeper, Hannover Medical School, Germany. 
  • “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. It is very encouraging that physicians in Europe may soon have a novel treatment option available that targets a new PAH treatment pathway.”

EMA & Winrevair in the Treatment of PAH

  • Through the CHMP recommendation, the EMA is the second regulatory body to recognize the potential of Winrevair in the treatment of PAH based on a review of pivotal efficacy and safety data. 
  • In March 2024, Winrevair (sotatercept-csrk) was approved by the US Food and Drug Administration (FDA).

STELLAR Study

The STELLAR study (NCT04576988) was a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which 323 patients with PAH (WHO Group 1 FC II or III) were randomized 1:1 to Winrevair (target dose 0.7 mg/kg) (n=163) or placebo (n=160) plus stable background therapy administered subcutaneously once every 3 weeks.

PAH Etiologies

  • The most common PAH etiologies were idiopathic PAH (59%), heritable PAH (18%), and PAH associated with connective tissue diseases (CTD) (15%). 
  • Most participants were receiving either three (61%) or two (35%) background drugs for PAH, and 40% were receiving prostacyclin infusions. 
  • The mean time from PAH diagnosis was 8.8 years. Patients had a WHO FC II (49%) or III (51%) at baseline.

Winrevair: Approval by FDA

  • In the US, Winrevair is FDA-approved for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events. 
  • Winrevair, the first activin signalling inhibitor therapy approved to treat PAH, improves the balance between pro-proliferative and anti-proliferative signalling to modulate vascular proliferation. 
  • In preclinical models, Winrevair induced cellular changes that were associated with thinner vessel walls, partial reversal of right ventricular remodelling, and improved haemodynamics.

Winrevair is the subject of a licensing agreement with Bristol Myers Squibb.

About Pulmonary Arterial Hypertension (PAH)

  • Pulmonary arterial hypertension (PAH) is a rare, progressive and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. 
  • Approximately 40,000 people in the US and 30,000 people in the EU are living with PAH. The disease progresses rapidly for many patients. 
  • PAH results in significant strain on the heart, leading to limited physical activity, heart failure and reduced life expectancy. 
  • The five-year mortality rate for patients with PAH is approximately 43%, based on data from the REVEAL registry (patients enrolled between March 2006 and December 2009).

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