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MSD’s WINREVAIR is intended to enhance patient exercise capacity and reduce PAH linked risks. Credit: Merck & Co., Inc. / Business Wire. The US Food and Drug Administration (FDA) has granted approval to Merck & Co (MSD)’s sotatercept-csrk, known by the brand name WINREVAIR, for the treatment of adults with pulmonary arterial hypertension (PAH). It is the first FDA-approved activin-signalling inhibitor therapy for PAH. PAH is a rare progressive and life-threatening disease in which blood vessels in the lungs narrow and thicken, leading to a huge strain on the heart. The treatment is indicated for use in PAH patients to improve exercise capacity, enhance WHO functional class and decrease the likelihood of worsening clinical events. The therapy, which had already received breakthrough therapy designation from the FDA, works by re-instating the balance between pro and anti-proliferative signalling to regulate the vascular cell proliferation linked to PAH. See Also: J&J’s nipocalimab gains FDA fast track status for FNAIT risk reduction Takeda’s ADZYNMA gains Japanese approval for cTTP treatment WINREVAIR is administered as a subcutaneous injection every three weeks. The latest approval is based on results from the STELLAR trial, a global, double-blind, placebo-controlled, multicentre, parallel-group Phase III study. It involved 323 adult patients and examined the effects of WINREVAIR compared to placebo, both given along with standard background therapies. There was a significant 41m improvement in the six-minute walk distance from the baseline at week 24 when WINREVAIR was added to background therapy. The treatment also substantially improved several secondary outcome measures, notably reducing morality or PAH clinical worsening event risks by 84% compared to background therapy alone. The company will make WINREVAIR available through select speciality pharmacies in the US by the end of next month. Merck Research Laboratories global clinical development senior vice-president, head and chief medical officer Dr Eliav Barr stated: “PAH remains a debilitating disease with high morbidity and mortality. “This approval of WINREVAIR is an important milestone and a testament to our science-led strategy and focus on the development of innovations that can help people affected by rare diseases like PAH.” Earlier this month, MSD completed the acquisition of Harpoon Therapeutics to bolster its oncology pipeline.

Foetal and neonatal alloimmune thrombocytopenia causes the immune system of a pregnant individual to mistakenly target the platelets of the foetus. Credit: Africa Studio / Shutterstock.com. Johnson & Johnson (J&J) has obtained fast track designation from the US Food and Drug Administration (FDA) for its nipocalimab to reduce foetal and neonatal alloimmune thrombocytopenia (FNAIT) risk in alloimmunised pregnant adults. The designation aims to expedite the development and review of drugs targeting serious conditions with unmet medical needs. This status also encourages frequent communication between the regulator and the treatment sponsor, in a bid to swiftly deliver new treatments to patients. A serious and rare condition, FNAIT causes the immune system of a pregnant individual to mistakenly target the platelets of the foetus, leading to potential bleeding and clotting issues. Nipocalimab is a fully human monoclonal antibody designed to reduce the levels of circulating immunoglobulin G (IgG) antibodies. See Also: Juvisé licenses J&J’s MS drug Ponvory for marketing outside North America Takeda’s ADZYNMA gains Japanese approval for cTTP treatment It is said to be the only therapy currently in clinical development to meet the requirements for alloimmunised pregnant individuals with FNAIT risk. Nipocalimab was awarded orphan drug designation by the FDA for its application in FNAIT In December 2023. Nipocalimab is also being investigated for its use in haemolytic disease of the foetus and newborn (HDFN), another alloimmune condition of pregnancy. Following promising Phase II safety and efficacy data from the UNITY clinical trial, the company is advancing nipocalimab into Phase III trials for HDFN. J&J vice-president and autoantibody and maternal foetal immunology disease area leader Katie Abouzahr stated: “Receiving fast track designation for nipocalimab in FNAIT underscores the urgency to address the unmet need for safe, effective, and targeted treatments to prevent FNAIT, a condition that could carry severe health consequences and even be fatal for the foetus or newborn. “We are committed to applying our decades of immunology leadership to pioneer innovative approaches to transform treatment for patients and their families affected by FNAIT and other alloantibody-driven diseases of pregnancy.”