MILAN (ITALPRESS) – The Italian Pharmaco Agency (AIFA) has approved the reimbursement of sotatercept, in association with other therapies, for the treatment of pulmonary arterial hypertension (IAP) in adult patients already in treatment for at least six months with triple therapy. The announcement was given today in Milan by MSD, a multinational biopharmaceutical company that developed the drug, during a press conference. Sotatercept also obtained from the European Medicines Agency (EMA) the qualification of Priority Medicine (PRIME), and the designation of an orphan drug for the IAP, as well as the status of innovation recognised by AIFA for the repaid indication, which certifies the added value of the drug compared to the already available therapies. Pulmonary arterial hypertension is a rare and progressive form of a wider condition called Pulmonary Hypertension (IP), characterized by the abnormalities of the pulmonary vascular wall that determine a rapid growth of the cells causing the thickening and narrowing of the pulmonary arteries. The progressive narrowing of the arteries leads to greater resistance to the blood flow and finally to the right ventricular failure. PAH results in a reduced life expectancy and those who suffer from it have an average survival of 5-7 years after diagnosis and affects about 400 thousand people globally, 3,500 in Italy. It is more common in women (especially in fertile age) than in men, with a ratio of 4 to 1. The meeting was attended by the delegated director and president of MSD Italia, Nicoletta Luppi, and Dr. Stefano Ghio, responsible for the SS Cardiomiopatie-Trapiantologia e Hypertension Polmonare dell’IRCCS Policlinico San Matteo di Pavia and president of the Italian Pulmonary Hypertension Network (IPHNET). “As MSD we invest in an important way in research and development. We are the first biopharmaceutical company in the world that invests in an important way in everything that is research and innovation”, said the president and managing director of MSD Italia Nicoletta Luppi. “For us what is important is to look at those therapeutic areas where there is still an important need for health. This therefore means that we invest both in pathologies that are very distributed worldwide but also in rare diseases because for us it is important also the individual patient, the single patient to whom we have to give answers”, said Luppi. “When we speak, as on this occasion, of pulmonary arterial hypertension we present today with sotatercept, an innovation that is certified by the Food and Drug Administration, certified as innovative also by the EMA and our AIFA with 4 phase 3 studies that attest not only innovation but also effectiveness. It is a drug that will be the first of a new class because we go to act on the cause of the disease with the possibility to regress the disease itself, its state of progress”, he added. MSD’s commitments “will also continue in this pathology. What I would like to emphasize is that both for rare diseases rather than for the most common diseases we have more than 50 new and innovative drugs in our pipeline in phase 2 and more than 30 innovative drugs in phase 3. We talk about drugs, we talk about vaccines, so we talk about therapy rather than prevention. Our commitment will continue to remain maximum in order to provide more and more answers to the need for health that remains unexpressed,” concluded the president and MSD. “The pulmonary arterial hypertension is a rare disease with infamous prognosis, it is a primitive disease of the pulmonary vascular bed in which the walls of these vessels are thickened, the lumen is reduced, the pressure inside of the blood vessels increasesto”, explained Dr. Stefano Ghio, president of the Italian Network of IPHNET Pulmonary Hypertension and head of the Unity Uncompensation, Cardiomyopathies and Pulmonary Hypertension of the Division of Cardiology of the IRCCS Policlinico S. Matteo di Pavia. “It is estimated that there are about 3,500 patients in Italy and is a disease that being rare is difficult to know, difficult to recognize, at least in the early stages and is difficult to cure outside of expert reference centers,” said Dr. Ghio. Who is suffering from this pathology, “it is very limited because he finds great problems to carry out all his daily activities starting from the most trivial: wash, go around, accompany children to school, work at home. She needs caregivers, she needs family support, so she is a very disabling disease,” she added. “Sotatercept has an innovative action mechanism, it is the first drug that has this mechanism of action that is a causal mechanism: It acts by inhibiting cellular proliferation, which is precisely the primum biological motivens of the disease itself. So it is a great promise of clinical improvement for patients,” he concluded. First in class organic drug of an innovative category of drugs, activna signal inhibitors, sotatercept is the first therapy to intervene directly on the biological mechanisms at the base of the disease, changing the course. The decision of the AIFA, approved by the CDA on 15 December 2025 and for which the publication of the determination of reimbursement in the Official Journal will make this decision active, follows the approval of the European Commission in August 2024 which was supported by the study of Phase 3 STELLAR. In the study, sotatercept has shown clinical benefits on several endpoints, including improved walking test in 6 minutes (6MWD) of 41 meters and reduced 84% of clinical worsening compared to standard therapy.
Thanks to its innovative action mechanism, sotatercept intervenes on the disequilibrium between pro-proliferation and anti-proliferation signals responsible for the pathological remodeling of lung arteries. The therapy is administered subcutaneously every three weeks and, after proper training, it can also be managed at home by the patient or caregiver. “Sotatercept represents a paradigm shift in the treatment of pulmonary arterial hypertension: Unlike the currently available medicines that modulate the metabolic pathways involved in the control of the endothelial function (Monoxide routes of Azoto, endotheline and prostacycline), this new drug directly interferes with the processes of cellular proliferation that are at the base of the vascular remodeling obstructive responsible for the increase of vascular resistancescular and lung pressure” said Nazzareno Cardiè professor. “Thanks to its innovative mechanism, the drug has been able to determine a significant reduction in pulmonary arterial resistances and also a significant improvement in the ability to exercise and morbid-mortality of patients suffering from pulmonary arterial hypertension.” Other studies, in addition to STELLAR, have demonstrated the safety and effectiveness of sotatercept. In ZENITH study, in high-risk adult patients, sotatercept resulted in a 76% reduction compared to standard endpoint therapy including death, pulmonary transplantation or IAP worsening. The study was interrupted early for excess clinical benefit than conventional therapy. Other studies have shown the effectiveness and safety of sotatercept even compared to other phenotypes of patients.
– photo xp9/Italpress –(ITALPRESS).
