Sotatercept: A Breakthrough Therapy for Pulmonary Arterial Hypertension in Spain

Pulmonary arterial hypertension (PAH) is a rare disease that can be disabling for those who suffer from it. It presents a significant challenge in its diagnosis and management because more than 50% of cases have no identified cause, which, along with the nonspecific nature of its symptoms, makes detection difficult and delays diagnosis. A new treatment, sotatercept, now offers hope.

Sotatercept offers new hope for PAH patients, marking Europe’s first activin signaling inhibitor and advancing personalized treatment in Spain

MSD presented sotatercept this Thursday, a new drug that, according to Joaquín Mateos, Medical Director of MSD in Spain, represents a milestone in the company’s discoveries in cardiovascular and cardiorespiratory diseases. This drug is used in combination with other treatments for pulmonary arterial hypertension (PAH) and is indicated for the treatment of PAH in adult patients in WHO functional class II or III to improve exercise capacity.

Sotatercept is the first activin signaling inhibitor therapy approved in Europe for the treatment of PAH, as it improves the balance between pro- and anti-proliferative signaling to modulate vascular proliferation. In preclinical models, an analog of the treatment induced cellular changes associated with thinner vascular walls, partial reversal of right ventricular remodeling, and hemodynamic improvement.

In Spain, sotatercept has been included in the portfolio of products and services of the National Health System since the beginning of August, which will allow patients to access this new therapeutic option.

Alejandro Cruz Utrilla, a specialist in the Pulmonary Hypertension Unit at the 12 de Octubre University Hospital in Madrid, commented that PAH has a prevalence of around 50 cases per million inhabitants. “Historically, it was thought that this rare disease affected young women between the ages of 35 and 45. However, the most recent population data from European and American registries reflect a different reality. We are beginning to see more patients, both men and women, around 60 years of age with cardiovascular and respiratory comorbidities,” he said.

Gregorio Pérez, a pulmonologist and coordinator of the Multidisciplinary Pulmonary Vascular Unit at Dr. Negrín Hospital in Las Palmas de Gran Canaria, explained the impact of the disease on patients, who often experience shortness of breath, lack of energy, and weakness, primarily due to the reduction in cardiac output. “All of this has a very negative impact on their functional capacity and their daily activities. Therefore, it is not surprising that they are prone to depression, anxiety, restlessness, and social and professional isolation.”

Pulmonary arterial hypertension (PAH) is a condition that causes increased pressure in the pulmonary artery and impairs right ventricular function. In recent decades, significant progress has been made in prognostic stratification of the disease, based on the state of right ventricular function. Furthermore, pulmonary vasodilator drugs have emerged that reduce pressure in the pulmonary artery by dilating small pulmonary vessels and thus improving right ventricular function.

Alejandro Cruz Utrilla explained that sotatercept has an innovative characterization, “it is the first drug of its class to inhibit activin signaling and it is a different mechanism of action”

In this regard, strengthening clinical care is essential for biomedical advances and new therapies to achieve their goal of benefiting patients. After treatment with sotatercept begins and during follow-up, a periodic risk reassessment should be performed, classifying risk into four strata (low, intermediate/low, intermediate/high, and high) to determine the need to escalate treatment if the patient is not at low risk.

In Spain, there are recognized centers of excellence such as the 12 de Octubre University Hospital and the Hospital Clínic of Barcelona. “Moving towards a more equitable and proactive approach, based on regular reassessments and multidisciplinary teams, is as crucial as the development of new treatments,” concluded Cruz Utrilla.

For his part, Gregorio Pérez commented that in addition to the novel therapeutic approach of sotatercept, other drugs are also being investigated, such as protein kinase modulators.

“These drugs can also be administered via inhalation, which is very interesting because they can act more directly. There is a lot of hope placed in gene and cell therapies, which will somehow restore the balance between BMPR2 and transforming growth factors, and another promising advance will be personalized medicine. We have to give each patient the treatment that benefits them most, and for that we need biomarkers, the most precise genotypic specification possible, and early diagnosis so that patients come in at less advanced stages.”

Clinical trials for sotatercept and other new therapies

Joaquín Mateos, medical director of MSD in Spain, commented on the importance of collaboration between the different actors involved in patient treatment and referred to public-private collaboration in clinical trials, saying, “At MSD, we believe that an excellent example of public-private collaboration is the implementation of clinical trials, where a private pharmaceutical company and researchers, who in many cases are researchers from public entities, participate.”

“At MSD, we have a robust clinical research program in the area of ​​cardiorespiratory diseases, encompassing prevalent conditions such as COPD, as well as rare diseases like pulmonary arterial hypertension. For this latter condition, we are developing therapies with innovative mechanisms of action. This demonstrates our commitment to providing more treatment options for patients and advancing the improvement of their quality of life,” said Mateos.

He explained that in 2024, MSD Spain promoted 194 clinical trials, “a significant number, which shows the commitment and strength of our subsidiary, with clinical trials being the basis of innovation and the advancement of science,” he said.

The clinical trial has led to the approval of this new drug by regulatory agencies. “It is a phase 3 clinical trial in patients with pulmonary arterial hypertension, which has shown positive data regarding patient functionality and a positive balance in terms of the drug’s efficacy and safety. We invested heavily in research, both in phase 2 and phase 3. These results have allowed the National Health System to recently include sotatercept in its portfolio of products and services, making it available to patients. We sincerely believe that this drug will represent a very important advance in patient treatment.”

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(Featured image by Simone van der koelen via Unsplash)

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