Drug developers must undergo a stringent set of testing and regulatory steps in the journey towards drug approval. In the European Union (EU), the Committee for Medicinal Products for Human Use (CHMP) plays an important role in this process. The CHMP assesses marketing applications, considers recommendations and offers opinions, and contributes to medicinal regulation. According to the European Medicines Agency (EMA), Winrevair (sotatercept), a therapy for pulmonary arterial hypertension (PAH), received a positive CHMP opinion. Essentially, the CHMP recommended that the drug receive a marketing application; this would allow adults with PAH to be treated across the EU.
Winrevair is an activin signaling inhibitor. Activins are proteins that play a role in stimulating blood vessel cells (i.e. the cells that will eventually become blood vessels). PAH sees activin overactivity. Winrevair works by binding to the ActRIIA receptors (the same that activins bind to) and regulating new blood vessel development. Administered subcutaneously every three weeks, Winrevair improves symptoms such as exercise intolerance, especially when used in conjunction with other PAH treatments.
The positive CHMP opinion follows data from a clinical study which explored the treatment’s safety and efficacy. 323 individuals enrolled. All participants were treated with either a monotherapy or combination therapy before adding Winrevair. The study found that:
- Winrevair reduced exercise intolerance. Both immediately after treatment, and after a 24-week period, Winrevair improved walking ability and exertion capacity.
- Adverse reactions may include nose bleeds, headaches, diarrhea, dizziness, and rashes. While some more significant adverse reactions have been noted, such as heightened blood pressure and low platelets, the treatment is well-tolerated overall.
What to Know About Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension is a chronic and progressive hypertension (high blood pressure) affecting the lungs. Normally, pulmonary arterial pressure sits around 14 mm Hg at rest. In people with pulmonary arterial hypertension, pressure at rest is 25 mm Hg or more. This rare disorder causes thickened or hardened pulmonary arteries. As the heart works harder to pump blood, the heart weakens – and the rest of the body subsequently receives less oxygenated blood.
- Who does PAH affect? PAH can affect people of different ages. This condition is most commonly seen in people between ages 30 to 60. PAH is also 2x more common in females than males.
- What causes PAH? In 15-20% of individuals, genetic mutations cause PAH. However, pulmonary arterial hypertension also results from congenital heart disease, sickle cell disease, liver disease, connective tissue and autoimmune diseases, and HIV.
- Are there symptoms? Many people are asymptomatic (i.e. don’t have symptoms) before the disease progresses. When symptoms appear, they may include fainting and dizziness, shortness of breath that worsens during exercise or exertion, chest pain, fatigue, swelling of the lower extremities, and cyanosis (bluish skin discoloration).
- How do you treat PAH? Several treatment options exist for PAH, including Winrevair, Uptravi, Adempas, Opsynvi, and Opsumit (among others).
