Katrina Barry was diagnosed with a rare and serious condition called pulmonary arterial hypertension (PAH) at the age of 25. After finding out that she had only two to five years left to live, she signed up for a study to test an experimental drug called sotatercept. This drug offered her a potential lifeline by corralling a growth factor that is overproduced by people with PAH. This marked the beginning of her treatment journey with the drug, which she started in March 2020 amid the Covid-19 pandemic. She diligently followed safety protocols to protect herself from getting infected with Covid while continuing her treatment.
The US Food and Drug Administration recently approved the medication that Barry has been taking for four years, which is now sold under the brand name Winrevair. This biologic drug is designed to trap proteins called activins that are overproduced in PAH, preventing the walls of blood vessels from thickening and causing stress on the heart. The drug has a different mechanism of action compared to existing treatments for PAH and has shown promising results in clinical trials.
While the drug has shown efficacy in increasing the distance walked by patients in a six-minute walk test, there are still many unknowns about its long-term benefits and potential side effects. The drug was fast-tracked for approval by the FDA due to the urgency of the condition, as untreated PAH can be fatal. The drug’s approval has brought hope to patients with PAH, offering a new treatment option for a disease that has limited therapeutic options.
Despite experiencing some side effects such as nosebleeds and dizziness, Barry has seen significant improvements in her condition since starting sotatercept. She has been able to resume many activities that she enjoyed before her diagnosis, such as hiking, skiing, and pursuing a master’s degree in public health. The drug has allowed her to reduce her reliance on oxygen therapy and remove bulky medical equipment, improving her quality of life significantly.
The cost of the drug, estimated at around $243,000 per year, has sparked debate about its affordability and accessibility for patients. While the wholesale cost is negotiable with insurance providers and may be more affordable for patients, its price tag is higher than some analysts had hoped for. However, the impact of the drug on improving patients’ quality of life and potentially reversing the progression of the disease suggests that it could be a game-changer for the treatment of PAH.
Dr. Aaron Waxman, Barry’s doctor and director of the pulmonary vascular disease program at Brigham and Women’s Hospital, has seen remarkable improvement in patients treated with sotatercept. The drug has the potential to reverse the disease and improve outcomes for patients with PAH. Barry believes that the drug has brought hope to those struggling with this fatal disease, providing a beacon of light for those who may have lost hope. As more data on the drug’s long-term benefits and safety profile emerges, it has the potential to revolutionize the treatment landscape for PAH patients.
