May 16, 2024
Vyndaqel for Rare Heart Condition Now Available on the NHS
Patients on the NHS suffering from a critical heart condition will now have access to a groundbreaking medication that can significantly lower the chances of hospitalization and death.
The medication, tafamidis, is the first approved treatment in England for patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM), a rare heart condition where protein deposits accumulate in the heart, often leading to heart failure and potentially fatal outcomes.
Individuals with this progressive illness can now take advantage of a daily capsule after the recommendation to provide this innovative treatment to over a thousand qualified patients.
Tafamidis works by slowing the accumulation of harmful protein deposits. Clinical trials demonstrated a 41% reduction in mortality risk for patients taking the medication compared to those on a placebo. Moreover, patients in the tafamidis group experienced fewer hospitalizations than those receiving a placebo.
This drug is available on the NHS starting today (13 May), funded temporarily by the Innovative Medicines Fund after approval from the National Institute of Health and Care Excellence (NICE).
Approximately 1,500 people in England have been diagnosed with ATTR-CM.
Common symptoms of the condition include shortness of breath, palpitations, abnormal heart rhythms, fatigue, fainting, and chest pain.
Previously, treatment options for ATTR-CM were limited, primarily focusing on managing symptoms and providing supportive care.
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