According to a story from Business Wire, the therapeutics company Alnylam Pharmaceuticals recently announced that the company’s drug patisiran has gained approval from the European Commission. Patisiran was approved for the treatment of hereditary transthyretin mediated amyloidosis in adult patients that have stage 1 or 2 polyneuropathy. The approval will help provide access to a new treatment for patients affected by this disease.
Amyloidosis is a group of diseases in which a type of abnormal protein, called amyloid fibrils, accumulates in the body. Hereditary transthyretin mediated (hATTR) amyloidosis is a certain subtype of the disease that can appear in people who have a family history heart failure or neuropathies of unknown cause. Transthyretin is a protein that is normally found in the liver and is responsible for transporting vitamin A. Symptoms of amyloidosis include fatigue, weight loss, lightheadedness upon standing, shortness of breath, bleeding, and leg swelling. Amyloidosis is ultimately fatal, but hATTR amyloidosis has a better prognosis than most types; some patients can survive for a decade or more with good treatment. Treatment approaches vary depending on the type of amyloidosis that is present. To learn more about hATTR amyloidosis and amyloidosis in general, click here.
Patisiran was only recently approved last month by the U.S. Food and Drug Administration. The approval by the European Commission means that patients on both sides of the Atlantic will gain access to the drug at almost the same time. This treatment is a new breakthrough for patients with hATTR amyloidosis, as patisiran has the potential to provide relief from the symptoms of polyneuropathy, allowing patients improved quality of life and more ability to live their day to day lives.
In trials, the drug was able to improve the condition of hATTR amyloidosis patients by several measures. Overall, 51 percent of trial participants treated with patisiran reported improved quality of life at 18 months; only 10 percent of patients being treated with placebo reported improvements at the same time. Patisiran also caused only minor side effects, such as infusion related reactions. However, premedications can reduce the impact of these reactions.
Overall, this approval is exciting news for Europeans with hATTR amyloidosis.
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Check out the Amyloidosis Foundation to learn more about this condition!