According to a story from Rare Disease Report, the pharmaceutical company Alnylam Pharmaceuticals announced the results of Phase 3 trials for its experimental drug patisiran. The drug is in the development for the treatment of hereditary ATTR amyloidosis. The data suggests that the treatment could have significant potential for this patient group.
Amyloidosis describes a number of different diseases that cause an abnormal protein called amyloid fibrils to accumulate in body tissue. These diseases are caused by the misfolding of a specific protein; which protein is involved depends on the type of amyloidosis. While some types of amyloidosis are acquired, ATTR is an inherited type. The protein that is affected in ATTR amyloidosis is called transthyretin. Symptoms can vary depending on the type, but typically include fatigue, diarrhea, enlargement of the tongue, bleeding, loss of sensation, feeling faint upon standing, weight loss, leg swelling, and enlargement of the spleen. Treatment options also vary depending on the type; in ATTR, a liver transplant can often cure the disease, because the mutated transthyretin that causes amyloids originates in the liver. To learn more about hereditary ATTR amyloidosis, click here.
The APOLLO Phase 3 trial was the largest clinical trial of hereditary ATTR patients so far and involved a total 225 patients from across the world. The results of the trials showed the potential effectiveness of patisiran. The data showed a 50 percent decrease in hospitalizations and overall mortality for those that received the drug over a period of 18 months. This suggests that patisiran has the potential to stop or even reverse the progression of hereditary ATTR amyloidosis, which could completely change the outcomes for many patients with this disease in a positive way.
Though benefits of patisiran were present in all stages of the illness, early intervention is still recommended in order to achieve the best outcomes and quality of life for patients. In addition, the drug appeared to be relatively safe for patients in the study as well. The vast majority of qualified patients saw enough improvement to continue treatment in an open label extension study. Clearly, patisiran has the potential to make a real difference in the lives of patients with hereditary ATTR amyloidosis.
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Interested in learning more about amyloidosis? Check out the Amyloidosis Foundation.