A Treatment for Hereditary ATTR Amyloidosis Just Got Approved by the FDA

UPDATE: Patisiran has been approved! Check out the story here.


According to a story from Xconomy, the U.S. Food and Drug Administration could be close to approving a new drug that uses a unique mechanism called RNA interference. The drug is called patisiran, and if approved it could be a new treatment option for patients with hATTR amyloidosis. Mary O’Donnell who leads the Amyloidosis Foundation, says that patients in the amyloidosis community have been waiting years for a new treatment option to be approved.

About Amyloidosis

Amyloidosis is a group of unusual, progressive diseases that are characterized by the buildup of abnormal amyloid fibrils in body tissue. There are 30 different kinds of amyloidosis, and each one is distinguished by the misfolding of a certain protein that results in the creation of amyloid fibrils. The cause of amyloidosis is varied and depends on the type; they can be acquired or can have a genetic basis. Hereditary transthyretin-associated amyloidosis (hATTR) is an example. Symptoms of amyloidosis include lightheadedness, fatigue, leg swelling, bleeding, shortness of breath, and weight loss. Prognosis depends on the type, but for most cases, patients can only survive for a few years without treatment. Hereditary ATTR amyloidosis tends to have a better survival rate than most other types. To learn more about amyloidosis, click here.

Limited Treatment Options

Patients with hATTR are typically treated with drugs that are originally intended for other purposes, and the approval of patisiran would make it the first drug specifically meant for the treatment of the disease. The drug consists of an infusion which is given once every three weeks. Data suggests that the drug can halt the progression of hATTR and can even improve function in some cases.

Until patisiran gains approval, the options for patients with hATTR amyloidosis are limited. One option is a liver transplant; this can actually cure the disease, but the operation carries a lot of potential risk. The other most common option is the use of an anti-inflammatory drug called diflusinal. Unfortunately, this option also carries the potentially for serious side effects.

Preparing For Approval

Meanwhile, the patient community eagerly awaits the approval of patisiran, but they also anticipate that the price of the drug could be very high and are in the process of coming up with support systems that can help patients afford treatment.

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