The US Food and Drug Administration’s (FDA) external panel of experts voted Wednesday to expand the use of a drug aimed at reducing genetic material in the treatment of a rare heart condition that can cause organ damage.
The FDA’s panel of experts approved by a majority of 9 votes to 3 that the benefits of this drug outweigh the risks associated with it in the treatment of patients suffering from a rare form of heart muscle disease known as cardiomyopathy, which is caused by a condition hereditary transthyretin-mediated amyloidosis (hATTR). This rare condition develops in people who have abnormalities in the TTR gene.
In patients with hATTR amyloidosis, a protein called transthyretin in the blood becomes abnormal and breaks down rapidly. This damaged protein accumulates in amyloid deposits in various tissues and organs in the body, including around nerves, affecting normal functions and leading to loss of sensation, heart problems, eye, kidney and thyroid disorders.
However, experts have expressed concern about how significant the benefits of this drug are. Dr. Edward Kasper, one of the expert panel members, noted that while no major risks have been reported, the benefits appear to be marginal.
The FDA, which usually follows the recommendations of its panel of experts but is not required to do so, will make a final decision on the drug by Oct. 8.
In previously published briefing papers, FDA advisors emphasized that the drug’s effects compared to a placebo are limited, of uncertain clinical significance, and may not be detectable by patients.
This drug, developed by US biopharmaceutical company Alnylam Pharmaceuticals, uses a Nobel Prize-winning technology called “small interfering RNA” (siRNA) to attack and reduce specific genetic material and stop the production of a deadly protein.
The active substance in this medicine, called patisiran and used in Onpattro, is an siRNA, a very short sequence of synthetic genetic material designed to bind to the genetic material of the cells responsible for transthyretin production and block it. This leads to a reduction in the production of abnormal transthyretin and, by implication, to a reduction in the symptoms associated with hATTR amyloidosis.
In 2018, patisiran became the first treatment of its kind licensed in the United States to treat nerve damage in adult patients with hereditary ATTR, and it was also approved in the European Union for the same indication.
Government data from the Centers for Disease Control and Prevention (CDC) indicate that approximately 5,000 to 7,000 new cases of hATTR-related cardiomyopathy are identified annually in the United States.
Source: 360medical.ro
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2023-09-16 18:31:09
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