Gene therapy is now dawning: FDA approves the first gene drug that can silence RNA
Ribonucleic acid (RNA) molecules "guide" our body to produce certain proteins. The idea behind RNA interference is that small molecules can be developed to neutralize these RNA messengers, effectively silencing the expression of certain genes that may cause harmful health effects. These destructive molecules are called small interfering RNAs (siRNAs). Recently, according to foreign media reports, the US Food and Drug Administration (FDA) approved the first drug to use this method for adult clinical treatment. This drug is used in patients with hereditary transthyretin amyloidosis (hATTR).
After breakthrough discoveries that how RNA interference was used to "silence" certain genes nearly 20 years later and related research won the Nobel Prize for more than a decade, the development of these siRNA clinical treatments was slower than originally hoped, researchers in the past Twenty years have spent a great deal of time looking for efficient ways to deliver these molecules to specific target organs and to produce nanoparticles that protect siRNA from degradation.
The FDA drug recently approved by the US Food and Drug Administration is groundbreaking, but can only be used on a rare disease that affects only about 50,000 people worldwide. The drug, called Onpattro (patisiran), disrupts the RNA machinery that produces transthyretin, a protein that promotes the accumulation of amyloid deposits in the body. The FDA has approved the designation of new drugs for the treatment of adult patients with symptoms of peripheral neuropathy (polyneuropathy) caused by hATTR. The disease can also cause a decline in heart function.
Alnylam, the research and development company behind the drug, said that there are three siRNA drugs in the later trials, and I hope to get approval for these drugs in the next one to two years. At the same time as FDA approval, the EU will soon approve Onpattro for public use. Although each patient currently spends $450,000 per year for ongoing treatment. The company guarantees that if the drug does not work in some patients, it will provide reimbursement to the insurer. It sounds like a little bit of domestic health care product advertising, of course, if it is really amazing or merit.
In China, nervous system drugs are also one of the drugs with the most rapid sales growth in recent years. In 2002, nervous system drugs occupied 5.9% of the entire pharmaceutical market share, and the total sales amount increased by 9.4%.
With the increasing trend of urbanization and aging, the elderly population in China has exceeded 120 million, and the incidence of geriatric diseases, especially mental diseases, is increasing year by year. With the increase of residents' income, the elderly's requirements for quality of life also increase. As a result, the demand for drugs to treat mental illnesses such as Alzheimer's has soared. In WHO drug classification, psychotropic drugs and nootropic drugs are grouped into one class. We briefly analyzed the development trend of this class of drugs through the clinical drug use data collected from more than 300 domestic key hospitals by Chinese Pharmaceutical Association.
The overall market size of this class of drugs is about 1.22 billion yuan, the consumption sum increased by 5.0% in 2002, but the sales quantity increased by 11.2%. The main reason why the consumption sum did not increase with the sales quantity is that several main varieties of this class of drugs, such as piracetam, cobalt bin amide and ginkgo biloba leaf, etc. In 2002, the price of selling units fell sharply due to bidding. Among these drugs, ginkgo biloba preparation occupies the largest market share, accounting for more than 1/3 of the market share, ganglioside ester and cobalt bin amide combined also occupy nearly 1/3 of the market share, piracetam and other "sitan" drugs occupy nearly 10% of the market share, the rest of the drugs occupy less than 5% of the market share. At present, more than 20 ginkgo leaf production enterprises, piracetam production enterprises more than 70, have entered the stage of price competition, profit repeatedly diluted.
In the first-line varieties, except ginkgo biloba leaves preparation maintained vigorous vitality, the growth of other varieties were weak, especially the traditional variety piracetam suffered from bidding
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