FDA approved the first drug for the etiologic treatment of cystic fibrosis
Last reviewed: 23.04.2024
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The US Food and Drug Administration (FDA) has approved the use of the first drug for the etiologic therapy of cystic fibrosis, reports AP.
Cystic fibrosis is a hereditary disease that is caused by any mutation in the gene of the regulator of transmembrane conduction in CFV (cystic fibrosis transmembrane conductance regulator). This protein, being an ion channel, regulates the transport of water and chlorine ions through the membranes of the cells of the mucous membranes. Due to changes in CFTR structure, the viscosity of mucus increases, which leads to its accumulation in the respiratory, digestive and genitourinary systems. This, in turn, is fraught with severe chronic infections of the relevant organs, because of which many patients do not live to adulthood.
Until now, there was no etiologic (acting on the cause of the disease) cystic fibrosis treatment - in such patients only symptomatic therapy was used.
The new preparation Kalydeco (ivacaftor, ivacafluor), developed by the American pharmaceutical company Vertex Pharmaceuticals, improves the work of CFTR, in which, due to amino acid mutation, glycine in the 155th position is replaced with aspartic acid (this variant of the protein is designated G551D-CFTR). In particular, it increases the probability of opening this ion channel under the action of cyclic adenosine monophosphate (cAMP) and increases the chlorine flow through the membranes, which is disturbed in cystic fibrosis.
The experimental course of ivakaftora improved the lung function indices by an average of 10% and helped them gain weight (cystic fibrosis usually has a deficit in body weight), and also significantly improved well-being.
Kalydeco is produced in the idea of tablets containing 150 milligrams of active substance and designed to be taken twice a day. The FDA approved it for use in adults and children older than six years. The efficacy and safety of medication in young children is currently being investigated.
The mutation, in which ivacafluor is active, lies at the heart of only four percent of cases of cystic fibrosis, that is, of about 30,000 Americans suffering from this disease, the drug can help only about 1200 (the drug will be prescribed only after the patient's G551D-CFTR is detected). Most cases of cystic fibrosis are associated with a mutation in which at 508 CFTR position there is no amino acid phenylalanine (variant of the protein CFTR-ΔF508). For this form of the disease Vertex has developed the drug VX-809, which is still undergoing clinical trials.
"Although [ivacafluor] is not meant for most patients, it proves that you can detect a mistake in the genes and rationally develop a drug that eliminates the problem," said the director of the cystic fibrosis program at the University of New York at Buffalo Dryci Borovits (Drucy Borowitz).
The development of Ivakaftor cost Vertex hundreds of millions of dollars. $ 75 million donated the Cystic Fissure Foundation. The annual course of treatment with this drug will cost 294 thousand dollars, which makes it one of the most expensive drugs.
"The price of a medicine is assigned in accordance with its value for such a small group of patients," Vertex executive vice president Nancy Wysenski explained to analysts. She noted that patients without medical insurance or those whose family income does not exceed 150 thousand dollars a year, the company will provide the drug for free. In addition, it will cover 30% of the Kalydeco price for certain groups of insured patients.
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