Expanding haemophilia treatment landscape with novel therapies |
Posted: January 11, 2023 |
Haemophilia B is commonly known as factor IX deficiency, and the prevalence of haemophilia B is 1 in 25,000 male births. It is less common than haemophilia A, which affects about 1 in 5,000 male babies born. There are mild to moderate illnesses in almost half of them. Treatment for haemophilia B mostly consists of replacement factor IX products (also plasma-derived or recombinant). The half-life, or length of effectiveness, of new variations of recombinant products that have recently been released, is extended via fusion with stabilising molecules and other modifications. To promote medical research for critical but uncommon disorders like haemophilia B, the FDA has a special designation. On November 22, the FDA approved the very first gene therapy, a pricey one-time treatment for the genetic blood-clotting illness haemophilia B. A modified virus is used by the pharmaceutical company CSL Behring, based in King of Prussia, Pennsylvania, to transport a gene to the patient's liver cells via Hemgenix. Patients with the condition are unable to produce factor IX, a protein necessary for blood coagulation. A single injection of Hemgenix should be sufficient to stop uncontrolled bleeding in persons with mild to severe haemophilia for eight years, and maybe longer, according to data from clinical studies. For instance, Hemgenix is regarded as an "orphan medicine" because it only benefits a tiny percentage of patients. Due to this designation, CSL Behring, the maker of Hemgenix, has been given exclusive access to the US marketplace for the ensuing seven years. Although the US government's use of incentives to promote scientific research is a beneficial strategy to hasten the development of therapies for rare conditions that would otherwise go untreated, this programme also has disadvantages. Furthermore, it argues that the US market is responsible for maintaining drug monopolies while other nations with pharmaceutical price restrictions profit from global research. Currently, US prescription medicine prices are two to six times higher than those in other countries. However, due to its exorbitant price, the therapy is the most costly medication in the world. Despite appearing to be successful, gene replacement therapy for the most prevalent form of haemophilia remains elusive. Fidanacogene elaparvovec, a haemophilia B gene therapy developed by Pfizer and Roche, is set to release pivotal data soon. The Benegene-2 trial, involving 45 male patients, is being finished by the partners. Despite the low patient population, there is a sizable pipeline of new medications for haemophilia. Although many represent slight advancements over existing methods, some drugs currently undergoing testing make use of more cutting-edge technology. Phase III trials are currently taking place for Fitusiran, an RNA-interference medication that "silences" a portion of the haemophilia gene's activity. It is anticipated to be an injection given once a month that inhibits antithrombins (which stop blood from clotting) and encourages the production of thrombin (which encourages it). It is designed to be used in both haemophilia A and haemophilia B. The development of several gene therapies is in its early stages. By inserting a corrected, functional copy of the gene into cells, they hope to return factor VIII production to normal levels without the need for additional treatment. There are currently concerns regarding this method's durability (how well it will perform over several years or more), and researchers worry that it may not be suitable for patients. However, many medical professionals, patients, and business analysts are optimistic about the future of drugs. While there is no cure for haemophilia right now, it is clear that research will continue to progress rapidly. With increasing levels of understanding of the disease and advances in gene editing technologies, scientists are hopeful that it is only a matter of time before a lasting cure is found. Finally, to keep up with the growth of genome-editing technology, institutional procedures, societal perception, and awareness must all be enhanced for the field's further advancement. BioIntel360 expects this technology to be seen as a promising solution that offers haemophilia sufferers and other genetic illness patients prospects for a higher quality of life, barring any ethical concerns.
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