Injection for $3,000,000
In Britain, they came up with a way to fight the "royal disease"
Lisa Brovko, "Apostrophe"
The European Medical Agency has approved the use of a genetic drug produced in the UK that can cure hemophilia, a blood clotting disorder. However, one dose costs about $3 million.
Why is this drug revolutionary
During the trials, a single injection of Roktavian (the so-called drug) deprived nine out of ten patients of hemophilia symptoms, and they no longer needed daily injections that were previously necessary to control their condition.
"When the results of the latest Roctavian study were announced in March, we were euphoric. For its approval in the UK, the price (about $ 3 million, – ed.) is obviously a cause for concern. But hemophilia is a disease that lasts a lifetime, and its treatment costs the NHS about $3,640 per month per patient. This has been increasing for decades. After one injection of Roktavian, they can live a normal life," explained Rashid Kazmi, a hematologist at the University Hospital of Southampton NHS Foundation Trust.
What is hemophilia?
Hemophilia is a rare disease caused by a genetic defect, which means that the body has problems producing a protein called factor VIII, which is necessary for blood clotting. There are 8,700 people living with this diagnosis in the UK alone.
Most people with hemophilia produce almost no factor VIII, so they are vulnerable to brain hemorrhages and internal bleeding from organs. And with the help of Roctavian, also known as valoctocogene roxaparvovec, fragments of the missing genetic code necessary for the production of factor VIII are implanted into a harmless virus delivered to the patient by a dropper. The virus then enters the liver and transfers the genes, allowing the body to independently produce factor VIII.
In the latest study, 134 patients received Roktavian infusion. After a year, 90% were effectively cured – they had normal or close to normal levels of factor VIII. Two years later, some relapsed, but 75 percent were still either moderately affected or quite healthy.
What are the side effects
Side effects from the preparation are nausea, headaches, inflammation of the liver. Another disadvantage: Roktavian is a one–time treatment, and if the patient relapses, a second dose cannot be administered, since the drug uses a virus to deliver genetic material to the liver, and the body's immune system creates antibodies that attack and destroy it if it gets into the body again.
"Given so much data on the safety and use of the drug, I think it will be available in the UK within a year," Kazmi expressed hope, noting that the drug approval authority of the National Health Service (National Institute for Health and Care Excellence) will agree on a significant discount for the manufacture of an expensive drug.
Who had hemophilia
One of the most famous carriers of hemophilia in history were Queen Victoria of Great Britain and Tsarevich Alexei Nikolaevich of Russia. Probably, the mutation in the genotype of Queen Victoria was de novo, that is, for the first time in the family, because this disease was not observed in the families of her parents. And the mentioned Tsarevich Alexei was a descendant of Queen Victoria. For these reasons, hemophilia is also called "Victorian disease" or "royal disease". Also, sometimes in royal families, marriages between close relatives were allowed to preserve the title, which is why the frequency of birth of a person with hemophilia was higher.
Portal "Eternal youth" http://vechnayamolodost.ru