Approval postponed
The candidate for the most expensive medicine in the world was sent for revision
The world's first gene therapy for hemophilia has not been released to the market, according to a press release from Biomarin Pharmaceutical. The new drug could become the most expensive in the world, its cost was estimated at three million dollars. However, the US regulatory authorities ordered the company to wait until the end of 2021 – thus it will have to confirm that the therapy is effective in the long term.
Hereditary diseases for a long time differed from all others in that they were incurable in principle. Mutations carried by every cell of the body were impossible to correct until recently. There remained only replacement or maintenance therapy, to which the patient is attached for life. For example, in the case of hemophilia, human cells do not produce enough factor VIII, one of the blood clotting proteins. In order not to suffer from bleeding, the patient needs constant injections of the missing protein, which entails expenses of several hundred thousand dollars a year.
Recently, a way to solve such problems once and for all has appeared – with the help of gene therapy. A neutralized virus is injected into the patient's blood, which "infects" the cells of the body and injects a missing copy of a healthy gene into them. Gene therapy has already been approved for several diseases – for example, some types of blindness.
However, this method of treatment is often not only effective, but also expensive. We have already talked in detail about where the high prices for gene therapy come from – in the blog "Two million injection". In short, the fact is that manufacturers can afford to ask for a high price for a single injection precisely because it avoids annual costs of hundreds of thousands of dollars and pays for itself in just a few years. That is why the most expensive medicine in the world today is a gene therapy drug. This is "Zolgensma" – a single injection designed to relieve patients from spinal muscular atrophy. Its cost in the USA is $ 2.125 million.
The development of Biomarin Pharmaceutical was supposed to become even more expensive. According to preliminary estimates, for getting rid of hemophilia And the company was going to ask for about three million dollars. Biomarin Pharmaceutical has already successfully conducted clinical trials: we have already written that according to the results of phase 1-2, the amount of factor VIII in the blood of patients has steadily increased, and the number of necessary injections has decreased on average from 136 to 5 annually.
Nevertheless, according to the observations of doctors, the concentration of protein in the blood gradually fell. And despite the fact that at the end of the trials, it still fell within the limits of the norm for almost all patients (some doctors described the condition as "mild hemophilia"), this raised concerns about long-term consequences.
The company submitted an application for registration of its drug based on the results of Phase 1-2 trials, considering them sufficiently successful. The third phase is now underway, patients have already received therapy, and final results are expected in November 2021. However, the US Food and Drug Administration (FDA) refused registration, citing differences in the design of phases 1-2 and 3 of the tests. The management suggested that the company wait for the end of phase 3 and then provide full data on the sustainability of the effect of treatment. Thus, the possible registration of the world's first gene therapy drug for hemophilia is postponed for at least more than a year.
It can be assumed that the FDA's decision is related to recent disputes over the cost of "Zolgensma". To many, it seems excessive, and the only justification is a possible gain in the long run: if the drug proves effective, it will pay off the costs of other types of therapy in five years. However, much less time has passed since the approval of Zolgensma, and no one still knows whether the result of such treatment will be sustainable. In the case of hemophilia A therapy, patients have the right to demand even greater stability – the cost of three million dollars will pay off only ten years after the injection – which means that more serious tests are needed as part of the trials.
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