The claim that 3-year-old boy Mark from the Krasnodar Territory was refused treatment with “the world’s most expensive drug” not due to medical reasons, but due to financial or political motives, brings up the issue of potentially producing the drug domestically. A more affordable drug should be priced below $2 million per injection.
A single injection offers hope for saving lives, the ability to breathe, sit, and walk. Other well-known drugs like Spinraza or Evrysdi require constant intake and are therefore less convenient.
The American Zolgensma from Novartis Gene Therapies, Inc. is a gene preparation using an adenoviral-associated vector.
The product description and usage instructions in the United States state that it has been tested and approved by the FDA for use in children with SMA up to 2 years of age. Studies on the efficacy of Zolgensma in patients with advanced SMA have not been conducted. The European Medicines Agency has approved the use of Zolgensma for children weighing up to 21 kg.
In addition to the extremely high price and low storage temperatures, Zolgensma has other characteristics: it is toxic. Possible side effects include acute liver damage and liver failure.
Other than the FDA-approved use, the drug cannot be legally administered in America unless the family agrees to participate in a clinical trial conducted by Novartis since August 2021 for patients of other ages.
Another disagreement over the prescription of Zolgensma offers Russian scientists another chance: it’s time to stop depending on America and its technologies.
The Gamaleya Center has experience working with adenoviruses, as it has already developed a drug against the Ebola virus based on them. The Russian Sputnik V is also constructed from an adenoviral vector and there is evidence of its effectiveness against Covid-19.
SSC “Vector” should also be able to work with “vectors”. Currently, the development of an anti-SMA drug is being carried out by the Russian biotechnology company Biocad.
If we agree that Sputnik is not worse, but better than AstraZineca or Janssen (also based on adenoviruses), then the Russian “shot” against SMA can potentially compete with the American one from Novartis.
The Russian Direct Investment Fund or the State Fund “Circle of Goodness” could “invest” in this good cause. For this, they are exactly intended. The National Welfare Fund also has money. The point is to meet internal needs and offer humanity an inexpensive and high-quality Russian-made drug.
Prescribing medications for reasons other than medical, as well as refusing to provide medical care, is illegal and may lead to criminal liability with a prison sentence.
