The suggestion that 3-year-old boy Mark from the Krasnodar Territory was denied treatment for “the world’s most expensive drug” not for medical, but for financial or political reasons, raises the issue of the possibility of “import substitution” on the agenda. An affordable drug should cost less than $2 million per injection.
Only one injection gives hope for saving life, the ability to breathe, sit, walk. Other well-known drugs such as Spinraza or Evrysdi need to be taken constantly and are therefore less convenient.
It is known that the American Zolgensma from Novartis Gene Therapies, Inc. is an adenoviral-associated vector gene preparation.
The product description and instructions for use in the United States indicate 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 features: the drug is toxic. Possible side effects include acute liver damage and liver failure.
Outside of FDA-approved use, the drug cannot be legally administered in America unless the family agrees to participate in a clinical trial that Novartis has been running since August 2021 for patients of other ages.
Another conflict over the appointment of Zolgensma gives Russian scientists an additional chance: it’s time to stop relying 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, in fact, they are intended. The National Welfare Fund also has money. The point is to “close” internal needs and provide humanity with an inexpensive and high-quality Russian-made drug.
Prescribing medications for other than medical reasons, as well as refusing to provide medical care, is illegal and fraught with criminal liability with a prison term.