Overcoming mistrust: SMA patients in Russia have started to receive a domestic drug

A few years ago, patients diagnosed with spinal muscular atrophy were virtually doomed: there was no cure for this severe orphan disease. But in less than a decade, the situation has changed radically. Today, doctors already have several drugs in their arsenal that are used in the treatment of spinal muscular atrophy (SMA). And in April 2024, the first domestic drug for the treatment of this disease was registered, and many Russian patients are already receiving it. However, some parents of children with SMA still have concerns about the Russian drug. Izvestia tells us what causes them and how doctors fight the distrust of parents of patients.

From despair to hope

The situation with the treatment of SMA in Russia has changed rapidly. Until 2019, children with type 1 SMA most often did not survive to the age of two, and doctors had no way to help them. At the end of 2019, the first medicines for the treatment of this disease were registered in the country, and in 2021, the Circle of Goodness Foundation began its work in Russia to provide the necessary drugs for children with SMA. By 2024, Russian doctors already had several drugs for the treatment of SMA at their disposal, and young patients were fully provided with them at the expense of the Circle of Goodness. The choice of the drug is made by the doctor in each specific case, depending on the individual characteristics of the patient and the course of the disease.

In April 2024, another step was taken towards guaranteed provision of medicines to Russian patients: the first domestically produced drug for the treatment of spinal muscular atrophy was registered in Russia. The Russian pharmaceutical company Generium produced an analog of Spinraza, one of the most expensive drugs in the world, produced by the pharmaceutical concern Biogen. The domestic drug, named Lantesense, became the world's first full chemical analog of nursinersen, the active ingredient of Spinraza.

Difficulties of choice

To date, the majority of patients receiving therapy are children. According to the Circle of Goodness Foundation, more than 600 children have received nursinersen since the funding mechanism was established. Until recently, the drug was supplied from abroad, but today more and more domestic analogs are being supplied to medical institutions. Many parents are understandably worried about the change of the drug.

"We, parents, are worried about the changes, because we are talking about the life and health of our children. The peculiarity of SMA treatment is such that the result of using any drug is not immediately noticeable - weeks and months must pass for visible changes. Of course, parents are afraid of losing the results they have been achieving for years. When the first drugs were introduced, people were ready to take any drug that promised improvement, because there was no alternative. Now, when there is more than one treatment option, when through the Circle of Goodness children consistently receive the drugs prescribed by doctors, of course, parents want their children to receive the best, most effective medicine," said Olga Germanenko, founder and director of the SMA Families Foundation.

Many parents of patients were particularly confused by the fact that clinical trials of the Russian drug have not been conducted on humans. It is for this reason that there are fears that unexpected side effects may be discovered in the process of its use. According to medical experts, such worries for patients are quite natural: after all, they do not know the details and peculiarities of production and registration of medicines. Nusinersen has long been well studied clinically and has been used to treat patients with SMA since 2017. Generium has reproduced nursinersen, which is completely identical in physicochemical and biological properties to the original drug, and proved it by large-scale equivalence studies. Since nursinersen is a chemically synthesized drug, this means that we are not talking about a bioanalog, which cannot be one hundred percent identical to the original drug, but essentially the same substance.

And in accordance with both Russian and international regulatory requirements, clinical trials are not required to register a chemically reproduced drug with an intrathecal method of administration, i.e. into the space surrounding the spinal cord - it is enough to prove that both drugs are identical. And the necessary experience of use in humans has already been obtained in full during the research and use of the original drug.

"Identity has been achieved in full, including for all excipients and impurities. At the beginning of July 2024, the first adult patients in the Moscow region received injections of Lantesense. In August, we gave the first injections to patients at the Scientific Center for Neurology. The other day some of them have already received their fifth injection of the drug. Of course, in order to fully assess its effect, we need to perform a number of tests over a fairly long period of time - from 10 to 14 months. But already now four patients in our center have noted a positive reaction of the back muscles, which is the most vulnerable part of the body in patients with SMA. No specific side effects have been noted so far," said Sergei Klyushnikov, a neurologist and candidate of medical sciences.

A plan for the future

Today, the domestic drug is being used to treat adults and children with SMA in dozens of Russian regions: more than 200 injections of the drug have already been performed. At the same time, according to official data, there are no registered adverse reactions to Lantesens injection in the Pharmacovigilance 2.0 database of the AIS of Roszdravnadzor. Which means, according to medics, it is likely to gradually replace the foreign-made drug in Russian clinics.

"The domestic drug is cheaper, in addition, it eliminates dependence on foreign supplies - the manufacturing company is ready to meet all available demand. Therefore, we can confidently say that both those who have already received therapy and new patients will switch to it," says Sergey Klyushnikov.

"Generium is open to dialog with both the professional community and parents of young patients. Online meetings are held, where you can get answers to your questions, and people are invited to the production site to see the drug synthesis process with their own eyes.

What should be done with mistrust? According to experts, it will settle down with time - primarily due to the accumulation of information, knowledge and experience. At the moment, an observational post-registration study has begun, during which doctors are collecting data on the safety and efficacy of the drug.

"We are grateful to Generium for their openness - they held several meetings with the patient community and answered the most difficult questions. Openness is very important in this issue. It is important that patients share their experiences, talk about the results, the specifics of the effects. It is important for doctors to share clinical results, both at professional congresses and in communication with patients. I think that as clinical data accumulate, fears and concerns will be eliminated," says Olga Germanenko.