In St. Petersburg, for the first time in Russia, the family of a child with SMA is trying to obtain through the courts the issuance of one of the most expensive drugs in the world - Zolgensma.
This gene therapy drug is intended to treat the life-threatening disease of spinal muscular atrophy, in which all muscles, including the respiratory muscles, gradually atrophy.
There are only three registered SMA drugs in the world.
Two of them - "Spinraza" and "Evrisdi" - are registered in Russia, they are required to be accepted for life.
Zolgensma is the third drug currently in the process of registration in the Russian Federation.
A single injection of this medicine is enough to recover, but the injection costs about 160 million rubles.
In Russia, you can count on the fingers of children who received the "shot of life", but they either got into the program of free access to the drug, winning the lottery from the manufacturer, or collected the necessary amount privately.
"Give birth to another"
Svetlana and Aleksey Gepalov moved to St. Petersburg almost two years ago.
Previously, they lived in Chita, then they moved to Irkutsk, at the place of work of their husband, a serviceman.
Kostya was born there in September 2018.
Almost immediately Svetlana noticed that the boy stopped moving his legs and raising his head.
On the 15th day of life, they saw a doctor, a specialist suspected SMA.
When Kostya was one and a half months old, the results of genetic tests came, which confirmed the diagnosis.
“We were immediately told: such children do not live, accept, give birth to another, but leave this one,” recalls Svetlana Gepalova.
Kostya's parents learned about the recruitment of the experimental drug Branaplam into the program of clinical trials (it was created by the same company that now produces Zolgensmu - Novartis), and since at that time there was not a single drug registered in Russia for SMA, they decided to use this chance.
“I had to move to St. Petersburg with lightning speed,” says Svetlana.
- Fortunately, my husband was able to transfer here.
But everything that we had in Irkutsk - an apartment bought with a military mortgage, all things - we had to leave. "
Kostya was selected for the program and is still participating in it.
"Branaplam" acts on the same principle as "Spinraza", and "Eurysdi" does not heal a "broken" gene, but stops the progress of the disease, and it must also be taken for life.
“There is an effect from“ Branaplam ”: his hands are working very well now, Kostya sits with support, holds his head while sitting, there is minimal, but still support on his feet.
He loves music, dance, repeats all the movements that are available to him, copies how the characters of his favorite cartoon transform from cars into robots, - Svetlana lists.
- For the first type of SMA, this is just wonderful!
But I saw children who were given Spinraza in the same condition as Kostya's.
And according to my feelings, the effect there was more powerful. "
The problem is that clinical trials of Branaplam have not yet been completed, how long they will last, whether the drug will be registered later and whether patients will receive it in the future is unknown, says Svetlana Gepalova.
While Kostya is listed as a participant in the trials, doctors cannot “transfer” him to lifelong therapy with a similar drug.
Therefore, two councils - regional and federal - came to the decision to recommend treatment with a drug that is enough to take once, that is, "Zolgensmoy".
Weight is more important than age
First, the Gepalovs tried to get the necessary medicine "peacefully": they turned to the Ministry of Health, from there they were sent to the Health Committee of St. Petersburg, where the family was again sent to the Ministry of Health.
“From last December to August, we were playing football from department to department,” Svetlana continues. “In May, we realized that officials were in no hurry to help us.
At the same time, we opened a training camp for Zolgensmu.
And on August 18 they filed a lawsuit ”.
The Gepalovs' case was the first in Russia when the plaintiffs are trying to obtain through the court the purchase and provision of Zolgensma.
The first hearing in the Kuibyshevsky District Court took place on 24 August.
And on August 27, the day before one of the regular meetings, in the absence of Kostya himself or his parents, a third council was held, which decided that the drug was not indicated for the child.
The decisions of the two previous councils, which recommended, on the contrary, to apply Zolgensma, were considered obsolete by the time the process began and these documents were not taken into account.
© Photo from personal archive
According to the lawyer Ildar Tukhvatullin, representing the interests of the Gepalovs, the procedure was carried out in violation of the law: according to him, the document with the conclusions of the commission lacks signatures and seals.
“The judge found the document inadmissible evidence and recognized that the consultation was illegal,” he told RT.
- The arguments of the city health service that the medicine is not in the standards of medical care, that the drug is expensive, that the disease is not included in the list of orphan diseases, that there are no clinical recommendations - all this was dismissed by the court.
However, the court still refused to provide the drug.
The fact is that up to 45 days are allocated for the purchase of a drug that is not registered in Russia, and the same amount is allocated for its import.
By that time, Kostya would have already been over two years old, and it is the age in the American instructions for "Zolgensme" that is listed as one of the strict contraindications for admission.
The court relied on this, although, according to Ildar Tukhvatullin, now experts believe that weight is a more important criterion than age.
“In the EU, since the summer of 2020, there has been a rule that the age of a child for Zolgensma therapy is not important, the main thing is that he weighs 13.5 kg - Kostya now has 10.5 kg.
- he explained.
- Moreover, we have a notarized opinion and American specialists, who also say that they are ready to conduct therapy with this drug if the child is older than two years old, but fits in weight.
That is, even by their standards, which the court referred to, it fits. "
"It hurts to hear that"
In October, the Gepalovs filed an appeal with the St. Petersburg City Court, but on December 15 he upheld the decision of the Kuibyshevsky District Court.
Now Kostya's parents have filed a second claim with the district court.
They have in their hands a fresh decision of the council, which, like the first two commissions, believes that the boy needs Zolgensma.
The date of the new meeting has not yet been set.
The collection for the medicine is also ongoing.
So far, 17.4 million rubles out of the 168 required have been donated for the injection for Kostya.
“Kostya loves animals very much, and for two years we gave him a parrot, Dasha.
He loves to communicate with other children, but now we try not to go especially anywhere, so that no one nearby sneezes or coughs.
When he sees a child running ahead of him, he says “top-top,” says Svetlana Gepalova.
- It hurts to hear that.
Well, nothing, baby, - I think then, - now we will somehow get the medicine and it will be "top-top".
Moreover, as doctors say, he has no atrophied muscles in the body.
That is, the legs are functioning by themselves, the muscles are in good condition, they just need a push. "
RT sent inquiries to the Ministry of Health of Russia and the Health Committee of St. Petersburg.
At the time of publication of the material, no answers were received.