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18.03.2026
PROMOMED has started a phase IV study of Risdiplam, a drug manufactured at the Biochemist JSC plant and intended for the treatment of orphan (rare) disease spinal muscular atrophy (SMA)*. The disease affects motor neurons, resulting in progressive weakness, atrophy, and muscle paralysis. The disease can make its debut not only in the first years of life, but also in old age, which requires special attention to timely diagnosis and the appointment of pathogenetic therapy. Treatment is designed to slow down the progression of SMA, prevent severe consequences of the disease, and thereby significantly improve the prognosis and quality of life of patients.
In 2025, PROMOMED completed the full development cycle and received a registration certificate for Risdiplam for the treatment of SMA in adults and children aged 16 days and older. Its action is aimed at increasing the level of motor neuron survival protein (SMN), which helps slow the progression of the disease. Taking Risdiplam increases the survival rate of patients, their ability to swallow remains, the likelihood of reaching the main stages of motor function development increases, and the need for hospitalization and artificial ventilation decreases.
The disability of patients of both childhood and adult age due to the development of genetic neurodegenerative diseases is the most difficult challenge of modern medicine. Additional attention to this problem was attracted by the story of Stephen Hawking, who suffers from amyotrophic lateral sclerosis (ALS) and the release of a gene replacement therapy drug (Zolgensma, INN onasemnogen abeparvovek) for the treatment of SMA. At the same time, clinical practice shows that even supertechnological gene replacement may not be effective enough in some patients. At the same time, research data indicate that tandem (combined) therapy, for example, taking Risdiplam after infusion with a gene replacement drug, demonstrates a significant improvement in the condition. Nevertheless, this approach remains experimental and has not yet been included in clinical guidelines. In this regard, doctors who monitor such patients, together with PROMMED specialists, initiated a study that will examine the possibility and feasibility of using the drug Risdiplam Prommed in patients of different age groups who previously received other pathogenetic therapy with SMA.
The study will allow us to collect evidence on the effectiveness and safety of this approach, which will form the basis for changing the clinical protocols for the treatment of this complex disease. In the future, after the creation of appropriate legal conditions, the availability of a domestic drug will increase the availability of drug therapy for patients with SMA and provide significant budget savings, including regional health systems.
Currently, six centers are included in the study, including the Scientific Research Clinical Institute of Pediatrics and Pediatric Surgery named after Academician Yu. E. Veltischev, the Russian Center for Neurology and Neuroscience, the National Medical Research Center for Children's Health, and the Moscow Regional Clinical Research Institute. Vladimir's.
The assessment of therapy will be carried out over two years in patients with SMA in infancy and in patients with late manifestation of the disease. Patients who have previously received other pathogenetic therapy, as well as those who previously had access to SMA therapy, will be able to participate in the study and receive the drug.
"There are only three drugs in the world for the pathogenetic therapy of SMA, one of which is Risdiplam. The accumulation of evidence on the effectiveness of SMA therapy in late manifestation and the possibility of using step-by-step therapy is an important step in understanding the algorithms for managing patients with SMA. Together with leading experts in the field of orphan diseases, we have developed a protocol and launched a study, the results of which will allow us to form these algorithms. After all, ensuring accessibility and expanding the possibilities of treating the most complex diseases is at the heart of PROMOMED's activities," said Victoria Shcherbakova, PROMOMED's medical director.
The decision to launch the commercial sale of Risdiplam can be made by PROMOMED Group solely taking into account the necessary legal conditions.
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