Pharmaceutical company Promomed, together with leading specialized experts, has initiated a unique clinical study that could radically change approaches to treating spinal muscular atrophy (SMA). The goal of the project is to study the efficacy and feasibility of tandem (combination) therapy using the drug Risdiplam in patients of various age groups, including those who have previously received other pathogenetic treatments.
Modern Drug Against a Severe Disease
Spinal muscular atrophy is a severe genetic neurodegenerative disease that leads to profound disability in children and adults. In 2025, Promomed completed the full development cycle and received a registration certificate for the drug Risdiplam, intended for the treatment of patients aged 16 days and older.
This drug works by increasing the level of survival motor neuron (SMN) protein, which helps slow the progression of the disease. While taking the drug, patients experience increased survival rates, higher chances of achieving important motor function developmental milestones, preservation of the ability to swallow independently, and a reduced need for hospitalization and mechanical ventilation.
Why is Combination Therapy Needed?
Today, high-tech gene replacement therapy (for example, Zolgensma) exists in global practice; however, clinical experience shows that even it may not be sufficiently effective for a number of patients. At the same time, preliminary data indicate that a tandem approach—where a patient is prescribed Risdiplam after the administration of a gene replacement drug—can lead to significant improvement in their condition.
Currently, this approach is considered experimental and has not yet been included in official clinical guidelines. To gather the necessary evidence base, Promomed is launching a large-scale therapy evaluation program that will last for two years.
The study will include patients with both infantile-onset SMA and late-onset manifestations of the disease. It is important to note that access to the drug within the project will be granted not only to those who have previously used other methods of pathogenetic therapy but also to patients for whom treatment was previously unavailable.
Geography of the Study and Impact on Healthcare
Six major medical institutions have already joined the project. They include:
- Veltischev Clinical Research Institute of Pediatrics and Pediatric Surgery;
- Russian Center of Neurology and Neurosciences;
- National Medical Research Center for Children’s Health;
- Vladimirsky Moscow Regional Research Clinical Institute (MONIKI).
Victoria Shcherbakova, Medical Director at Promomed, emphasized the importance of the project:
“There are only three pathogenetic therapy drugs for SMA in the world, one of which is Risdiplam. Accumulating an evidence base on the efficacy of SMA therapy in late-onset disease and the possibility of using step-by-step therapy is an important stage in understanding the algorithms for managing patients with SMA.”
The successful completion of the study and the subsequent updating of clinical protocols will not only save more lives but also provide significant savings in budget funds, including the resources of regional healthcare systems.
