The Circle of Kindness Foundation has expanded the criteria for prescribing three vital drugs, including Zolgensma (onasemnogene abeparvovec) — a drug for spinal muscular atrophy from Swiss Novartis. Following a meeting of the expert council, the Foundation’s press service reported adjustments to the categories of children eligible for medications with INN mercaptamine bitartrate and idebenone.
The Ministry of Health found compelling clinical data supporting the safety and effectiveness of Zolgensma in patients with four or more copies of the SMN2 reserve gene, which is crucial for producing SMN protein to preserve motor neuron cells.
Consequently, the agency has removed the restriction on the number of copies of the reserve gene from the state register, noting that approximately 95% of such patients develop the disease in childhood, but in some patients symptoms may not appear immediately. The Circle of Kindness highlighted that Zolgensma is registered in several countries, including the USA, Japan, Israel, and Argentina, without restrictions on the number of SMN2 gene copies, while such restrictions remain in the European Union.
In addition to these changes, the meeting expanded the categories of children eligible for treatment with idebenone (Raxone, for Leber’s hereditary optic neuropathy) in terms of age, visual acuity, and phase of the disease. Changes were also made to the categories of children eligible for therapies involving mercaptamine bitartrate (or cysteamine bitartrate). It is planned to differentiate between new therapy applications and those for continued therapy.
Previously, the All-Russian Union of Patients advocated for increasing the age limit for patients covered by the Circle of Kindness Charitable Foundation, an initiative supported by the Civic Chamber, which noted the earlier decision to raise the age limit to 18 years.
The Circle of Kindness Foundation was established in January 2021, following a decree signed by Russian President Vladimir Putin. Its primary mission is to enhance the organization and financial provision of medicines and medical products for children suffering from life-threatening and rare diseases, with a list of 100 conditions covered by the foundation.
