The expert council of the "Circle of Good" proposed adding hemophilia A to the list of diseases

Revuforj is already being used at the N.N. Blokhin National Medical Research Center of Oncology, and it has proven its effectiveness during therapy. The expert council proposed to include in the categories for receiving the drug children with AML and ALL with refractory/recurrent course of the disease with the impossibility of standard chemotherapy, as well as with the presence of the chimeric gene KMT2A, determined by any available method. The decision to include the drug has been submitted for approval to the board of trustees of the state fund.

The expert council of the "Circle of Good" also considered the issue of including hemophilia A and the drug for its therapy Altuvoct in the list of nosologies. It is proposed to prescribe efanesoctocog alfa to children with established non-inhibitory form of hemophilia A, previously treated with F VIII, and to patients in whom the course of the disease cannot be controlled. For example, in case of repeated episodes (at least three times a year) of objectively confirmed hemarthrosis or bleeding requiring hospitalization, signs of progression of chronic hemophilic arthropathy or bleeding requiring additional administration of F VIII concentrate.

For patients with pulmonary arterial hypertension, the state fund's expert council proposed purchasing Opsamit in the form of dispersible tablets, which are developed specifically for children. Currently, the drug, according to the State Register of Medicines, is registered in the Russian Federation only in the form of film-coated tablets.

Head of the scientific department of pediatric cardiology and arrhythmology, head of the pediatric cardiology department of the Research Institute of Pediatrics and Pediatric Surgery named after Yu.E. Veltischev, PhD Irina Miklashevich, noted at a meeting of the expert council that the drug will be able to expand the possibilities of therapy for children with such a disease. Currently, for the treatment of pulmonary arterial hypertension, "Circle of Good" purchases Apbravi (selexipag) from J&J.

Macitentan is suggested for use in patients over two years of age with mean pulmonary artery pressure > 20 mmHg, pulmonary vascular resistance index ≥3 Wood units, pulmonary capillary wedge pressure ≤15 mmHg, and WHO FC II and III.

In early August 2025, the expert council of the "Circle of Good" decided to include Niemann-Pick disease type C and low-grade gliomas in the list of diseases, as well as to approve the categories of children who are indicated for the use of the corresponding drugs - two of which are not currently registered in Russia. We are talking about the drugs arimoclomol, tovorafenib and satralizumab (TN Enspring from the Swiss Roche).