The Board of Trustees of the Circle of Goodness has approved the purchase of a drug for the treatment of hemophilia A

The drugs in question are Altuvoct (efanesoctocog alfa) from Swedish Orphan Biovitrum for patients with hemophilia A, Revuforj (rebumenib) from the American pharmaceutical company Syndax for the treatment of acute myeloid (AML) and acute lymphoblastic (ALL) leukemia, as well as the drug Opsamit (macitentan) from J&J in dispersible tablets for children with pulmonary arterial hypertension.

The specified form of Opsumit, which was developed specifically for the treatment of children, is not yet registered in Russia. Currently, the drug is available for use only in the form of film-coated tablets.

The Expert Council of the Circle of Good Foundation proposed to include hemophilia A in the list of diseases in early August 2025. At the same time, the members of the Council indicated that therapy with Altuvoct should be available for children with established non-inhibitory hemophilia A who had previously received treatment with F VIII, and patients whose 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, with signs of progression of chronic hemophilic arthropathy or bleeding requiring additional administration of F VIII concentrate.

It was noted at the meeting that the Revuforj drug proposed for the treatment of AML and ALL is already being used at the N.N. Blokhin National Medical Research Center of Oncology. Representatives of the center confirmed that it has demonstrated its effectiveness during therapy. The drug can be used to treat children with refractory/recurrent disease with the impossibility of standard chemotherapy, as well as with the presence of the chimeric KMT2A gene, determined by any available method.

Over the past few months, the board of trustees of the Circle of Good has approved the inclusion of three diseases at once in the list of diseases whose treatment is financed by the state fund: neuromyelitis optica (Devic's disease), Niemann-Pick disease type C, and low-grade glioma.

Opticomyelitis is a chronic autoimmune disease that affects the optic nerves and spinal cord, leading to profound disability and death. For children with this diagnosis, the state fund will purchase satralizumab. In Russia, only one drug with this INN is registered - Enspring from the Swiss Roche.

For patients with Niemann-Pick disease type C and low-grade glioma , the drugs arimoclomol (TM Miplyffa) from the American Zevra Therapeutics, which is not yet registered in Russia, as well as tovorafenib (TM Ojemda) from the American Day One Biopharmaceuticals, will be purchased.