The Center recommends considering the possibility of conducting negotiations on managed access agreements (MAAs) for the medicinal product agalsidase alfa, which is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (α-galactosidase deficiency) – state HTA conclusion published https://surl.li/ipifqg
Why is this important?
The current standard of treatment for patients with Fabry disease is intravenous enzyme replacement therapy. To date, patients have access to therapy only with the medicinal product agalsidase beta, however, to ensure an individual approach and reduce the risk of medicinal product shortages, it is necessary to expand the list of products that patients with this orphan disease can receive.
Without proper treatment, the disease inevitably leads to the development of renal and cardiac failure and early strokes, which significantly shortens patients' life expectancy and causes premature disability.
According to state HTA data, agalsidase alfa and agalsidase beta may have a similar efficacy profile in terms of all-cause mortality, the incidence of renal complications and the incidence of cardiovascular disorders, as well as a similar safety profile. Currently, there are no clear data indicating a clinical advantage of one medicinal product over another. The choice and prescription of agalsidase alfa or agalsidase beta is made by the physician on the basis of an individual approach, taking into account the patient's clinical condition and needs.
It is expected that providing access to the medicinal product agalsidase alfa will increase the coverage of patients with Fabry disease with vital enzyme replacement therapy, which is critically important for preventing irreversible organ damage and reducing the level of disability.
