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Existing studies show that earlier initiation of treatment with omaveloxolone leads to better outcomes. Patients who began treatment sooner have shown improvement over a longer period. Currently, this is the first and only therapy that slows the progression of the disease. In February 2024, the European Medicines Agency approved omaveloxolone for treating FA after it was registered by the FDA a year earlier.

Professor Maria Mazurkiewicz-Bełdzińska (Chair of the Polish Society of Pediatric Neurologists and Head of the Developmental Neurology Clinic at the Medical University of Gdańsk) cited an example from her center, where a nine-year-old patient with FA is being treated. She pointed to the challenge of deciding what to do with this patient, as the drug is not approved for very young children. The girl is still in good condition and could potentially benefit greatly from the treatment. However, Mazurkiewicz-Bełdzińska emphasized that the Emergency Access to Drug Technologies (RDTL) cannot be applied in her case because she falls outside the age specification in the Summary of Product Characteristics (SPC).

Professor Barbara Steinborn (Head of the Department and Clinic of Developmental Neurology at Poznań University of Medical Sciences) also described two patients—a seven-year-old diagnosed just weeks ago and a fourteen-year-old who started treatment in August 2024 after bioethical committee approval. The parents are also keen to treat the younger child, but obtaining RDTL approval appears highly unlikely, and the nearest center conducting clinical trials with pediatric participants is in the United States. Dr. Marek Migdał, Director of the Institute “Monument-Children’s Health Center,” emphasized that the current form and dosage of the drug are not tailored for young children. A clinical trial involving patients aged 2 to 15 is ongoing, but it is not expected to conclude until 2027.

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