Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Limitations of current therapies

4 evidence topics · 16 sources

Summary

Before the approval of levacetylleucine in September 2026, no therapy was approved for ataxia-telangiectasia in any market, and no treatment was known to slow or stop the neurological decline. Published guidance rests on expert opinion and small non-randomized studies: the European Respiratory Society statement reports no randomized controlled trials of respiratory treatments and no placebo-controlled trials of prophylactic antibiotics or immunoglobulin replacement, and the United Kingdom guidance for children states that it is not an evidence-based guideline. Evidence for symptomatic drugs comes from open-label studies of amantadine (17 participants, 8 weeks), 4-aminopyridine (4 participants), and nicotinamide riboside (24 participants, 4 months). Oral betamethasone reduced the ICARS total score by a median of 13 points in a crossover trial of 13 participants; in a two-year study of 6 participants, the neurological scores of 4 participants deteriorated and all 6 had transient adrenal dysfunction. Intra-erythrocyte dexamethasone did not meet its primary endpoint in the ATTeST trial (175 participants treated) or in the NEAT trial (105 participants), and its sponsor ceased clinical development in January 2026. A 2026 systematic review of 13 studies with 314 participants concluded that no intervention had demonstrated sustained efficacy in adequately powered controlled trials. Physical, occupational, and speech therapies may maintain function and do not slow neurodegeneration.

Absence of a treatment that slows neurological decline

“There is no treatment known to slow or stop the progression of the neurologic deficits associated with A-T.” (opens the source at this quote in a new tab)

“Although physical and occupational therapies and exercise may help maintain overall physical well-being and quality of life, there are currently no treatments capable of slowing or halting the neurologic decline in A-T.” (opens the source at this quote in a new tab)

Systematic review of pharmacological treatment of ataxia in pediatric ataxia-telangiectasia

“Thirteen studies (3 RCTs, 10 single‐arm trials) involving 314 participants (mean age 10.87 years) were included.” (opens the source at this quote in a new tab)
“No intervention has demonstrated sustained efficacy in adequately powered controlled trials.” (opens the source at this quote in a new tab)
“The recurrent pattern of promising open‐label findings followed by negative phase 3 results highlights a translational gap attributable to methodological limitations, including small sample sizes, heterogeneous populations, and variable outcome measures.” (opens the source at this quote in a new tab)
“Current evidence remains insufficient to guide clinical practice.” (opens the source at this quote in a new tab)

Absence of an approved therapy before September 2026

“To date, there are no approved treatments for ataxia-telangiectasia anywhere.” (opens the source at this quote in a new tab)

“Ataxia telangiectasia is a rare, multisystem disorder with progressive cerebellar neurodegeneration and no approved treatments. The efficacy of corticosteroids, including erythrocyte encapsulated dexamethasone sodium phosphate (eDSP), which have been studied for two decades in this disease, has not yet been proven in randomised trials.” (opens the source at this quote in a new tab)

“There is no cure for ataxia-telangiectasia, and treatment options for the neurological symptoms of the disease have been limited.” (opens the source at this quote in a new tab)

“Until today, there were no approved treatments for A-T anywhere in the world.” (opens the source at this quote in a new tab)