Name: Aqneursa (levacetylleucine) in Ataxia-telangiectasia: Special populations
Published: 2026-10-02T18:22:10-04:00

Pregnancy: risk summary

“There are no available data on AQNEURSA use in pregnant females to evaluate a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.” (opens the prescribing information at this quote)
“In animal reproduction studies, an increase in embryo-fetal death (post implantation loss/resorption), decrease in fetal body weight, and increase in external and skeletal malformations were observed in rats and rabbits when levacetylleucine was administered in pregnant rats and rabbits during the period of organogenesis.” (opens the prescribing information at this quote)
“These effects were observed in rats and rabbits at the doses that were approximately 1.4-fold and 6-fold, respectively, the maximum recommended human dose (MRHD) in patients taking 4 grams of AQNEURSA per day” (opens the prescribing information at this quote)
“The background risk of major birth defects and miscarriage for the indicated population is unknown.” (opens the prescribing information at this quote)
“In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.” (opens the prescribing information at this quote)

Pregnancy: animal data

“Increases in the mean number of resorptions, mean post-implantation losses and skeletal malformations (thoracic arch of the vertebra-fused thoracic arch, misaligned thoracic arch, hemicentric thoracic centrum, and misaligned thoracic centrum; vertebral column-scoliosis, and ribs-absent costal cartilage and short rib) were observed at a dose of 600 mg/kg/day, which is 1.4-fold the 4 g/day MRHD of levacetylleucine based on body surface area.” (opens the prescribing information at this quote)
“Increased embryo-fetal death (post-implantation loss/resorption), decreased fetal body weight, and increase in external (open or partially open eyes, hyperflexion of limbs) and skeletal malformations (misshapen maxillae and premaxillae, and small interparietal bones) were observed at the dose of 1250 mg/kg/day, which is 6-fold the MRHD of levacetylleucine based on body surface area.” (opens the prescribing information at this quote)

Pregnancy: European Union labeling

“There are no data on the use of levacetylleucine in pregnant women. Studies in animals have shown reproductive toxicity (see section 5.3).” (opens the source at this quote in a new tab)
“Aqneursa is not recommended during pregnancy and in women of childbearing potential not using contraception.” (opens the source at this quote in a new tab)
“In embryofoetal development studies, levacetylleucine did not induce adverse developmental effects at doses up to 1 000 mg/kg/day in rats (2.0-fold human exposure).” (opens the source at this quote in a new tab)
“external and skeletal malformations were observed at 1 250 mg/kg/day (7.1-fold of human exposure) with a no observed adverse effect level of 675 mg/kg/day (4.9-fold human exposure).” (opens the source at this quote in a new tab)

Lactation

“There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production.” (opens the prescribing information at this quote)
“The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.” (opens the prescribing information at this quote)

“A decision must be made as to whether to discontinue breast-feeding or to discontinue/abstain from Aqneursa therapy, taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman.” (opens the source at this quote in a new tab)

Females and males of reproductive potential

“AQNEURSA may cause embryo-fetal harm when administered to a pregnant female” (opens the prescribing information at this quote)
“For a female of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA.” (opens the prescribing information at this quote)
“Advise a female of reproductive potential to use effective contraception during treatment and for 7 days after the last dose if AQNEURSA is discontinued.” (opens the prescribing information at this quote)

“No human data on the effect of levacetylleucine on fertility is available. Animal studies indicate no effects of levacetylleucine on male or female fertility (see section 5.3).” (opens the source at this quote in a new tab)

Pediatric use in A-T and the 15 kg weight limit

“The safety and effectiveness of AQNEURSA for the treatment of ataxia in patients with A-T have been established in 47 pediatric patients weighing ≥ 15 kg in Trial 2” (opens the prescribing information at this quote)
“The safety and effectiveness of AQNEURSA have not been established in pediatric patients weighing <15 kg.” (opens the prescribing information at this quote)

Pediatric use in NPC and supporting pharmacokinetic data

“The safety and effectiveness of AQNEURSA for the treatment of NPC have been established in 23 pediatric patients weighing ≥15 kg in Trial 1.” (opens the prescribing information at this quote)
“Use of AQNEURSA for this indication is supported by evidence from one adequate and well-controlled study in adults and pediatric patients weighing ≥15 kg with additional pharmacokinetic data from 40 adults and 17 pediatric patients who participated in two open-label studies” (opens the prescribing information at this quote)

Pediatric limits in the European Union: 6 years and 20 kg

“The safety and efficacy of Aqneursa in children aged less than 6 years or weighing under 20 kg have not yet been established. No data are available.” (opens the source at this quote in a new tab)

“For children weighing 15-20 kg, it is predicted a 24% higher exposure however no PK data are yet available.” (opens the source at this quote in a new tab)
“No PK data from children aged below 6 years have been submitted as part of this application, whereas children of at least 4 years were planned to be enrolled.” (opens the source at this quote in a new tab)
“A dose range-finding toxicity study in juvenile rats was already completed, and a final study report is not yet available for the definitive study.” (opens the source at this quote in a new tab)

Geriatric use

“NPC and A-T are largely diseases of pediatric and young adult patients.” (opens the prescribing information at this quote)
“Clinical studies of AQNEURSA did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients.” (opens the prescribing information at this quote)

“Only two subjects from study IB1001-301 had an age over 65 years (66 and 67 years) and none were above 68 years.” (opens the source at this quote in a new tab)

Renal and hepatic impairment

“The effect of renal impairment, hepatic impairment, or pregnancy on levacetylleucine pharmacokinetics is unknown.” (opens the prescribing information at this quote)

“The effect of renal or hepatic impairment on the PK of levacetylleucine has not been studied.” (opens the source at this quote in a new tab)

“No formal PK study investigating the effect of renal impairment on levacetylleucine PK was performed, since the product is rapidly metabolized into L-leucine and acetate and both are used in further catabolic pathways.” (opens the source at this quote in a new tab)
“No impact of renal impairment is expected.” (opens the source at this quote in a new tab)
“No impact of hepatic impairment is expected.” (opens the source at this quote in a new tab)
“No studies were done in patients with renal impairment. Patients with moderate and severe hepatic insufficiency were excluded from levacetylleucine clinical studies.” (opens the source at this quote in a new tab)

Age and sex subgroups in the pooled NPC safety data

“There were no relevant differences in safety variables across age groups in the pooled studies.” (opens the source at this quote in a new tab)