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Aqneursa (levacetylleucine)

Ataxia-telangiectasia

Also known as IB1001
Manufacturer
IntraBio
Last updated
206 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatment

Other product development or post-marketing obligations required by the FDA

10 evidence topics · 5 sources

Summary: postmarketing obligations for levacetylleucine

The FDA approval letter for the A-T supplement (NDA 219132/S-002, 18 September 2026) lists no postmarketing requirement or postmarketing commitment and contains no pediatric assessment section. It requires submission of structured product labeling within 14 days, patent information within 30 days, and compliance with the reporting requirements for an approved NDA. The original approval letter of 24 September 2024 for the NPC indication set eight postmarketing requirements under section 505(o) (PMR 4683-1 to 4683-8), comprising seven nonclinical toxicology and carcinogenicity studies and one clinical drug-drug interaction trial with a P-gp probe substrate, and one postmarketing commitment (PMC 4683-9), a food effect trial. The agreed final report dates run from December 2024 to June 2029. That letter states that the application was exempt from the Pediatric Research Equity Act assessment requirement because the product has orphan drug designation for the indication. The prescribing information revised in September 2026 states that animal studies of carcinogenic potential and of fertility have not been conducted.

A-T supplement approval letter: labeling and reporting obligations

“As soon as possible, but no later than 14 days from the date of this letter, submit the content of labeling [21 CFR 314.50(l)] in structured product labeling (SPL) format using the FDA automated drug registration and listing system (eLIST), as described at FDA.gov.” (opens the source at this quote in a new tab)

“All promotional materials that include representations about your drug product must be promptly revised to be consistent with the labeling changes approved in this supplement, including any new safety-related information [21 CFR 314.70(a)(4)].” (opens the source at this quote in a new tab)

“We remind you that you must comply with reporting requirements for an approved NDA (21 CFR 314.80 and 314.81).” (opens the source at this quote in a new tab)

Pediatric Research Equity Act status

“Because this drug product for this indication has an orphan drug designation, you are exempt from this requirement.” (opens the source at this quote in a new tab)

Postmarketing requirements from the 2024 approval: basis for the nonclinical studies

“of serious risks with levacetylleucine use related to embryofetal development, fertility, early embryonic development, pre- and postnatal development, and carcinogenicity.” (opens the source at this quote in a new tab)

“Furthermore, the active postmarket risk identification and analysis system as available under section 505(k)(3) of the FDCA will not be sufficient to assess these serious risks.” (opens the source at this quote in a new tab)

“Therefore, based on appropriate scientific data, FDA has determined that you are required to conduct the following studies:” (opens the source at this quote in a new tab)

Postmarketing requirements from the 2024 approval: nonclinical studies
RequirementStudyFinal report submission
PMR 4683-1“Conduct an embryo-fetal development (EFD) toxicity and toxicokinetic study with levacetylleucine in the rodent.”“12/2024”
PMR 4683-2“Conduct an embryo-fetal development toxicity and toxicokinetic study with levacetylleucine in the non-rodent.”“12/2024”
PMR 4683-3“Conduct a juvenile toxicity and toxicokinetic study with levacetylleucine.”“05/2025”
PMR 4683-4“Conduct a fertility and early embryonic development (FEED) toxicity study with levacetylleucine.”“12/2024”
PMR 4683-5“Conduct a pre- and postnatal development (PPND) toxicity study with levacetylleucine.”“01/2025”
PMR 4683-6“Conduct a 26-week carcinogenicity study with levacetylleucine, administered by daily oral gavage, in an appropriate transgenic mouse model.”“03/2027”
PMR 4683-7“Conduct a long-term (2-year) GLP-complaint carcinogenicity study by oral gavage administration in rats.”“06/2029”
Postmarketing requirements from the 2024 approval: clinical drug-drug interaction trial

“Finally, we have determined that only a clinical trial (rather than a nonclinical or observational study) will be sufficient to identify an unexpected serious risk of drug-drug interactions with P-gp substrates.” (opens the source at this quote in a new tab)

RequirementTrialTrial completionFinal report submission
PMR 4683-8“Conduct a clinical drug-drug interaction (DDI) trial in healthy volunteers to evaluate the effect of levacetylleucine on a probe substrate of P-gp to thoroughly assess its inhibitory potential and its impact on patient safety.”“09/2026”“09/2027”
Postmarketing commitment from the 2024 approval: food effect trial
CommitmentTrialTrial completionFinal report submission
PMC 4683-9“Conduct a dedicated food effect trial to evaluate the impact of food on exposure of levacetylleucine granules for oral suspension”“09/2025”“03/2026”
Status of the required studies as reflected in the September 2026 prescribing information

“Animal studies to evaluate the carcinogenic potential of levacetylleucine have not been conducted.” (opens the prescribing information at this quote)

“Animal studies to evaluate effects of levacetylleucine on fertility have not been conducted.” (opens the prescribing information at this quote)

“Levacetylleucine inhibits P-gp [see Clinical Pharmacology (12.3)]. However, the clinical significance of this finding has not been fully characterized.” (opens the prescribing information at this quote)

Rare pediatric disease priority review voucher: sponsor obligations

“FDA may revoke the PRV if the rare pediatric disease product for which the PRV was awarded is not marketed in the U.S. within 1 year following the date of approval.” (opens the source at this quote in a new tab)

“The sponsor of an approved rare pediatric disease product application who is awarded a PRV must submit a report to FDA no later than 5 years after approval that addresses, for each of the first 4 post-approval years:” (opens the source at this quote in a new tab)

“the actual amount of product distributed in the U.S.” (opens the source at this quote in a new tab)

Other product development: phase 3 trial in CACNA1A-related disorders

“The Phase III study (IB1001-304) is a 12-week, randomized, double-blind, placebo-controlled crossover trial designed to assess the safety, tolerability, and efficacy of levacetylleucine in this population.” (opens the source at this quote in a new tab)

“Levacetylleucine is investigational for the treatment of CACNA1A-related disorders and has not been approved by the FDA, MHRA, EMA, Swissmedic, or any other regulatory authority for this indication.” (opens the source at this quote in a new tab)

“This is a multinational, randomized, placebo-controlled, double-blinded, cross-over Phase III study that will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) versus Placebo for the treatment of CACNA1A Disorders.” (opens the source at this quote in a new tab)