Aqneursa (levacetylleucine)
Metabolic Modifier - Neimann Pick Disease Type C (NPC) Oral Granules In Packet (Ea)
Last reviewed · Source: First Databank (FDB) drug data
Overview
Levacetylleucine is a modified amino acid.
Levacetylleucine is a modified amino acid that is slightly soluble in aqueous solution. Animal studies suggest that levacetylleucine has the potential to reduce stored intracellular lipids; however, the distinct molecular target for levacetylleucine and mechanism of action in the treatment of Niemann-Pick type C disease (NPC) are unknown.
The time course of pharmacodynamic response of levacetylleucine is unknown. Peak plasma concentrations of levacetylleucine are achieved in about 1 hour (range 0.5-2.5 hours). The estimated half-life is approximately 1 hour.
Levacetylleucine is metabolized into acetate and L-leucine by ubiquitously expressed enzymes; levacetylleucine is not metabolized by cytochrome P-450 (CYP) enzymes.
Generic name: levacetylleucine
Drug class: Metabolic Modifier - Neimann Pick Disease Type C (NPC)
Therapeutic class: Metabolic Modifiers
Reference product & biosimilars
No reference, biosimilar or interchangeable associated with this drug.
Today’s intelligence on Aqneursa
Indications
The following indications are listed for Aqneursa:
- Ataxia-Telangiectasia
- Niemann-Pick Disease Type C
Also known as: Louis-Bar Syndrome, Niemann-Pick Type C.
Dosing
No dosing information is available for this tab.
Levacetylleucine is administered orally as a suspension 2 to 3 days a day, with or without food.
The drug is commercially available in individual unit-dose packets containing granules that must be reconstituted prior to administration. One packet contains 1 g levacetylleucine.
The suspension may be administered by mouth or via a gastrostomy tube (G-tube).
| Drug label | Dosing type | Instructions |
|---|---|---|
| Aqneursa 1 Gram Granule Packet | Maintenance | Take by oral route 3 times per day as 2 packets in the morning, 1 packet in the afternoon, and 1 packet in the evening. |
No dosing information is available for this tab.
Drug interactions
Cytochrome P-450 (CYP) isoenzymes are not involved in the metabolism of levacetylleucine.
Levacetylleucine does not inhibit CYP1A2, 2B6, 2C8, 2C9, 2C19, 2D6, or 3A4 and does not induce CYP 1A2, 2B6, or 3A4.
Levacetylleucine is a substrate of organic anion transporter (OAT)1 and OAT3; levacetylleucine is not a substrate of organic cation transporter (OCT)2, breast cancer resistance protein (BCRP), or P-glycoprotein (P-gp).
Levacetylleucine inhibits P-gp; however, the clinical importance of this is not known. The drug also inhibits BCRP, bile salt export pump (BSEP), OAT1 and OAT3, but it does not inhibit organic anion transporting polypeptide (OATP)1B1 and OATP1B3.
There are no contraindication interactions for this drug.
Severe Interaction: Action is required to reduce the risk of severe adverse interaction.
Levacetylleucine/Acetylleucine
SEVERITY LEVEL: 2-Severe Interaction: Action is required to reduce the risk of severe adverse interaction.
MECHANISM OF ACTION: The D-enantiomer (N-acetyl-D-leucine) portion of racemic acetylleucine (or N-acetyl-DL-leucine) may compete with levacetylleucine for monocarboxylate transporter uptake in the intestines.(1,2)
CLINICAL EFFECTS: Concurrent administration of N-acetyl-DL-leucine or N-acetyl-D-leucine may result in decreased efficacy of levacetylleucine.(1)
PREDISPOSING FACTORS: None determined.
PATIENT MANAGEMENT: The manufacturer of levacetylleucine recommends that the concomitant use of N-acetyl-DL-leucine or N-acetyl-D-leucine be avoided.(1)
DISCUSSION: In an animal model, administration of N-acetyl-DL-leucine resulted in greater maximum concentration (Cmax) and area-under-curve (AUC) of the D-enantiomer than the L-enantiomer, while elimination was similar for both enantiomers. Therefore, the D-enantiomer was thought to inhibit intestinal uptake of the L-enantiomer.(2)
There are no moderate interactions for this drug.
Contraindications
*None.
There are no contraindication contraindications for this drug.
There are no severe contraindications for this drug.
- No disease contraindications
Adverse reactions
The most common adverse reactions (>=5%) reported with levacetylleucine in clinical studies were abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
More Frequent
- Thrombocytopenic disorder
More Frequent
- Acute abdominal pain
- Dysphagia
- Upper respiratory infection
- Vomiting
Less Frequent
- Accidental fall
- Lacerations
- Urinary tract infection
Rare or Very Rare
- Acne rosacea
Precautions
No enhanced messaging available.
The safety and effectiveness of levacetylleucine have been established in pediatric patients weighing >=15 kg.
There are no adequate data regarding use of levacetylleucine in pregnant women. Based on findings from animal reproductive studies, levacetylleucine may cause embryo-fetal harm when administered to pregnant females. (See Fetal/Neonatal Morbidity and Mortality under Cautions)
The decision to continue or discontinue levacetylleucine during pregnancy should consider the patient's need for the drug, the potential drug-related risk to the fetus, and the potential adverse outcomes from untreated disease.
It is not known whether levacetylleucine is distributed into human milk, or if the drug has any effects on the breastfed infant or on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for levacetylleucine and any potential adverse effects on the breastfed infant from the drug or underlying maternal condition.
Niemann-Pick type C disease (NPC) is largely a disease of pediatric and young adult patients. Safety and efficacy of levacetylleucine have not been established in geriatric patients 65 years of age and older.
ICD codes
| Code | Type | Description |
|---|---|---|
| Ataxia-telangiectasia | ||
| G11.3 | ICD10CM | Cerebellar ataxia with defective DNA repair |
| Niemann-pick disease type C | ||
| E75.242 | ICD10CM | Niemann-pick disease type C |
ICD code information from First Databank (FDB).
Formulary status
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Last reviewed · Source: First Databank (FDB) drug data
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