| Drug Name |
AQNEURSA™ (levacetylleucine) |
| Active Ingredient |
levacetylleucine |
| Date of Approval |
September 24, 2024 |
| Manufacturer |
IntraBio Inc. |
| Approval Pathways and Indications |
Approval Pathway: NDA
Indication: AQNEURSA™ is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. |
| Therapeutic Class |
Niemann-Pick disease type C Agent |
| Formulation |
For oral suspension: 1 gram levacetylleucine in a unit-dose packet |
| MoA |
The distinct molecular target for levacetylleucine in the treatment of NPC is unknown |
| Dosing and Administration |
The recommended dosage of AQNEURSA™ is based on the patient's body weight and can be administered up to three times daily. For patients weighing 15 to less than 25 kg, the recommended dosage is 1 gram in the morning and evening. For those between 25 and 35 kg, the recommended dose is 1 gram in the morning, afternoon, and evening. Patients weighing 35 kg or more should take 2 grams in the morning and 1 gram each in the afternoon and evening. AQNEURSA™ is administered as an oral suspension prepared by mixing one packet contents with 40 mL of water, orange juice, or almond milk and taken within 30 minutes of mixing. If a dose is missed, patients should skip it and resume the next scheduled dose. |
| Administered by self or by HCP |
Administered by Self |
| Place in Therapy |
Niemann-Pick disease type C is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives.
AQNEURSA™ is the only FDA-approved stand-alone therapy for the treatment of NPC.
Most common adverse reactions are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. This medication can also cause fetal harm. It is recommended for females of reproductive potential to use effective contraception during treatment and for 7 days after the last dose if AQNEURSA™ is discontinued. |
| Expected Market Launch Date |
Aqneursa is available now |
| New Molecular Entity (NME) or Existing Formulation |
New Molecular Entity |
| Expected Cost |
Data Unavailable |
| Product Discontinuation |
N/A |
| Clinical Trials |
The FDA approval of AQNEURSA™ is based on the IB1001-301 clinical trial (NCT05163288), a multinational, randomized, double-blind, placebo-controlled, crossover study evaluating its effect on neurological symptoms in 60 pediatric (aged 4 years and older) and adult patients with confirmed NPC. The trial met its primary and all secondary efficacy endpoints. Within 12 weeks, AQNEURSA™ showed significant improvements in neurological symptoms and functional benefits important for daily life. The primary outcome, a modified version of the Scale for the Assessment and Rating of Ataxia (fSARA), focused on gait, sitting, stance, and speech. AQNEURSA™-treated patients had a greater improvement in fSARA scores compared to placebo, with a mean treatment difference of -0.4 (95% CI -0.7 to -0.2) and a p-value of <0.001. These results were supported by consistent findings on the original fSARA. |
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IntraBio. Aqneursa Prescribing Information. Available at: https://intrabio.com/wp-content/aqneursa-prescribing-information.pdf. Accessed September 26, 2024.
Drugs.com. FDA Approves Aqneursa (levacetylleucine) for Niemann-Pick Type C. Available at: https://www.drugs.com/newdrugs/fda-approves-aqneursa-levacetylleucine-niemann-pick-type-c-6371.html. Accessed September 26, 2024.
Drugs.com. Aqneursa (Levacetylleucine): FDA Approval History. Available at: https://www.drugs.com/history/aqneursa.html. Accessed September 26, 2024.
IntraBio. IntraBio Announces U.S. FDA Approval of Aqneursa for the Treatment of Niemann-Pick Disease Type C. Available at: https://intrabio.com/news/intrabio-announces-u-s-fda-approval-of-aqneursa-for-the-treatment-of-niemann-pick-disease-type-c/. Accessed September 26, 2024.
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