AQNEURSA is a foundational therapy that is indicated for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing ≥15 kg.
The only FDA-approved stand-alone therapy for NPC
A chemically altered amino acid (protein building block)*
Well studied in a clinical trial of 60 people with NPC
FDA, US Food and Drug Administration.
*The exact way that AQNEURSA works to treat NPC is not known.
What are the AQNEURSA trial details?
AQNEURSA was well studied in a robust clinical trial.
The trial examined if AQNEURSA could affect the neurological symptoms and functional abilities of people with NPC. This was measured using a modified version of an assessment tool called the Scale for Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (or fSARA).
What is measured using the fSARA?
fSARA measures:
Your healthcare provider may perform the SARA, which includes all of the tests done for the fSARA plus tests for coordination using:
Each of these tests is scored. How the total score changes over time shows how symptoms are changing—if the number goes down, it means the symptoms are getting better overall; if the number goes up, it means the symptoms are getting worse.
In the clinical trial, treatment with AQNEURSA resulted in a reduction in the total fSARA score number compared with responses to placebo after 12 weeks,† indicating that there were improvements in many activities of daily living that support everyday functional independence, including walking, balance, speech, and fine motor skills.
Talk to your healthcare provider about whether AQNEURSA could be right for you or someone you care for.
Functional benefits were seen within 12 weeks
†People with NPC who received AQNEURSA first followed by placebo showed a greater improvement in the fSARA score with AQNEURSA. They had a mean change from baseline of -0.5 points with AQNEURSA, then 0 points with placebo. This was also seen in people who received placebo first followed by AQNEURSA. They had a mean change from baseline of -0.3 points with placebo, then -0.7 points with AQNEURSA.
These adverse reactions happened more often with AQNEURSA than in the 30 people who received a placebo—in that group, only 1 person had an upper respiratory infection.
These findings are based on early results that may change when the final analysis is complete.
The extension phase is examining if AQNEURSA can affect how the disease changes over time for people with NPC. This is measured using a tool called the 5-domain Niemann-Pick disease type C Clinical Severity Scale, also known as the NPC-CSS.
In the extension phase of the clinical trial, treatment with AQNEURSA resulted in potential disease-modifying effects, which means it may change the way the disease develops, as shown using the NPC-CSS. When compared with what would be expected for people with NPC, treatment with AQNEURSA potentially stopped the disease from getting worse over time.
| NPC-CSS at 1 year with AQNEURSA | NPC-CSS at 1 year with no treatment |
|---|---|
| -0.1 | 1.6 |
| This means the disease improved at 1 year with AQNEURSA | This means the disease got much worse at 1 year in patients who did not receive any treatment |
In addition, the functional benefits shown at 12 weeks were maintained at 1 year, as measured using the SARA.
| SARA at 12 weeks with AQNEURSA | SARA at 1 year with AQNEURSA |
|---|---|
| -2.0 | -1.9 |
| This means symptoms improved at 12 weeks with AQNEURSA | This means the symptom improvement at 1 year with AQNEURSA was similar to the symptom improvement at 12 weeks |
Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.
The most common side effects of AQNEURSA in people treated for Niemann-Pick disease type C include abdominal pain, dysphagia, upper respiratory tract infections and vomiting.
The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.
These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .
AQNEURSA® (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children weighing ≥15 kg.
AQNEURSA® (levacetylleucine) is indicated for the treatment of ataxia in adults and children with ataxia-telangiectasia (A-T) weighing ≥15 kg.
Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.
The most common side effects of AQNEURSA in people treated for Niemann-Pick disease type C include abdominal pain, dysphagia, upper respiratory tract infections and vomiting.
The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.
These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .
AQNEURSA® (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children weighing ≥15 kg.
AQNEURSA® (levacetylleucine) is indicated for the treatment of ataxia in adults and children with ataxia-telangiectasia (A-T) weighing ≥15 kg.
