Drug Trials Snapshots: AQNEURSA
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.
Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the AQNEURSA Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).
Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).
AQNEURSA (levacetylleucine)
ak nur' sah
IntraBio Ltd
Original Approval date: September 24, 2024
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
AQNEURSA is a prescription medicine that is used to treat the neurological manifestations of Niemann-Pick disease, Type C (NPC) in adults and children who weigh 15 kg or more.
How is this drug used?
AQNEURSA is an oral suspension that is taken by mouth or given through a surgically placed feeding tube called a gastrostomy (G) tube, twice daily.
Who participated in the clinical trials?
The FDA approved AQNEURSA based on evidence from a clinical trial of 60 patients with NPC. The trial was conducted at 11 of sites in seven countries in Europe and Australia.
How were the trials designed?
AQNEURSA was evaluated in one clinical trial of 60 patients with NPC. Study 1 was a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study. Patients 4 years of age or older were randomized 1:1 to receive AQNERUSA at the recommended dose or placebo for 12 weeks, followed by immediate crossover to other treatment for 12 weeks. The primary efficacy endpoint was the total score on the functional Scale for Assessment and Rating of Ataxia (fSARA) at 12 weeks.
The primary efficacy outcome was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). The fSARA consists of the gait, sitting, stance and speech disturbance domains of the original SARA with modifications to the scoring responses.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many males and females were enrolled in the clinical trial used to evaluate the efficacy of AQNEURSA.
Figure 1. Baseline Demographics by Sex
Source: Adapted from FDA Review
Figure 2 summarizes how many participants by race were enrolled in the clinical trial used to evaluate the efficacy of AQNEURSA.
Figure 2. Baseline Demographics by Race
Source: Adapted from FDA Review
Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of AQNEURSA.
Figure 3. Baseline Demographics by Age
Source: Adapted from FDA Review
Figure 4 summarizes how many participants by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of AQNEURSA.
Figure 4. Baseline Demographics by Ethnicity
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Baseline Demographics
|
Demographic |
AQNEURSA |
Placebo |
Total |
|---|---|---|---|
|
Sex, n (%) |
|||
|
Female |
14 (46.7) |
13 (43.3) |
27 (45.0) |
|
Male |
16 (53.3) |
17 (56.7) |
33 (55.0) |
|
Age, years |
|||
|
Mean (SD) |
24.2 (16.6) |
28.6 (15.2) |
26.4 (15.9) |
|
Median (min, max) |
18.5 (7, 63) |
26.5 (5, 67) |
25.0 (5.0, 67.0) |
|
Age group, years, n (%) |
|||
|
Pediatric (<18) |
15 (50.0) |
22 (73.3) |
23 (38.3) |
|
Adult (≥18) |
15 (50.0) |
8 (26.7) |
37 (61.7) |
|
Race, n (%) |
|||
|
White |
26 (86.7) |
28 (93.3) |
54 (90.0) |
|
Asian |
2 (6.7) |
0 |
2 (3.3) |
|
Other |
2 (6.7) |
2 (6.7) |
4 (6.7) |
|
Ethnicity, n (%) |
|||
|
Not Hispanic or Latino |
25 (83.3) |
26 (86.7) |
51 (85.0) |
|
Not reported |
1 (3.3) |
1 (3.3) |
2 (3.3) |
|
Unknown |
4 (13.3) |
3 (10.0) |
7 (11.7) |
|
Country of participation, n (%) |
|||
|
Australia |
0 |
6 (20.0) |
6 (10.0) |
|
Switzerland |
8 (26.7) |
4 (13.3) |
12 (20.0) |
|
Czech Republic |
3 (10.0) |
1 (3.3) |
4 (6.7) |
|
Germany |
8 (26.7) |
9 (30.0) |
17 (28.3) |
|
Great Britain |
8 (26.7) |
2 (6.7) |
10 (16.7) |
|
Netherlands |
3 (10.0) |
6 (20.0) |
9 (15.0) |
|
Slovakia |
0 |
2 (6.7) |
2 (3.3) |
Source: Adapted from FDA Review
Abbreviations: SD, standard deviation
What are the benefits of this drug?
In the clinical trial, clinicians rated patients’ neurological symptoms on the fSARA. There was less worsening of symptoms after 12 weeks in patients with NPC when they received AQNEURSA compared to when they received placebo.
What are the benefits of this drug (results of trials used to assess efficacy)?
Table 2. Summary of fSARA Efficacy Results
|
Parameter |
Estimated Mean fSARA Score (SE) |
|---|---|
|
AQNEURSA |
5.1 (0.1) |
|
Placebo |
5.6 (0.1) |
|
Treatment difference (95% CI) |
-0.4 (-0.7, -0.2) |
Source: Adapted from AQNEURSA Prescribing Information
The treatment difference in the adjusted means was calculated using a mixed model for repeated measures that included period, sequence, treatment, baseline value, and baseline-period interaction.
Abbreviations: CI, confidence interval; fSARA, functional Scale for the Assessment and Rating of Ataxia; SE, standard error
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: Because of the small sample size, a difference in how well the drug worked between male and females could not be determined.
- Race: The number of patients of races other than White was small; therefore, differences in how AQNEURSA worked among races could not be determined.
- Age: AQNEURSA was tested in patients with NPC between 5 to 67 years old. The effect of AQNEURSA was similar for all age groups.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
The treatment difference by age group and sex is displayed in Figure 5.
Figure 5. Forest Plot of Treatment Difference With 95% CI for fSARA Total Score by Subgroups
Source: Adapted from FDA Review
Subjects in Sequence 1 received AQNEURSA for the first 12 weeks, followed by immediate crossover to placebo for another 12 weeks.
Similarly, subjects in Sequence 2 received placebo for the first 12 weeks, followed by immediate crossover to AQNEURSA for another 12 weeks.
Abbreviations: CI, confidence interval; fSARA, functional Scale for the Assessment and Rating of Ataxia; N, number of subjects in treatment sequence
What are the possible side effects?
The most common side effects of AQNEURSA are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
What are the possible side effects (results of trials used to assess safety)?
Table 3 summarizes adverse reactions in patients with NPC who were treated with AQNEURSA or placebo for 12 weeks. Only the first cross-over period is included in this table.
Table 3. Adverse Reactions in ≥5% of Patients With NPC in Trial 1
|
Adverse Reaction |
AQNEURSA |
Placebo |
|---|---|---|
|
Upper respiratory tract infection |
5 (17) |
1 (3) |
|
Abdominal pain |
2 (7) |
0 (0) |
|
Dysphagia |
2 (7) |
0 (0) |
|
Vomiting |
2 (7) |
0 (0) |
Source: AQNEURSA Prescribing Information
Abbreviations: NPC, Niemann-Pick disease, Type C
Were there any differences in side effects of the clinical trials among sex, race, and age?
- Sex: In this small trial size, a difference in side effects between males and females could not be determined.
- Race: The number of patients in the clinical trial was small, and the majority of patients were White; therefore, differences in side effects to AQNEURSA among races could not be determined.
- Age: AQNEURSA was tested in patients with NPC between 5 and 67 years old and similar side effects occurred across age groups.
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.