Drug Trials Snapshots: LEQSELVI
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 LEQSELVI Prescribing Information for all 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).
LEQSELVI (deuruxolitinib)
lek-sel-vee
Sun Pharmaceutical Industries, Inc.
Original Approval Date: July 25, 2024
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
LEQSELVI is a Janus kinase (JAK) inhibitor that is indicated for the treatment of adults with severe alopecia areata (AA).
How is this drug used?
LEQSELVI is an oral tablet that is taken twice daily.
Who participated in the clinical trials?
The FDA approved LEQSELVI based on evidence from two clinical trials of 1,209 patients with AA, who had at least 50% scalp hair loss as measured by the Severity of Alopecia Tool (SALT) for more than six months. The trials were conducted at 135 sites in five countries including the United States, Canada, France, Spain, and Poland.
How were the trials designed?
LEQSELVI was evaluated in two clinical trials of 1,209 patients with severe alopecia areata.
How were the trials designed?
The two primary trials were multicenter, randomized, double-blind, placebo-controlled phase 3 clinical trials (AA-1, NCT04518995 and AA-2, NCT04797650). They evaluated a total of 1,209 adult subjects with AA, who had at least 50% scalp hair loss as measured by SALT for more than six months. In both trials, subjects received LEQSELVI 8 mg twice daily, deuruxolitinib 12 mg twice daily, or placebo twice daily for 24 weeks. Deuruxolitinib 12 mg is not approved.
The trial population ranged from 18 to 65 years of age. Among the subjects enrolled, 64% were female; 74% were White, 9% were Black or African American, 6% were Asian; and 8% identified as Hispanic or Latino. At baseline, subjects had average current episode of hair loss of approximately four years, with 59% of subjects having complete or near complete scalp hair loss (defined as ≥95% scalp hair loss). The mean pooled baseline SALT scores across treatment groups ranged from 85.9 to 88.6 with a mean duration of current episode of hair loss of ranging 3.7 to 3.9 years. Approximately 73% of subjects had eyebrow hair involvement and 70% of subjects had eyelash hair involvement.
The primary endpoint for both trials assessed the proportion of subjects who achieved at least 80% scalp hair coverage (SALT score of ≤20) at Week 24. Key secondary outcomes included the percentage of responders (defined as “satisfied” or “very satisfied”) at Week 24 on the Satisfaction of Hair Patient-Reported Outcome (SPRO) and the percentage of subjects achieving an absolute SALT score of ≤20 at Week 20, 16, 12, and 8.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many male and female patients were enrolled in the combined clinical trials used to evaluate the efficacy of LEQSELVI.
Figure 1. Baseline Demographics by Sex, Efficacy Population
Source: Adapted from FDA Review
Figure 2 summarizes how many patients by race were enrolled in the combined clinical trials used to evaluate the efficacy of LEQSELVI.
Figure 2. Baseline Demographics by Race, Efficacy Population
Source: Adapted from FDA Review
Figure 3 summarizes how many patients by age were enrolled in the combined clinical trials used to evaluate the efficacy of LEQSELVI.
Figure 3. Baseline Demographics by Age, Efficacy Population
Source: Adapted from FDA Review
Figure 4 summarizes how many patients by ethnicity were enrolled in the combined clinical trials used to evaluate the efficacy of LEQSELVI.
Figure 4. Baseline Demographics by Ethnicity, Efficacy Population
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Demographics Table, Efficacy Population
| Demographic | LEQSELVI 8 mg BID N=600 n (%) | Deuruxolitinib 12 mg BID N=342 n (%) | Placebo N=267 n (%) | Pooled N=1209 n (%) |
|---|---|---|---|---|
| Sex | ||||
| Female | 385 (64.2) | 214 (62.6) | 174 (65.2) | 773 (63.9) |
| Male | 215 (35.8) | 128 (37.4) | 93 (34.8) | 436 (36.1) |
| Age group, years | ||||
| <40 | 339 (56.5) | 187 (54.7) | 135 (50.6) | 661 (54.7) |
| ≥40 | 261 (43.5) | 155 (45.3) | 132 (49.4) | 548 (45.3) |
| Race | ||||
| White | 443 (73.8) | 253 (74.0) | 198 (74.2) | 894 (73.9) |
| Black or African American | 52 (8.7) | 33 (9.6) | 23 (8.6) | 108 (8.9) |
| Asian | 26 (4.3) | 25 (7.3) | 17 (6.4) | 68 (5.6) |
| American Indian or Alaska Native | 5 (0.8) | 1 (0.3) | 1 (0.4) | 7 (0.6) |
| Native Hawaiian or other Pacific Islander | 3 (0.5) | 1 (0.3) | 1 (0.4) | 5 (0.4) |
| Other | 18 (3.0) | 6 (1.8) | 6 (2.2) | 30 (2.5) |
| Not reported | 53 (8.8) | 23 (6.7) | 21 (7.9) | 97 (8.0) |
| Ethnicity | ||||
| Unknown | 56 (9.3) | 23 (6.7) | 21 (7.9) | 100 (8.3) |
| Hispanic or Latino | 53 (8.8) | 22 (6.4) | 22 (8.2) | 97 (8.0) |
| Not Hispanic or Latino | 491 (81.8) | 297 (86.8) | 224 (83.9) | 1012 (83.7) |
Source: Adapted from FDA Review
Abbreviations: BID, twice dail
What are the benefits of this drug?
LEQSELVI was shown to improve hair growth in subjects with severe AA, with around 30% of subjects experiencing 80% or more scalp hair after 24 weeks of treatment, and around 23% of subjects experiencing 90% or more scalp hair after 24 weeks of treatment.
What are the benefits of this drug (results of trials used to assess efficacy)?
Assessment of scalp hair loss was based on the SALT score. At Week 24, a greater proportion of subjects had a SALT ≤20 response (80% or more scalp hair) and SALT ≤10 response (90% or more scalp hair) with LEQSELVI 8 mg twice daily compared to placebo.
Table 2. Clinical Response at Week 24 in Adult Subjects With Severe AA in Trials AA-1 and AA-2
| SALT T Score | Trial AA-1 | Trial AA-2 | ||
|---|---|---|---|---|
| Placebo N=140 | LEQSELVI 8 mg BID N=351 | Placebo N=127 | LEQSELVI 8 mg BID N=249 | |
| ≤20, % | 1 | 29 | 1 | 32 |
| Difference from placebo, % (95% CI) | 28 (23, 33) | 31 (25, 37) | ||
| ≤10, % | 0a | 20 | 0a | 24 |
| Difference from placebo, % (95% CI) | 21 (16, 25)a | 24 (19, 30)a | ||
Source: Adapted from LEQSELVI Prescribing Information
a Not adjusted for multiplicity
Abbreviations: AA, alopecia areata; BID, twice daily; CI, confidence interval; SALT, Severity of Alopecia Too
Table 3. Evaluation of Patient Satisfaction With Scalp Hair Coverage at Week 24 in Adult Subjects With Severe AA in Trials AA-1 and AA-2
| Satisfaction Rating | Trial AA-1 | Trial AA-2 | ||
|---|---|---|---|---|
| Placebo N=140 % | LEQSELVI 8 mg BID N=351 % | Placebo N=127 % | LEQSELVI 8 mg BID N=249 % | |
| Very satisfieda | 2 | 19 | 0 | 18 |
| Satisfieda | 3 | 23 | 2 | 28 |
| Neither satisfied nor dissatisfied | 16 | 19 | 13 | 15 |
| Dissatisfied | 25 | 18 | 18 | 16 |
| Very dissatisfied | 54 | 21 | 67 | 23 |
Source: Adapted from LEQSELVI Prescribing Information
a In Trial AA-1, the proportions of responders (defined as subject who were “satisfied” or “very satisfied”) on LEQSELVI was 42% compared to 5% on placebo. In Trial AA-2, the proportion of responders on LEQSELVI was 46% compared to 2% on placebo.
Abbreviations: AA, alopecia areata; BID, twice daily
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: LEQSELVI worked better in females compared to males.
- Race: LEQSELVI worked similarly in patients regardless of race.
- Age: LEQSELVI worked better in patients younger than 50 years of age compared to patients older than 50 years of age.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
Treatment effects for the primary endpoint were generally consistent across age, sex, race, and ethnicity subgroups. Within each dose group, treatment effects were larger in subjects with age <50 years old than in subjects with age ≥50 years, and treatment effects were larger in female subjects than in male subjects.
Table 4. Response at Week 24 by Demographic Subgroups With SALT Scores ≤20, Efficacy Population
| Demographic | Trial AA-1 | Trial AA-2 | ||||
|---|---|---|---|---|---|---|
| Placebo N=140 n/Nw (%)a | LEQSELVI 8 mg BID N=351 n/Nw (%)a | Deuruxolitinib 12 mg BID N=215 n/Nw (%)a | Placebo N=127 n/Nw (%)a | LEQSELVI 8 mg BID N=249 n/Nw (%)a | Deuruxolitinib 12 mg BID N=127 n/Nw (%)a | |
| Age, years | ||||||
| <50 | 0/91 (0.1) | 77/239 (31.7) | 68/150 (43.5) | 1/90 (0.1) | 60/181 (31.7) | 41/91 (43.5) |
| ≥50 | 1/37 (2.6) | 17/79 (21.6) | 15/50 (28.7) | 0/29 (2.6) | 17/52 (21.6) | 5/29 (28.7) |
| Sex | ||||||
| Female | 0/81 (0.2) | 67/196 (33.2) | 55/122 (42.9) | 1/80 (0.2) | 63/161 (33.2) | 34/77 (42.9) |
| Male | 1/47 (2) | 27/122 (22.7) | 28/78 (34.9) | 0/39 (2) | 14/72 (22.7) | 12/43 (34.9) |
| Race | ||||||
| White | 1/91 (1) | 73/221 (32.1) | 62/135 (43.2) | 1/93 (1) | 68/190 (32.1) | 42/103 (43.2) |
| Black or African American | 0/14 (0) | 3/30 (10) | 2/23 (8.7) | 0/6 (0) | 0/10 (0) | 1/4 (25) |
| Asian | 0/9 (0) | 8/22 (36.4) | 10/21 (47.6) | 0/7 (0) | 2/3 (66.7) | 1/4 (25) |
| American Indian or Alaska Native | 0 | 1/2 (50) | 1/1 (100) | 0/1 (0) | 0/2 (0) | 0 |
| Native Hawaiian or other Pacific Islander | 0/1 (0) | 0/3 (0) | 1/1 (100) | 0 | 0 | 0 |
| Other | 0/4 (0) | 6/15 (41.1) | 2/6 (33.3) | 0/1 (0) | 0/1 (0) | 0 |
| Ethnicity | ||||||
| Hispanic or Latino | 0/10 (0) | 9/26 (37.4) | 2/10 (19.1) | 1/9 (0) | 5/22 (37.4) | 4/9 (19.1) |
| Not Hispanic or Latino | 1/109 (1) | 81/264 (29.7) | 76/177 (41.1) | 0/99 (1) | 65/184 (29.7) | 40/102 (41.1) |
Source: Adapted from FDA Review
a Counts are based on non-missing data and percentages are based on the MI data
Abbreviations: BID, twice a day; MI, multiple imputation; N, number of patients in treatment arm; n, number of patients meeting criteria; Nw, number of patients with data; SALT, Severity of Alopecia Too
Table 5. Response at Week 24 by Demographic Subgroups With SALT Scores ≤20, Efficacy Population (Pooled)
| Demographic | Placebo N=267 n/Nw (%)a | LEQSELVI 8 mg BID N=600 n/Nw (%)a | Deuruxolitinib 12 mg BID N=342 n/Nw (%)a |
|---|---|---|---|
| Age, years | |||
| <50 | 1/181 (0.1) | 137/420 (31.7) | 109/241 (43.5) |
| ≥50 | 1/66 (2.6) | 34/131 (21.6) | 20/79 (28.7) |
| Sex | |||
| Female | 1/161 (0.2) | 130/357 (33.2) | 89/199 (42.9) |
| Male | 1/86 (2) | 41/194 (22.7) | 40/121 (34.9) |
| Race | |||
| White | 2/184 (1) | 141/411 (32.1) | 104/238 (43.2) |
| Black or African American | 0/20 (0.9) | 3/40 (14.2) | 3/27 (11.3) |
| Asian | 0/16 (0) | 10/25 (36.4) | 11/25 (47.6) |
| American Indian or Alaska Native | 0/1 (100) | 1/4 (50) | 1/1 (100) |
| Native Hawaiian or other Pacific Islander | 0/1 (0) | 0/3 (0) | 1/1 (100) |
| Other | 0/5 (0) | 6/16 (41.1) | 2/6 (33.3) |
| Ethnicity | |||
| Hispanic or Latino | 1/19 (0) | 14/48 (37.4) | 6/19 (19.1) |
| Not Hispanic or Latino | 1/208 (1) | 146/448 (29.7) | 116/279 (41.1) |
Source: Adapted from FDA Review
a Counts are based on non-missing data and percentages are based on the MI data.
Abbreviations: BID, twice a day; MI, multiple imputation; N, number of patients in treatment arm; n, number of patients meeting criteria; Nw, number of patients with data; SALT, Severity of Alopecia Tool
What are the possible side effects?
LEQSELVI may cause serious side effects, including:
- Serious infections
- Increased risk of death in people 50 years of age and older who have at least one heart disease risk factor and are taking a JAK inhibitors
- Cancer and immune system problems
- Increased risk of major cardiovascular events such as heart attack, stroke, or death
- Blood clots in the veins of the legs (deep vein thrombosis), lungs (pulmonary embolism), or brain (cerebral venous sinus thrombosis)
- Tears (perforation) in the stomach or intestines
- Changes in certain laboratory test results:
- increased fat (lipid) levels in blood (triglycerides and cholesterol)
- low red blood cell counts
- low lymphocyte or neutrophil counts
What are the possible side effects (results of trials used to assess safety)?
Among 1,319 subjects enrolled in the placebo-controlled clinical trials, 640 subjects received LEQSELVI 8 mg twice daily, 380 subjects received deuruxolitinib 12 mg twice daily, and 299 subjects received placebo twice daily for up to 24 weeks. Adverse Reactions occurring at ≥1% in the LEQSELVI 8 mg or deuruxolitinib 12 mg twice daily group and at a higher rate than in the placebo group are presented below. A total of 20 (3.1%) of subjects treated with LEQSELVI 8 mg were discontinued from the trials due to adverse reactions.
Table 6. Adverse Events, Safety Population
| Adverse Reaction | Placebo N=299 n (%)a | LEQSELVI 8 mg BID N=640 n (%)a | Deuruxolitinib 12 mg BID N=380 n (%)a |
|---|---|---|---|
| Acneb | 13 (4.3) | 66 (10.0) | 52 (12.6) |
| Headache | 30 (9.4) | 83 (12.4) | 44 (10.5) |
| Nasopharyngitis | 21 (6.7) | 54 (8.1) | 33 (7.7) |
| Blood creatine phosphokinase increased | 7 (2.2) | 35 (5.3) | 27 (7.4) |
| Hyperlipidemiac | 10 (3.1) | 30 (4.4) | 19 (5.2) |
| Fatigued | 12 (3.9) | 26 (3.9) | 20 (4.9) |
| Skin and soft tissue infectionse | 2 (0.8) | 11 (1.6) | 15 (4.0) |
| Anemiaf | 3 (1.0) | 18 (2.6) | 16 (3.4) |
| Weight increased | 4 (1.4) | 19 (2.9) | 10 (2.5) |
| Neutropeniag | 3 (0.7) | 10 (1.4) | 10 (2.8) |
| Lymphopenia | 2 (0.6) | 2 (0.3) | 7 (2.0) |
| Thrombocytosis | 0 | 18 (2.7) | 6 (1.6) |
| Herpesi | 2 (0.6) | 8 (1.2) | 6 (1.6) |
Source: LEQSELVI Prescribing Information
a Study size adjusted percentages
b Acne includes: acne, dermatitis acneiform, and acne pustular
c Hyperlipidemia includes: blood cholesterol increased, low density lipoprotein increased, blood triglycerides increased, hypercholesterolemia, hyperlipidemia, hypertriglyceridemia, and dyslipidaemia
d Fatigue includes: fatigue, asthenia, hypersomnia, somnolence, and lethargy
e Skin and soft tissues infections includes: folliculitis, impetigo, skin infection, subcutaneous abscess, furuncle, paronychia, and pustule
f Anemia includes: anemia, hematocrit decreased, hemoglobin decreased, iron deficiency anemia, and red blood cell count decreased
g Neutropenia includes: neutropenia and neutrophil count increased
h Thrombocytosis includes: thrombocytosis and platelet count increased
i Herpes includes: oral herpes, herpes simplex, genital herpes simplex, and nasal herpes
Were there any differences in side effects among sex, race, and age?
- Sex: The occurrence of side effects was similar in males and females.
- Race: The occurrence of side effects was similar amongst all races.
- Age: The occurrence of side effects was similar in patients younger than 50 years of age and patients older than 50 years of age.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Table 7. Overview of Adverse Reactions by Subgroup, Safety Population
| Subgroup | All Grades | Grades 3 to 4 | ||||
|---|---|---|---|---|---|---|
| Placebo N=299 n/Ns (%) | LEQSELVI 8 mg BID N=640 n/Ns (%) | Deuruxolitinib 12 mg BID N=380 n/Ns (%) | Placebo N=299 n/Ns (%) | LEQSELVI 8 mg BID N=640 n/Ns (%) | Deuruxolitinib 12 mg BID N=380 n/Ns (%) | |
| Sex | ||||||
| Female | 169/199 (84.9) | 368/414 (88.9) | 213/242 (88.0) | 7/199 (3.5) | 20/414 (4.8) | 14/242 (5.8) |
| Male | 85/100 (85.0) | 187/226 (82.7) | 105/138 (76.1) | 6/100 (6.0) | 10/226 (4.4) | 9/138 (6.5) |
| Age group, years | ||||||
| 18 to <50 | 191/221 (86.4) | 421/487 (86.4) | 241/287 (84.0) | 11/221 (5.0) | 22/487 (4.5) | 16/287 (5.6) |
| ≥50 | 63/78 (80.8) | 134/153 (87.6) | 77/93 (82.8) | 2/78 (2.6) | 8/153 (5.2) | 7/93 (7.5) |
| Race | ||||||
| American Indian or Alaska Native | 1/1 (100) | 3/6 (50.0) | 1/1 (100) | 0/1 (0) | 0/6 (0) | 0/1 (0) |
| Asian | 13/18 (72.2) | 23/27 (85.2) | 24/29 (82.8) | 1/18 (5.6) | 2/27 (7.4) | 1/29 (3.4) |
| Black or African American | 23/31 (74.2) | 50/63 (79.4) | 20/36 (55.6) | 2/31 (6.5) | 4/63 (6.3) | 2/36 (5.6) |
| Native Hawaiian or other Pacific Islander | 1/2 (50.0) | 3/3 (100) | 1/1 (100) | 0/2 (0) | 0/3 (0) | 0/1 (0) |
| Not applicable | 21/21 (100) | 52/53 (98.1) | 23/23 (100) | 1/21 (4.8) | 2/53 (3.8) | 2/23 (8.7) |
| Other | 4/7 (57.1) | 19/21 (90.5) | 6/6 (100) | 2/7 (28.6) | 0/21 (0) | 1/6 (16.7) |
| White | 191/219 (87.2) | 405/467 (86.7) | 243/284 (85.6) | 7/219 (3.2) | 22/467 (4.7) | 17/284 (6.0) |
| Ethnicity | ||||||
| Hispanic or Latino | 16/24 (66.7) | 45/57 (78.9) | 18/25 (72.0) | 0/24 (0) | 0/57 (0) | 2/25 (8.0) |
| Not Hispanic or Latino | 217/254 (85.4) | 453/525 (86.3) | 277/332 (83.4) | 12/254 (4.7) | 28/525 (5.3) | 19/332 (5.7) |
| Unknown | 0/0 (NA) | 5/5 (100) | 0/0 (NA) | 0/0 (NA) | 0/5 (0) | 0/0 (NA) |
| Missing | 21/21 (100) | 52/53 (98.1) | 23/23 (100) | 1/21 (4.8) | 2/53 (3.8) | 2/23 (8.7) |
| Country of participation | ||||||
| United States | 254/299 (84.9) | 555/640 (86.7) | 318/380 (83.7) | 13/299 (4.3) | 30/640 (4.7) | 23/380 (6.1) |
Source: Adapted from FDA Review
Safety population includes patients from studies 2001, 3001, and 3002 who received placebo or study drug twice daily for up to 24 weeks.
Abbreviation: BID, twice daily; N, number of patients in the safety population; n, number of patients with given characteristic; NA, not applicable; Ns, total number of patients in each categor
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 used in clinical trials 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.