Drugs

Drug Trial Snapshot: LIBTAYO

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race, and age 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 health provider about the risks and benefits of a drug. Refer to the LIBTAYO Prescribing Information for complete information.

LIBTAYO (cemiplimab-rwlc)
Lib-TIE-oh
Regeneron
Approval date:September 28, 2018


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

LIBTAYO is a drug for the treatment of one type of skin cancer called cutaneous squamous cell carcinoma(CSCC). It is approved to treat patients whose cancer has spread through the body (metastatic) or is locally advanced and who cannot have radiation or surgery to remove all the cancer.

How is this drug used?

LIBTAYO is an injection. It is given by a healthcare professional directly into the vein (an intravenous infusion) over 30 minutes every 3 weeks.

What are the benefits of this drug?

LIBTAYO treatment led to tumor shrinkage or tumor disappearance in 47% of treated patients.

What are the benefits of this drug (results of trials used to assess efficacy)?

The efficacy results for both trials based on confirmed objective response rate (ORR), as assessed by independent central review (ICR), and ICR-assessed duration of response are presented below.

Table 2. Efficacy Results for Trial 1 and Trial 2

Efficacy Endpoints*

Metastatic CSCC
N = 75

Locally Advanced CSCC
N = 33

Combined CSCC
N = 108

Confirmed Objective Response Rate

Objective response rate (95% CI)

46.7%
(35.1%, 58.6%)

48.5%
(30.8%, 66.5%)

47.2%
(37.5%, 57.1%)

Complete response (CR) rate†

5.3%

0%

3.7%

Partial response (PR) rate

41.3%

48.5%

43.5%

Duration of Response

Range in months

2.8 – 15.2+

1 – 12.9+

1 – 15.2+

Patients with DOR ≥ 6 months, n %

21 (60%)

10 (63%)

31 (61%)

CI: confidence interval; +: Denotes ongoing at last assessment

* Median duration of follow up: metastatic CSCC: 8.1 months; locally advanced CSCC: 10.2 months; combined CSCC: 8.9 months

† Only includes patients with complete healing of prior cutaneous involvement; locally advanced CSCC patients in Trial 2 required biopsy to confirm complete response.<

LIBTAYO Prescribing Information

 

Were there any differences in how well the drug worked in clinical trials among sex, race and age?

  • Sex: LIBTAYO works similarly in men and women.
  • Race: Most patients in the trials were White. Differences in how well LIBTAYO works among races could not be determined.
  • Age: LIBTAYO works similarly in patients younger and older than 65 years of age.

Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?

Exploratory subgroup efficacy analysis for sex, age, and geographic region is presented below. Race analysis was not conducted because of predominatly White population in the trials.

Table 3. Subgroup Analysis of Objective Response Rate in Combined CSCC Trials

Demographic Characteristic

#
Responses/N

ORR1 (95% CI)

Sex

Men

46/92

50.0 (39.4 , 60.6)

Women

5/16

31.2 (11.0 , 58.7)

 

Age

<65 years

16/32

50.0 (31.9 , 68.1)

≥65 years

35/76

46.1 (34.5 , 57.9)

 

Region

U.S.

30/69

43.5 (31.6 , 56.0)

Rest of World

21/39

53.8 (37.2 , 69.9)

ORR, objective response rate; CI, confidence interval;

Adapted from FDA Review

What are the possible side effects?

LIBTAYO can cause serious and potentially deadly immune reactions including inflammation of the lungs, gut, liver, kidneys, hormonal glands and skin as well as infusion related reactions.

The most common adverse reactions are fatigue, rash and diarrhea.

What are the possible side effects (results of trials used to assess safety)?

The table below summarizes side effects that occurred in patients from both trials combined.

Table 4. Adverse Drug Reactions in ≥10% of Patients with Advanced CSCC Receiving LIBTAYO in Trial 1 and Trial 2

Adverse Reactions

LIBTAYO N=163

All Grades
%

Grade 3-4
%

Skin and Subcutaneous Tissue

Rash*

25

1.2

Pruritus†

15

0

Gastrointestinal

Diarrhea‡

22

0.6

Nausea

19

0

Constipation

12

0.6

General and Administration Site

Fatigue§

29

2

Musculoskeletal and Connective Tissue

Musculoskeletal pain#

17

3

Metabolism and Nutrition

Decreased appetite

10

0

* Rash is a composite term that includes rash maculopapular, rash, dermatitis, rash generalized, dermatitis bullous, drug eruption, erythema, rash erythematous, rash macular, rash pruritic, and skin reaction.

† Pruritus is a composite term that includes pruritus and pruritus allergic.

‡ Diarrhea is a composite term that includes diarrhea and colitis.

§ Fatigue is a composite term that includes fatigue and asthenia.

#Musculoskeletal pain is a composite term that includes: musculoskeletal pain, back pain, myalgia, neck pain, pain in extremity.

LIBTAYO Prescribing Information

 

Were there any differences in side effects among sex, race and age?

  • Sex: Occurrence of side effects was similar in men and women.
  • Race: Most of the patients in the trials were White. Differences in side effects among races could not be determined.
  • Age: Occurrence of side effects was similar in patients younger and older than 65 years of age.

Were there any differences in side effects of the clinical trials among sex, race, and age groups?

Exploratory subgroup analysis for sex and age is presented below. Race analysis was not conducted because of predominatly White population in the trials.

Table 5. Adverse Events by Sex

 

Sex Subgroups

 

Any grade event n (%)

 

Grade ≥3  events n (%)

 

SAEs n (%)

Men (N=138)

131 (95)

53 (38)

38 (28)

Women (N=25)

24 (96)

9 (36)

8 (32)

Table 6. Adverse Events by Age Group

 

Age Subgroups

 

Any grade event n (%)

 

Grade ≥3  events n (%)

 

SAEs n (%)

< 65 years (N=45)

43 (96)

19 (22)

4 (9)

≥ 65 - < 75 years (N=58)

54 (93)

21 (36)

17 (29)

≥ 75 years (N=60)

58 (96)

31 (52)

25 (42)

SAEs=serious adverse events

Adapted from FDA Review and Clinical Trial Data

WHO WAS IN THE STUDIES?

Who participated in the clinical trials?

The FDA approved LIBTAYO based on evidence from two clinical trials (Trial 1/ NCT02383212, and Trial 2/NCT02760498) that enrolled 163 patients with advanced cutaneous squamous cell carcinoma(CSCC). The trials were conducted in United States, Australia and Europe.

Demographics of patients who were assessed for the benefit of LIBTAYO (efficacy population) is presented in Table 8 under MORE INFO.

The figure below summarizes how many men and women in both trials combined received at least one dose of LIBTAYO and provided data for evaluation of side effects (safety population).

Figure 1. Baseline Demographics by Sex (safety population)

Pie chart summarizing how many men and women were in the clinical trials. In total, 138 men (85%) and 25 women (15%) participated in the clinical trials.

Clinical Trial Data

The figure and table below summarize the percentage of patients by race in safety population.

Figure 2. Baseline Demographics by Race (safety population)

Pie chart summarizing the percentage of patients by race enrolled in the trials. In total, 157 Whites (96%), and 6  patients of other races Black (4%), participated in the clinical trials.

Clinical Trial Data

Table 1. Baseline Demographics by Race (safety population)

 

Race

 

 Number of Patients

 

 Percentage of Patients

  White

157

96

  Black or African American

1

1

  Asian

2

1

  Unknown/not reported

3

2

Clinical Trial Data

The figure below summarizes the percentage of patients by age in safety population.

Figure 3. Baseline Demographics by Age (safety population)

Pie charts summarizing how many individuals of certain age groups were in the clinical trial. In total, 45 were below 65 years old (28%) and  118 participants were 65 and older (72%).

Clinical Trial Data

Who participated in the trials?

The table below summarizes baseline demographics for trials population.

 

Table 7. Baseline Demographics (safety population)

 

Demographic Parameters

Metastatic CSCC
(N = 98)

Locally Advanced CSCC
(N = 65)

Combined CSCC
(N = 163)

Sex, n (%)

Men

87 (88.8)

51 (78.5)

138 (84.7)

Women

11 (11.2)

14 (21.5)

25 (15.3)

Race, n (%)

White

96 (98.0)

61 (93.8)

157 (96.3)

Black or African American

1 (1.0)

0

1 (0.6)

Asian

1 (1.0)

1 (1.5)

2 (1.2)

Unknown

0

2 (3.1)

2 (1.2)

Not reported

0

1 (1.5)

1 (0.6)

Age

Mean years (SD)

69.9 (10.83)

71.6 (11.75)

70.6 (11.2)

Median (years)

70

72

71

Min, max (years)

38,93

45,96

38,96

Age Group, n(%)

< 65 years

27 (27.6)

18 (27.7)

45 (27.6)

≥ 65 years

71 (72.4)

47 (72.3)

118 (72.4)

Ethnicity, n (%)

Hispanic or Latino

2 (2.0)

2 (3.1)

4 (2.5)

Not Hispanic or Latino

95 (96.9)

61 (93.8)

156 (95.7)

Unknown

1 (1.0)

2 (3.1)

3 (1.8)

Region, n (%)

United States

49 (50)

52 (80)

101 (61.9)

Europe

12 (12.2)

10 (15.4)

22 (13.5)

 Australia

37 (37.8)

3 (4.6)

40 (24.5)

Clinical Trial Data

Table 8. Baseline Demographics (efficacy population)

Demographic Parameters

Metastatic CSCC
(N = 75)

Locally Advanced CSCC
(N = 33)

Combined CSCC
(N = 108)

Sex, n (%)

Men

66 (88.0)

26 (78.8)

92 (85.2)

Women

9 (12.0)

7 (21.2)

16 (14.8)

Race, n (%)

White

74 (98.7)

31 (93.9)

105 (97.2)

Black or African American

1 (1.3)

0 (0)

1 (0.9)

Unknown

0 (0)

2 (6.1)

2 (1.9)

Age

Mean years (SD)

71 (11)

70 (13)

71 (12)

Median (years)

72

70

71

Min, max (years)

38, 93

47, 96

38, 96

Age Group, n(%)

< 65 years

20 (26.7)

12 (36.4)

32 (29.6)

≥ 65 years

55 (73.3)

21 (63.6)

76 (70.4)

Ethnicity, n (%)

Hispanic or Latino

2 (2.7)

1 (3.0)

3 (2.8)

Not Hispanic or Latino

72 (96.0)

31 (93.9)

103 (95.4)

Unknown

1 (1.3)

1 (3.0)

2 (1.9)

Region, n (%)

United States

41 (54.7)

28 (84.8)

69 (63.9)

Europe

10 (13.3)

5 (15.2)

15 (13.9)

Australia

24 (32.0)

0 (0)

24 (22.2)

FDA Review

How were the trials designed?

The FDA approved LIBTAYO based on evidence from two clinical trials that enrolled patients with advanced cutaneous squamous cell carcinoma(CSCC). Both trials included patients with metatatic cancer and with locally advanced cancer.

All patients received LIBTAYO intravenously every 2 weeks for up to 48 weeks in Trial 1 or up to 96 weeks in Trial 2. Treatment continued until progression of disease, unacceptable toxicity, or completion of planned treatment.

The trial measured the proportion of patients who experienced partial shrinkage or complete disappearance of their tumors while receiving LIBTAYO.

How were the trials designed?

The efficacy and safety of LIBTAYO in patients with metastatic (nodal or distant) cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who were not candidates for curative surgery or curative radiation were evaluated in two open-label, multi-center, non-randomized, multicohort trials.

The primary efficacy outcome measure of the trial was objective response rate (ORR). ORR was determined by blinded central review according to a protocol-specific modified Response Evaluation Criteria in Solid Tumors (mRECIST), using an integrated composite response based on radiographic (MRI), photographic (digital clinical photography), and histological data.

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.

PRESCRIBING INFORMATION

 

Back to Drug Trials Snapshots

 

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