U.S. flag An official website of the United States government
  1. Home
  2. Drugs
  3. Development & Approval Process | Drugs
  4. Drug Approvals and Databases
  5. Drug Trials Snapshots: ALECENSA
  1. Drug Approvals and Databases

Drug Trials Snapshots: ALECENSA

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 ALECENSA Prescribing Information for complete information.

ALECENSA (alectinib)
a-le-sen'-sah
Genentech
Approval date: December 11, 2015


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

ALECENSA is a drug used to treat a type of lung cancer called non-small cell lung cancer (NSCLC) that is advanced (metastatic). It is to be used in patients who have a specific gene rearrangement in what is called the anaplastic lymphoma kinase (ALK) gene. It should be used only in patients whose cancer has worsened after, or who could not tolerate treatment with, another drug called Xalkori (crizotinib).

How is this drug used?

ALECENSA is a capsule that is taken two times a day with food.

What are the benefits of this drug?

In one trial, 38% of patients taking ALECENSA had a partial shrinkage of their cancer, which lasted an average of 7.5 months. In a second trial, 44% of patients had a partial shrinkage of their tumor, which lasted an average of 11.2 months.

For the two trials combined, 61% of patients who had measurable cancer lesions in the brain prior to taking ALECENSA had a complete or partial reduction of the lesions in the brain which lasted an average of 9.1 months.

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

The tables below summarize efficacy results for the clinical trials.

Table 2. Efficacy Results in Studies 1 and 2

Efficacy ParameterStudy 1 (N=87)Study 2 (N=138)
IRC*
Assessment**
Investigator AssessmentIRC*
Assessment**
Investigator Assessment
Objective Response Rate (95% CI)38%
(28; 49)
46%
(35; 57)
44%
(36; 53)
48%
(39; 57)
Number of Responders33406166
Duration of Response, median in months (95% CI)7.5
(4.9, Not Estimable)
NE
(4.9, Not Estimable)
11.2
(9.6, Not Estimable)
7.8
(7.4, 9.2)

*Independent Review Committee
**18 patients in Study 1 and 16 patients in Study 2 did not have measurable disease at baseline as per IRC assessment and were classified as non-responders in the IRC analysis.
ALECENSA Prescribing Information

Table 3. CNS Objective Response in Patients with Measurable CNS Lesions in Studies 1 and 2

Efficacy ParameterN=51
CNS Objective Response Rate (95% CI)61% (46, 74)
Complete Response18%
Partial Response43%
CNS Duration of Response, median in months (95% CI)9.1 (5.8, not evaluable)

CNS=Central Nervous System
ALECENSA Prescribing Information

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

  • Sex: ALECENSA worked similarly in men and women.
  • Race: The majority of patients in the clinical trial were white. Differences in response to ALECENSA among races could not be determined.
  • Age: The majority of patients in the clinical trial were younger than 65 years of age. Differences in response to ALECENSA between patients below and above 65 years of age could not be determined.

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

The table below summarizes efficacy results by subgroup.

Table 3. Subgroup Analysis of Objective Response Rate

 Study 1Study 2
 % (95% CI)% (95% CI)
ITT population37.9 (27.7, 49.0)44.2 (35.8, 52.9)
Sex
Male41.2 (35.6, 57.9)47.5 (34.6, 60.7)
Female35.4 (22.2, 50.5)41.6 (30.4, 53.4)
Age Group
<65>35.2 (24.2, 47.5)45.2 (36.2, 54.4)
≥65 years50.0 (24.7, 75.4)35.7 (12.8, 64.9)
Race
White38.4 (27.2, 50.5)43.0 (32.8, 53.7)
Asian57.1 (18.4, 90.1)50.0 (32.9, 67.1)
Other14.3 (0.3, 57.9)33.3 (7.5, 70.1)

Clinical Trial Data

What are the possible side effects?

The most common side effects of ALCENSA are tiredness, constipation, swelling in hands, feet, ankles, and eyelids and muscle pain. Treatment with ALECENSA may cause sunburn when patients are exposed to sunlight.

ALECENSA may cause serious side effects, including liver problems, life-threatening swelling (inflammation) of the lungs, slow heartbeat, and severe muscle problems.

ALECENSA can harm an unborn baby.

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

The tables below summarize adverse reactions, including electrolyte abnormalities, in the clinical trials.

Table 4. Adverse Reactions in ≥ 10% (All Grades) or ≥ 2% (Grade 3-4) of Patients in Studies 1 and 2

Adverse ReactionsALECENSA
N=253
All Grades (%)Grades 3-4 (%)*
Fatiguea411.2
Constipation340
Edemab300.8
Myalgiac291.2
Cough190
Rashd180.4
Nausea180
Headache170.8
Diarrhea161.2
Dyspnea163.6e
Back pain120
Vomiting120.4
Increased weight110.4
Vision disorderf100

* Per Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
a Includes fatigue and asthenia.
b Includes peripheral edema, edema, generalized edema, eyelid edema, and periorbital edema.
c Includes myalgia and musculoskeletal pain.
d Includes rash, maculopapular rash, acneiform dermatitis, erythema, generalized rash, papular rash, pruritic rash, and macular rash.
e Includes one Grade 5 event
f Includes blurred vision, vitreous floaters, visual impairment, reduced visual acuity, asthenopia, and diplopia.
ALECENSA Prescribing Information

Table 5. Laboratory Abnormalities Occurring in >20% of Patients in Studies 1 and 2

ParameterALECENSA
(N=250)
All Grades (%)Grades 3-4 (%)*
Chemistry
Increased AST513.6
Increased Alkaline Phosphatase471.2
Increased CPKa434.6
Hyperbilirubinemia392.4
Hyperglycemiab362.0
Increased ALT344.8
Hypocalcemia320.4
Hypokalemia294.0
Increased Creatininec280
Hypophosphatemia212.8
Hyponatremia202.0
Hematology
Anemia562.0
Lymphopeniad224.6

* Per CTCAE version 4.0
a n=218 for CPK (with baseline values missing for 91 of these patients).
b n=152 for fasting blood glucose (with baseline values missing for 5 of these patients).
c Only patients with creatinine increases based on ULN definition.
d n=217 for lymphocytes (with baseline values missing for 5 of these patients)
ALECENSA Prescribing Information

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

  • Sex: The risk of side effects was similar in men and women
  • Race: The majority patients in the clinical trial were white. Differences in side effects among races could not be determined.
  • Age: The majority of patients in the clinical trial were younger than 65 years of age. Differences in side effects between patients below and above 65 years of age could not be determined.

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

The table below summarizes adverse events during the clinical trial by subgroup.

Table 6. Subgroup Analysis of Treatment-Emergent Adverse Events–All Grades

 ALECENSA (N=253)
Demographic SubgroupTotal, Nn (%)
Sex
Male115113 (98.3%)
Female138136 (98.6%)
Age Group
<65>217214 (98.6%)
>= 65 years3635 (97.2%)
Race
White186183 (98.4%)
Asian4646 (100%)
Other2120 (95.2%)

Clinical Trial Data

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved ALECENSA based on the evidence from two clinical trials of 253 patients with NSCLC with the ALK mutation. The trials were conducted in the USA, Canada, Europe, Asia, and Australia.

The figure below summarizes how many men and women were in the clinical trials.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many men and women were in the clinical trial of the drug ALECENSA.  In total, 115 men (45%) and 138 women (55%) participated in the clinical trial used to evaluate the drug ALECENSA.

Clinical Trial Data

Figure 2 and Table 1 below summarize the percentage of patients by race enrolled in the clinical trials.

Figure 2. Baseline Demographics by Race

Pie chart summarizing the percentage of patients by race enrolled in the ALECENSA clinical trial. In total, 186 Whites (74%), 4 Blacks (1.6%), 46 Asian (18.2%), 11 Other (4.3%), and 4 participants where Race was Unknown (1.6%) participated in the clinical trial.

Clinical Trial Data

Table 1. Demographics of Efficacy Trials by Race

RaceNumber of PatientsPercentage
White18673.5%
Asian4618.2%
Other114.3%
Black or African American41.6%
Unknown41.6%
American Indian or Alaska Native10.4%
Multiple10.4%

Clinical Trial Data

Figure 3 summarizes the percentage of patients by age enrolled in the clinical trials.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many individuals of certain age groups were enrolled in the ALECENSA clinical trial.  In total, 217 participants were below 65 years old (86%) and 36 participants were 65 and older (14%).

Clinical Trial Data

Who participated in the trials?

The table below summarizes demographics of patients in the clinical trials.

Table 7. Baseline Demographics of Patients in the Clinical Trials

Demographic ParametersN=253
Sex
Male115 (45.5%)
Female138 (54.5%)
Age
Mean (SD)52.6 (11.3)
Median (Range)53.0 (22-81)
Age Group
<65>217 (85.8%)
≥65 years36 (14.2%)
Race
White186 (73.5%)
Asian46 (18.2%)
Other11 (4.3%)
Black or African American4 (1.6%)
Unknown4 (1.6%)
American Indian or Alaska Native1 (0.4%)
Multiple1 (0.4%)

Clinical Trial Data

How were the trials designed?

The benefit and side effects of ALECENSA were evaluated in two clinical trials of patients with NSCLC with the ALK mutation. All patients received ALECENSA. The benefit of ALECENSA was evaluated by measuring if and how much the tumor size changed during treatment and how long that response lasted.

How were the trials designed?

The safety and efficacy of ALECENSA were established in two single-arm, multicenter clinical trials. Patients with locally advanced or metastatic ALK-positive NSCLC, who had progressed on crizotinib, with documented ALK positive NSCLC based on an FDA-approved test, and Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0-2 were enrolled in both studies.

The major efficacy outcome measure in both studies was objective response rate (ORR) according to Response Evaluation Criteria in Solid Tumors (RECIST v1.1) as evaluated per Independent Review Committee (IRC).

Additional outcome measures as evaluated by the IRC included duration of response (DOR), central nervous system objective response rate (CNS ORR), and central nervous system duration of response (CNS DOR).

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