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Drug Trials Snapshots: VYEPTI

 

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 VYEPTI Package Insert for complete information.

VYEPTI (eptinezumab-jjmr)
vye ep' tee
Lundbeck
Approval date: February 21, 2020


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

VYEPTI is a drug used for the preventive treatment of migraine in adults.

A migraine is a type of headache that can be associated with nausea, vomiting, and/or sensitivity to light or sound.

How is this drug used?

VYEPTI is an injection given directly into the vein (intravenous infusion) by a healthcare provider every three months. It takes about 30 minutes to receive the VYEPTI infusion.

What are the benefits of this drug?

Over the three-month treatment period, patients treated with VYEPTI experienced fewer days of migraine headaches in comparison to patients who received the placebo treatment.

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

The tables below summarize efficacy results for the evaluated patients in Trials 1 and 2. The primary outcome was the change in monthly migraine days (MMD).

Table 1. Efficacy Endpoint Results in Trial 1

 

VYEPTI
100 mg

N=221

VYEPTI
300 mg

N=222

Placebo

 

N=222

Monthly Migraine Days (MMD) – Months 1-3

 

Change from baseline

-3.9

-4.3

-3.2

Difference from placebo

-0.7

-1.1

 

p-value

0.018

<0.001

 

50% MMD responders – Months 1-3

 

% Responders

49.8%

56.3%

37.4%

Difference from placebo

12.4%

18.9%

 

p-value

0.009*

<0.001

 

75% MMD responders – Months 1-3

 

% Responders

22.2%

29.7%

16.2%

Difference from placebo

6.0%

13.5%

 

p-value

NS**

<0.001

 

* Nominal statistical significance
** NS = Not statistically significant

VYEPTI Prescribing Information

Table 2. Efficacy Endpoint Results in Trial 2

 

VYEPTI
100 mg

N=356

VYEPTI
300 mg

N=350

Placebo

 

N=366

Monthly Migraine Days (MMD) – Months 1-3

 

Change from baseline

-7.7

-8.2

-5.6

Difference from placebo

-2.0

-2.6

 

p-value

<0.001

<0.001

 

50% MMD responders – Months 1-3

 

% Responders

57.6%

61.4%

39.3%

Difference from placebo

18.2%

22.1%

 

p-value

<0.001

<0.001

 

75% MMD responders – Months 1-3

 

% Responders

26.7%

33.1%

15.0%

Difference from placebo

11.7%

18.1%

 

p-value

<0.001

<0.001

 

VYEPTI Prescribing Information

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

  • Sex: VYEPTI worked similarly in men and women.
  • Race: The majority of patients were White. The number of patients in other races was limited; therefore, differences in how VYEPTI worked among races could not be determined.
  • Age: VYEPTI worked similarly in all tested age groups. The number of patients older than 65 years was insufficient to determine if VYEPTI worked differently in this age group.

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

The figures below summarize efficacy results by subgroups in Trials 1 and 2, respectively.

Figure 5. Forest Plot of Difference from Placebo in Monthly Migraine Days Change from Baseline Over Weeks 1-12 by Subgroup – VYEPTI 300 mg-Trial 1

Figure 5

 

Figure 6. Forest Plot of Difference from Placebo in Monthly Migraine Days Change from Baseline Over Weeks 1-12 by Subgroup – VYEPTI 100 mg-Trial 1

Figure 6

 

Figure 7. Forest Plot of Difference from Placebo in Monthly Migraine Days Change from Baseline Over Weeks 1-12 by Subgroup – VYEPTI 300 mg-Trial 2

Figure 7

 

Figure 8. Forest Plot of Difference from Placebo in Monthly Migraine Days Change from Baseline Over Weeks 1-12 by Subgroup – VYEPTI 100 mg-Trial 2

Figure 8

ALD403 300=VYEPTI 300 mg

FDA Statistical Review

What are the possible side effects?

VYEPTI may cause serious allergic reactions including swelling of face, tongue or throat, hives, trouble breathing, facial redness and rash.

The most common side effects of VYEPTI are stuffy nose and scratchy throat (nasopharyngitis) and allergic reactions.

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

The table below summarizes adverse reactions in adult patients with chronic or episodic migraine headaches in combined trials (safety population).

Table 4. Adverse Reactions Occurring with an Incidence of At Least 2% for VYEPTI At Least 2% Greater Than Placebo in Trials 1 and 2

Adverse Reactions

VYEPTI 100 mg
N=579
%

VYEPTI 300 mg
N=574
%

Placebo
N=588
%

Nasopharyngitis

6

8

6

Hypersensitivity reactions*

1

2

0

* Hypersensitivity reactions includes multiple related adverse event terms, such as hypersensitivity, pruritus, and flushing/hot flush that occurred on the day of dosing.

VYEPTI Prescribing Information

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

  • Sex: The occurrence of side effects was similar in men and women.
  • Race: The majority of patients were White. The number of patients in other races was limited; therefore, differences in side effects among races could not be determined.
  • Age: The occurrence of side effects was similar in patients younger and older than 40 years of age. The number of patients older than 65 years was insufficient to determine if the occurrence of side effects in this age group differ from the younger patients.

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

The table below summarizes adverse events by subgroups in combined Trials 1 and 2.

Table 5. Rates of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) by Sex, Race and Age Subgroups Occurring in VYEPTI Treatment Arms (safety population)

 

Sex

Race

Age

Men
N = 154
n (%)

Women
N = 999
n (%)

White
N = 1037
n (%)

Black or African American
N = 88
n (%)

Other*
N = 28
n (%)

< 40 y
N = 549
n (%)

≥ 40 y
N = 604
n (%)

TEAEs

79
(51)

528
(53)

530
(51)

59
(67)

18
(64)

297
(54)

310
(51)

SAEs

1
(1)

13
(1)

12
(1)

2
(2)

0

5
(1)

9
(1)

*Other races = Asian, American Indian, Native Hawaiian, Multiple, Other, or Not Reported

Adapted from FDA Clinical Review

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved VYEPTI based primarily on evidence from two clinical trials (Trial 1/ NCT02559895 and Trial 2/ NCT02974153) of 1741 patients with chronic or episodic migraine headaches. Trials were conducted at 212 sites in United States, Georgia, Russia, Ukraine and European Union.

Figure 1 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. In total, 1510 women (87%) and 231 men (13%) participated in the clinical trial.

Adapted from FDA Review

Figure 2 summarizes the percentage of patients by race in the clinical trials.

Figure 2. Baseline Demographics by Race

Pie chart summarizing the percentage of patients by race enrolled in the clinical trial. In total, 1539 White (88%), 156 Black or African American  (9%) and 46 Other (3%)

*Other includes Asian, American Indian or Alaska Native, Native Hawaiian or Other Pacific Islander Multiple and Other

Adapted from FDA Review

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

Figure 3. Baseline Demographics by Age

Pie charts summarizing how many individuals of certain age groups were enrolled in the clinical trial. In total,  618 (35%) were 18 to 35 years, 1104 were 35 to 64 years (63%), 19 were 65 years and older (1%).

Adapted from FDA Review

Figure 4 summarizes the percentage of patients by ethnicity in the clinical trials.

Figure 4. Baseline Demographics by Ethnicity

 Pie charts summarizing how many individuals of certain ethnicity were enrolled in the clinical trial. In total,  202 patients were Hispanic or Latino (12%), and 1539 (88%) patients were not Hispanic or Latino.

Adapted from FDA Review

Who participated in the trials?

The table below summarizes demographics of the safety population.

Table 7. Baseline Demographics of the Safety Population

 

 

Demographic Parameters

VYEPTI 100 mg
N = 579
n (%)

VYEPTI 300 mg
N = 574
n (%)

Placebo
N=588
n (%)

Overall
N = 1741
n (%)

Sex

 

 

 

 

  Men

93 (16)

61 (11)

77 (13)

231 (13)

  Women

486 (84)

513 (89)

511 (87)

1510 (87)

Race

 

 

 

 

  White

528 (91)

509 (89)

502 (85)

1539 (88)

  Black or African American

38 (7)

50 (9)

68 (12)

156 (9)

  Asian

2 (<1)

2 (<1)

3 (0.5)

7 (<1)

  American Indian or Alaska Native

1 (<1)

3 (0.5)

2 (<1)

6 (<1)

  Native Hawaiian or Other Pacific Islander

1 (<1)

2 (<1)

1

4 (<1)

  Multiple

8 (1)

7 (1)

9 (1.5)

24 (1)

  Other

1(<1)

1 (<1)

3 (0.5)

5 (<1)

Age (years)

 

 

 

 

  Median (years)

41

40

40

40

  Min, max (years)

18, 68

18, 71

18, 71

18, 71

Age Group

 

 

 

 

  ≤ 35 years

186 (32)

203 (35)

229 (39)

618 (36)

  > 35 years and < 65 years

390 (67)

364 (63)

350 (60)

1104 (63)

  ≥ 65 years

3 (1)

7 (1)

9 (2)

19 (1)

Ethnicity

 

 

 

 

  Hispanic or Latino

75 (13)

58 (10)

69 (11.7)

202 (12)

  Not Hispanic or Latino

504 (87)

516 (90)

519 (88)

1539 (88)

Region/Country

 

 

 

 

  United States

382 (66)

381 (66)

417 (71)

1180 (68)

  Republic of Georgia, Russia,    
  and Ukraine

147 (25)

140 (24)

120 (20)

407(23)

  European Union

50 (9)

53 (9)

51 (9)

154 (9)

Adapted from FDA Clinical Review

How were the trials designed?

The benefit and side effects of VYEPTI were evaluated in two clinical trials of adult patients 18 – 71 years of age with a history of migraine headaches. The trials had similar designs.

Trial 1 enrolled patients with a history of episodic migraine headaches and Trial 2 enrolled patients with chronic migraine headaches. Patients were assigned to receive one of two doses of VYEPTI or placebo injections every 3 months for a total of 12 months in Trial 1, and for a total of 6 months in Trial 2. Neither the patients nor the health care providers knew which treatment was being given until the trial was completed.

The benefit of VYEPTI in comparison to placebo was assessed based on the change in the number of migraine days per month during the first 3-month treatment period.

How were the trials designed?

The efficacy and safety of VYEPTI were evaluated in two similar, randomized, multicenter, placebo-controlled clinical trials.

Trial 1 enrolled patients with episodic migraine. VYEPTI or placebo was administered by intravenous infusion every 3 months for 12 months; however, the primary endpoint was measured at 12 weeks.

Trial 2 enrolled patients with chronic migraine. VYEPTI or placebo was administered by intravenous infusion every 3 months for 6 months; the primary endpoint was measured at 12 weeks.

The primary efficacy endpoint for both trials was the change from baseline in mean monthly migraine days over Months 1-3. Secondary endpoints included the percentages of patients with 50% or greater and 75% or greater reductions from baseline in monthly migraine days over Months 1-3.

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.

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