Tab A - VIMIZIM® (elosulfase alfa)

VIMIZIM^® (elosulfase alfa) improves endurance at 6 months

In a 24-week clinical trial, people who received VIMIZIM infusions every week improved their endurance as measured by the 6-minute walk test (6MWT).

The number of people included in the study was intentionally chosen to see the effect of VIMIZIM on the 6-minute walk test and the 3-minute stair climbing test. Other tests or measures were assessed to see if they might be valid for future study.

VIMIZIM^® (elosulfase alfa) improves endurance at 6 months

In a 24-week clinical trial, people who received VIMIZIM infusions every week improved their endurance as measured by the 6-minute walk test (6MWT).

The number of people included in the study was intentionally chosen to see the effect of VIMIZIM on the 6-minute walk test and the 3-minute stair climbing test. Other tests or measures were assessed to see if they might be valid for future study.

This graph shows that by the end of a 24-week trial, people who took VIMIZIM once a week walked an average of 23.9% farther than at baseline

People who took VIMIZIM every other week did not show much improvement, and their results were similar to the people who took a placebo

Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis). In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis.

Maintaining weekly infusions is essential to making the most out of your treatment

6MWT change from baseline (m)^{1, 18, 21}

^{aA baseline is a measurement of a person’s performance before they begin treatment in a clinical trial. To participate in the clinical trial for VIMIZIM, people had to be able to walk more than 30 meters but less than 325 meters in 6 minutes.}^{bA placebo is a harmless substance that does not contain any medicine.}

6MWT change from baseline (m)^{1, 18, 21}

^{aA baseline is a measurement of a person’s performance before they begin treatment in a clinical trial. To participate in the clinical trial for VIMIZIM, people had to be able to walk more than 30 meters but less than 325 meters in 6 minutes.}^{bA placebo is a harmless substance that does not contain any medicine.}

This graph shows that by the end of a 24-week trial, people who took VIMIZIM once a week walked an average of 23.9% farther than at baseline

People who took VIMIZIM every other week did not show much improvement, and their results were similar to the people who took a placebo

Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis). In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis.

Maintaining weekly infusions is essential to making the most out of your treatment

6MWT change from baseline (m)^{1, 18, 21}

VIMIZIM^® (elosulfase alfa) also showed positive results across many efficacy measures

Although statistical significance was only observed in the 6MWT results, favorable improvement was seen in the majority of exploratory efficacy endpoints.

VIMIZIM^® (elosulfase alfa) also showed positive results across many efficacy measures

Although statistical significance was only observed in the 6MWT results, favorable improvement was seen in the majority of exploratory efficacy endpoints.

ENDPOINT
(Placebo:VIMIZIM) / Mean (CI)

6-minute walk test (6MWT)
(59:58) / 22.46 (4.01, 40.91)

3-minute stair-climbing test (3MSCT)
(59:58) / 1.14 (-2.14, 4.43)

Time required to exhale (FET)
(59:58) / 42.50 (-14.91, 99.91)

Amount of air exhaled in a forced breath (FVC)
(59:58) / 3.26 (-3.13, 9.64)

Amount of air inhaled (FIVC)
(59:58) / -2.59 (-65.66, 60.48)

Talk to your doctor about your individual VIMIZIM treatment goals

Knowing what to expect from your treatment with VIMIZIM can help you know what “success” looks like. For some people, this might mean that the impact of Morquio A becomes stabilized. Work with your doctor to come up with treatment goals that are right for you.

See the side effects of vimizim

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The scale values were standardized to a common scale of measurement by dividing the estimate and confidence interval (CI) bounds for each endpoint by the standard error of that estimate.

ADDITIONAL ENDPOINTS: FEV1 favors VIMIZIM (59:58) / 1.82 (-5.52, 9.17), MVV favors VIMIZIM (59:58) / 10.31 (-1.79, 22.42), Growth rate favors VIMIZIM (40:44) / 0.39 (-0.08, 0.87), Z-score height favors VIMIZIM (40:44) / 0.14 (-0.03, 0.31), MPS HAQ caregiver favors VIMIZIM (58:57) / 0.85 (-1.14, 2.85), MPS HAQ mobility favors VIMIZIM (59:57) / 0.26 (-0.28, 0.80), MPS HAQ self-care favors Placebo (59:57) / -0.07 (-0.47, 0.33).

FEV1=forced expiratory volume in 1 second; MVV=maximum voluntary ventilation; Z-score height=how far a measurement deviates from the average; MPS HAQ caregiver=caregiver-completed MPS Health Assessment Questionnaire; MPS HAQ mobility=patient-completed MPS Health Assessment Questionnaire on mobility; MPS HAQ self-care=patient-completed MPS Health Assessment Questionnaire on self-care.