Symptom improvement with MEPSEVII™

MEPSEVII® (vestronidase alfa-vjbk) was found to be effective in clinical trials

MEPSEVII® (vestronidase alfa-
vjbk) was found to be effective in clinical trials

The effectiveness of MEPSEVII was determined using several tests, including:

Six-minute walk test (6MWT)
which measures how far someone can walk in 6 minutes. It is an effective way of measuring how well the different systems in the body work together

Breathing ability
which is measured to see how healthy the respiratory system is

Liver and spleen size
which measures whether these organs are of normal or abnormal size

Due to the nature of MPS VII and the differences in the patients in this trial, you should talk with your doctor about what you may expect from treatment with MEPSEVII

In clinical trials, some patients who took MEPSEVII experienced improvement or maintained walking distance, improvement in breathing ability, and reduction in organ size

Observed walking distance after 184 weeks for 10 of 12 patients who took MEPSEVII

At the end of 184 weeks, 3 of the 10 patients had at least a 60-meter (197-foot) improvement in distance walked compared to when they started treatment with MEPSEVII

Of the 3 patients who improved on their 6MWT, 2 patients improved in their balance and overall movement ability^a

^aDetermined using the Bruininks-Oseretsky Test of Motor Proficiency (BOT-2).

Relatively stable course in the remaining 7 of 10 patients, including those who used assistive devices

Patient 10 did not use an assistive device at the start of the study but started using an assistive device at Week 8 of his treatment. Patients 6 and 9 consistently used an assistive device at all visits. A solid line indicates the unassisted assessments and a dotted line indicates the assisted assessments.

Breathing ability

After 120 weeks of exposure to MEPSEVII, 1 out of 3 patients showed:

21% improvement in breathing ability^a compared to the start of the study

The same patient showed:

105-meter improvement (344 feet) in walking distance (6MWT) compared to the start of the study

^aAs measured by forced vital capacity (FVC), which is the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible.

Organ size

In one study, 7 out of 12 patients’ liver and spleen sizes were assessed. Most sizes were normal or below normal in size at the beginning of the study and were unchanged after treatment with MEPSEVII for up to 48 weeks

In a separate study, liver and spleen sizes were reduced after 36 weeks of treatment with MEPSEVII in the 2 out of 3 patients who had abnormally enlarged livers and spleens before treatment with MEPSEVII.

In this study, liver size was reduced by 24% in one patient, and 53% in the other patient. Spleen size was reduced by 28% in one patient, and 47% in the other patient.

In yet another study of 8 patients under 5 years of age who took MEPSEVII for 48 weeks and up to an additional 240 weeks, no substantial changes in liver or spleen size were observed.

Observed walking distance after 184 weeks for 10 of 12 patients who took MEPSEVII

At the end of 184 weeks, 3 of the 10 patients had at least a 60-meter (197-foot) improvement in distance walked compared to when they started treatment with MEPSEVII

Of the 3 patients who improved on their 6MWT, 2 patients improved in their balance and overall movement ability^a

^aDetermined using the Bruininks-Oseretsky Test of Motor Proficiency (BOT-2).

Relatively stable course in the remaining 7 of 10 patients, including those who used assistive devices

Patient 10 did not use an assistive device at the start of the study but started using an assistive device at Week 8 of his treatment. Patients 6 and 9 consistently used an assistive device at all visits. A solid line indicates the unassisted assessments and a dotted line indicates the assisted assessments.

Breathing ability

After 120 weeks of exposure to MEPSEVII, 1 out of 3 patients showed:

21% improvement in breathing ability^a compared to the start of the study

The same patient showed:

105-meter improvement (344 feet) in walking distance (6MWT) compared to the start of the study

^aAs measured by forced vital capacity (FVC), which is the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible.

Organ size

In one study, 7 out of 12 patients’ liver and spleen sizes were assessed. Most sizes were normal or below normal in size at the beginning of the study and were unchanged after treatment with MEPSEVII for up to 48 weeks

In a separate study, liver and spleen sizes were reduced after 36 weeks of treatment with MEPSEVII in the 2 out of 3 patients who had abnormally enlarged livers and spleens before treatment with MEPSEVII.

In this study, liver size was reduced by 24% in one patient, and 53% in the other patient. Spleen size was reduced by 28% in one patient, and 47% in the other patient.

In yet another study of 8 patients under 5 years of age who took MEPSEVII for 48 weeks and up to an additional 240 weeks, no substantial changes in liver or spleen size were observed.

All patients treated with MEPSEVII experienced a reduction of urinary of GAGs, which was sustained with continued treatment.

What is MEPSEVII?

MEPSEVII is used to treat children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome). The effect of MEPSEVII on the central nervous system (brain and spinal cord) symptoms of MPS VII has not been determined.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about MEPSEVII?

A severe allergic reaction called anaphylaxis has occurred with MEPSEVII treatment, as early as the first dose.
Your doctor will monitor you closely for symptoms of an allergic reaction while you are receiving MEPSEVII and for 60 minutes after your injection.
Your doctor will immediately discontinue the MEPSEVII infusion if you experience anaphylaxis.
Your doctor should talk to you about the signs and symptoms of anaphylaxis and about getting medical treatment if you have symptoms after leaving the doctor’s office or treatment center.

What are the possible side effects of MEPSEVII?

The most common side effects of MEPSEVII are:
- Leakage of MEPSEVII into the surrounding tissue during infusion
- Diarrhea
- Rash
- Severe allergic reaction (anaphylaxis)
- Infusion site swelling
- Swelling around the infusion site
- Severe itching of the skin
One patient experienced a seizure during a fever while taking MEPSEVII.

Before receiving MEPSEVII, tell your doctor about all of your medical conditions, including if you:

are pregnant, think you may be pregnant, or plan to become pregnant. There is not enough experience to know if MEPSEVII may harm your unborn baby.
are breastfeeding or plan to breastfeed. There is not enough experience to know if MEPSEVII passes into your breast milk. Talk with your doctor about the best way to feed your baby while you receive MEPSEVII.

These are not all the possible side effects of MEPSEVII. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Ultragenyx at 1-888-756-8657.

Please see full Prescribing Information for additional Important Safety Information including serious side effects.