Pronunciation:

VEL-man-ase AL-fa - tycv

Brand Names:

• Lamzede

Dosage Forms:

• Powder for Solution

Warnings:

Hypersensitivity Reactions Including AnaphylaxisPatients treated with velmanase alfa-tycv have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during velmanase alfa-tycv administration. If a severe hypersensitivity reaction (eg anaphylaxis) occurs, discontinue velmanase alfa-tycv immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to velmanase alfa-tycv may be considered .

Classifications:

Therapeutic—

Enzyme Replacement

Pharmacologic—

Velmanase Alfa

Uses of This Medicine:

Velmanase alfa-tycv injection is used to treat non-central nervous system (CNS) symptoms of alpha-mannosidosis. Alpha-mannosidosis is a rare genetic disease caused by a deficiency of an enzyme called alpha-mannosidase.

This medicine is to be given only by or under the direct supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of velmanase alfa-tycv injection in children. Safety and efficacy have been established.

Older adults—

Alpha-mannosidosis is a disease that usually affects children and young adults. Appropriate studies on the relationship of age to the effects of velmanase alfa-tycv injection have not been performed in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Proper Use of This Medicine:

A nurse or other trained health professional will give you this medicine in a hospital. This medicine is given through a needle placed in one of your veins. It is usually given once every week.

Your doctor will give you other medicines (eg, allergy medicine, fever medicine, or steroid medicine) before you receive this medicine to help prevent unwanted effects.

Missed dose—

Call your doctor or pharmacist for instructions.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress closely and at regular visits to make sure this medicine is working properly and to decide if you should continue to receive it. Blood tests may be needed to check for unwanted effects.

Using this medicine could harm your unborn baby. If you are a woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before starting this treatment. Use an effective form of birth control during treatment with this medicine and for 14 days after the last dose to keep from getting pregnant. Tell your doctor right away if you think you have become pregnant.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you have a rash, itching, hoarseness, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth after using this medicine.

This medicine may cause infusion-related reactions, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you start to have fever, chills or shaking, dizziness, trouble breathing, itching or rash, lightheadedness, or fainting after receiving this medicine.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

Back pain

bladder pain

bloody or cloudy urine

change in hearing

chest tightness

chills

cough

diarrhea

difficult, burning, or painful urination

ear drainage or pain

fainting

fast heartbeat

fever

frequent urge to urinate

general feeling of discomfort or illness

headache

hives, itching, skin rash

irritation

joint pain, stiffness, or swelling

loss of appetite

lower back or side pain

muscle aches and pains

nausea

redness of the skin

shivering

sore throat

stomach pain

stuffy or runny nose

sweating

swelling of the eyelids, face, lips, hands, or feet

trouble breathing

unusual tiredness or weakness

vomiting

Less common

Agitation

coma

confusion

decreased urine output

depression

dizziness

hostility

lethargy

muscle twitching

rapid weight gain

seizures

stupor

swelling of the face, ankles, or hands

Incidence not known

Blurred vision

chest pain or discomfort

deafness

delusions

dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position

fast, irregular, pounding, or racing heartbeat or pulse

hallucinations

heart murmur

shakiness and unsteady walk

sleepiness or unusual drowsiness

unsteadiness, trembling, or other problems with muscle control or coordination

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Difficulty in moving

joint pain

toothache

Incidence not known

Decreased appetite

joint swelling or warmth

lack or loss of strength

large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs

watering of the eyes

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.