Fosdenopterin

Fosdenopterin is used to lower the risk of death in children with molybdenum cofactor deficiency (MoCD) Type A.

Molybdenum (moe-LIB-den-um) cofactor deficiency is a rare condition in newborns that impacts brain function and worsens over time. Babies with MoCD start to show symptoms within a week after birth, and this condition can lead to serious developmental delays and life-threatening health issues.

Fosdenopterin may also be used for reasons not mentioned in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your child's dose. Use the medicine exactly as directed.

Fosdenopterin is given using an infusion pump attached to a catheter placed into a vein. A healthcare provider can teach you how to properly use the infusion pump by yourself.

You may need to keep a diary of the dates and times for each dose you give to your child. You may also need to record information about the specific vials and amounts of fosdenopterin you use each time. Take this information with you every time you take your child to the doctor.

Read and carefully follow any Instructions for Use provided with your medicine. Ask your doctor or pharmacist if you don't understand all instructions.

Fosdenopterin must be mixed with a liquid (diluent) before using it. When using injections by yourself, be sure you understand how to properly mix and store the medicine.

Do not shake the mixed medicine.

Prepare an injection only when you are ready to give it. Each infusion should be finished within 4 hours after you mixed the medicine with the diluent.

Do not use if the medicine looks cloudy, has changed colors, or has particles in it. Call your pharmacist for new medicine.

Fosdenopterin doses are based on weight. Your child's dose needs may change if the child gains or loses weight.

Store unmixed fosdenopterin in a freezer. Keep each vial in its original carton and protect from light.

Take the medicine out of the freezer and let it reach room temperature for 30 minutes before mixing with the diluent. You may also warm the medicine in your hands for 3 to 5 minutes. Do not heat the medicine.

You may store mixed medicine in the refrigerator or at room temperature. If stored in a refrigerator, allow the mixture to reach room temperature before giving the injection. Complete the injection within 4 hours after mixing the medicine.

Follow all directions on your medicine label and package.

Tell each of your child's healthcare providers about all the child's medical conditions, allergies, and medicines.

Your doctor will do a genetic test to confirm if your baby has MoCD.

Fosdenopterin may be given before the genetic test results are ready. Treatment will be stopped if the test shows that your baby does not have MoCD.

Get emergency medical help if your child shows signs of an allergic reaction: hives; difficulty breathing; swelling of the face, lips, tongue, or throat.

Call your doctor right away if your child has:

• a fever;
• severe or ongoing vomiting or diarrhea;
• bruising, swelling, discomfort, or skin changes around the catheter;
• a cough with mucus, or trouble breathing;
• a seizure; or
• signs of an ear infection, such as fever, ear pain, drainage from the ear, or fussiness.

Seek emergency medical help if your child has a skin rash or serious sunburn symptoms like skin redness, burning, or blisters.

Common side effects of fosdenopterin may include:

• problems with the catheter or IV line;
• fever;
• vomiting or diarrhea; or
• cold symptoms like a stuffy nose, sneezing, or cough.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Usual Adult Dose for Combined Molybdoflavoprotein Enzyme Deficiency:

0.9 mg/kg IV once a dayComments:-Dosing is based on actual body weight; infuse at a rate of 1.5 mL/min with non-DEHP tubing and a 0.2-micron filter.-Therapy may be started in patients with a diagnosis or presumptive diagnosis of molybdenum cofactor deficiency (MoCD) Type A; for a presumptive diagnosis, confirm MoCD Type A immediately after initiation with genetic testing and discontinue this drug if the diagnosis is not confirmed.Use: To reduce the risk of mortality in patients with MoCD Type A.

Usual Pediatric Dose for Combined Molybdoflavoprotein Enzyme Deficiency:

Patients less than 1 year: Dosing is based on gestational ageGestational age less than 37 weeks (preterm neonates): -Initial Dosage: 0.4 mg/kg IV once a day -Dose at one month: 0.7 mg/kg IV once a day-Dose at three months: 0.9 mg/kg IV once a dayGestational age 37 weeks and above (term neonates): -Initial Dosage: 0.55 mg/kg IV once a day -Dose at one month: 0.75 mg/kg IV once a day-Dose at three months: 0.9 mg/kg IV once a dayPatients 1 year or older: 0.9 mg/kg IV once a day Comments:-Dosing is based on actual body weight; infuse at a rate of 1.5 mL/min with non-DEHP tubing and a 0.2-micron filter (volumes below 2 mL may require syringe administration through slow intravenous push). -Therapy may be started in patients with a diagnosis or presumptive diagnosis of molybdenum cofactor deficiency (MoCD) Type A; for a presumptive diagnosis, confirm MoCD Type A immediately after initiation with genetic testing and discontinue this drug if the diagnosis is not confirmed.Use: To reduce the risk of mortality in patients with MoCD Type A.

Give the medicine as soon as you can, then give the next injection at least 6 hours later and return to the regular daily injection schedule. Do not give two doses at one time.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Fosdenopterin could make your child more likely to get sunburned. Avoid sunlight or tanning beds. Make sure the child wears protective clothing, a hat, and sunglasses while outside. Also apply sunscreen (SPF 30 or higher) if your child is at least 6 months old.

Other drugs may affect fosdenopterin, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your child's current medicines and any medicines the child starts or stops using.