Frequently Asked Questions

1) What is Pheburane®?

P = PALATABLE

Pheburane® is the palatable, safe, and effective treatment option for certain Urea Cycle Disorders (UCDs) used along with a specific diet.

 

While taking Pheburane®, it is still possible to experience an acute excess of ammonia in the blood. If this happens, you may develop symptoms such as feeling sick (nausea), being sick (vomiting), confusion, combativeness, slurred speech, difficulty walking, and even loss of consciousness. This is a medical emergency, and medical assistance should be sought immediately.

 

P = PELLETS

Pheburane® pellets are made up of sugar cores with a layer of medication and a special coating designed to mask the awful taste of NaPB. Pheburane® helps the body to eliminate the waste product (nitrogen), thereby reducing the amount of toxic ammonia in the body. Pheburane® must be used along with a low protein diet.

 

Pheburane® coated pellets must be swallowed within 10 seconds so that the coating does not dissolve.

2) Is Pheburane® FDA-approved?

P = PROVEN

Pheburane® was approved by the US Food and Drug Administration in 2022 as a safe and effective treatment option of NaPB for children and adults, used along with a specific diet to treat certain UCDs.

 

Pheburane® is not used for the treatment of acute hyperammonemia, which can be life-threatening and requires emergency medical treatment.

3) Is Pheburane® safe for me or my child?

P = PROVEN

Sodium phenylbutyrate (NaPB) has been the standard of care for the treatment of UCDs for over 25 years.

 

Pheburane® was approved by the US Food and Drug Administration in 2022 as a safe and effective treatment option of NaPB for children and adults, used along with a specific diet.

 

Pheburane® is not used for the treatment of acute hyperammonemia, which can be life-threatening and requires emergency medical treatment.

4) How do I take Pheburane®? / How do I give my child Pheburane®?

Always take Pheburane® exactly as your healthcare provider has told you. The total daily dose of Pheburane® must be divided into equal doses.

 

Plan to take your Pheburane® dose at the same time as food consumption (meal or snack):

  • Swallow with a drink such as water, fruit juice, or protein-free infant formulas.
  • Sprinkle onto a spoonful of apple sauce or carrot puree* and swallow immediately. It is important to swallow right away to prevent the coating from dissolving.

*Taking Pheburane® with other foods has not been studied and is not recommended. Pheburane® should only be taken with soft foods if the child is old enough to eat soft foods.

 

Do not chew Pheburane® oral pellets directly or mix into liquids. If you miss a dose of Pheburane®, take it as soon as possible. Each does of Pheburane® should be taken at least 3 hours apart. If you have missed a dose within 3 hours of the next does, take only one dose the next time it is scheduled.

 

See How to take Pheburane® Guide for downloadable step-by-step instructions or see How to take Pheburane® section for more information.

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Read the Offical Instructions for Use before taking Pheburane® oral pellets and each time you get a refill.

5) How do I get Pheburane®?

P = PRICE

Please see the How to Get Pheburane section for information about the UNIK Patient Support Program. Unik Support offers specialized services to help you or your child, including a Savings program. Medunik USA provides Pheburane® and all its added benefits at a similar cost to other NaPB products.

Important Safety Information

What is Pheburane®?

  • Pheburane® is a prescription medicine, used along with a specific diet, for the long-term management of adults and children with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinate synthetase (AS).
  • Episodes of sudden, rapid increase of ammonia in the blood (acute hyperammonemia) may happen in people during treatment with Pheburane®. Pheburane® is not used for the treatment of acute hyperammonemia, which can be life-threatening and requires emergency medical treatment.

Beforetaking Pheburane®, tell your healthcare provider about all of your medical conditions, including if you:

  • have heart problems.
  • have kidney or liver problems.
  • have diabetes (Pheburane® contains sucrose), or have a history of fructose intolerance, glucose-galactose malabsorption, or sucrose-isomaltase insufficiency.
  • are pregnant or plan to become pregnant. It is not known if Pheburane® will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. It is not known if Pheburane® passes into your breastmilk. Talk to your healthcare provider about the best way to feed your baby during treatment with Pheburane®.

Tell your healthcare provider about all the medicines you or your child take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

 

Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with Pheburane®. Especially tell your healthcare provider if you or your child take:

  • corticosteroids
  • valproic acid
  • haloperidol
  • probenecid

Know the medicines you take. Keep a list of them to show your or your child’s healthcare provider and pharmacist when you get a new medicine.

What are the possible side effects of Pheburane®?

Pheburane® can cause serious side effects, including: Nervous system problems (neurotoxicity).

 

Call your healthcare provider right away if you get any of the following symptoms during treatment with Pheburane®:

  • sleepiness
  • tiredness
  • lightheadedness
  • vomiting
  • nausea
  • headache
  • confusion

Low potassium levels in your blood (hypokalemia). Your healthcare provider will monitor your blood potassium levels during treatment with PHEBURANE and treat if needed.

Conditions related to swelling (edema).Pheburane® contains salt (sodium), which can cause swelling from salt and water retention. Your healthcare provider will decide if PHEBURANE is right for you if you have certain medical conditions that can cause swelling, such as heart failure, liver problems or kidney problems.

The most common side effects of Pheburane® include:

  • absent or irregular menstrual periods
  • decreased appetite
  • body odor
  • bad taste or avoiding foods that you ate prior to getting sick (taste aversion)

 

Your healthcare provider may do certain blood tests to check you or your child for side effects during treatment with Pheburane®.

 

These are not all the possible side effects of Pheburane®.

 

Call your doctor for medical advice about side effects.

 

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch. or call 1-800-FDA-1088.

 

Please read the Full Prescribing Information, the Patient Information. and the Instruction for Use. before taking Pheburane® oral pellets and each time you get a refill.