Pheburane

sodium phenylbutyrate

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This is a summary of the European public assessment report (EPAR) for Pheburane. It explains how the Agency assessed the medicine to recommend its authorisation in the European Union (EU) and its conditions of use. It is not intended to provide practical advice on how to use Pheburane.

For practical information about using Pheburane, patients should read the package leaflet or contact their doctor or pharmacist.

What is Pheburane and what is it used for?

Pheburane is a medicine that contains the active substance sodium phenylbutyrate. It is used to treat patients who have urea-cycle disorders. These patients are not able to get rid of waste nitrogen from the body because they lack some enzymes that are usually found in the liver. In the body, waste nitrogen is in the form of ammonia, which is toxic when it accumulates, especially for the brain. Pheburane is used in patients who lack one or more of the following enzymes: carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinate synthetase. It can be used in patients with the following forms of the disease:

  • 'early-onset' disease in patients who show a complete lack of one or more of these enzymes within the first month of life;
  • 'late-onset' disease in patients who show a partial lack of one or more of these enzymes after the age of one month and have had high blood ammonia levels that affected the brain’s activity.

Pheburane is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but Pheburane granules are available at a lower strength and contain different excipients (inactive ingredients) to mask the unpleasant taste of the active substance. The reference medicine for Pheburane is Ammonaps.

How is Pheburane used?

Pheburane is available as granules (483 mg/g). It can only be obtained with a prescription and treatment should be supervised by a doctor who has experience in treating patients with urea-cycle disorders.

Pheburane is used with a special low-protein diet to reduce the intake of nitrogen. The daily dose of Pheburane is adjusted for each patient individually and depends on the patient’s diet, height and weight. Regular blood tests are needed to find the correct daily dose.

The daily dose of Pheburane should be divided into equal amounts and given with each meal. The granules can be sprinkled onto food immediately before being swallowed or placed in the mouth and swallowed immediately with a drink.

Pheburane may be a life-long treatment unless the patient has a successful liver transplant.

How does Pheburane work?

Eating protein brings nitrogen into the body, which is then transformed into ammonia. Patients with urea-cycle disorders cannot get rid of ammonia from the body, so it can reach high levels, leading to serious problems including disability, brain damage and death. The active substance in Pheburane, sodium phenylbutyrate, is converted into a substance called phenylacetate in the body. Phenylacetate combines with the amino acid glutamine, which contains nitrogen, to form a substance that can be removed from the body by the kidneys. This allows the levels of nitrogen in the body to decrease, reducing the amount of ammonia produced.

How has Pheburane been studied?

Studies in patients have been limited to tests to determine that Pheburane is bioequivalent to the reference medicine, Ammonaps. Two medicines are bioequivalent when they produce the same levels of the active substance in the body.

What are the benefits and risks of Pheburane?

Because Pheburane is a hybrid medicine and is bioequivalent to the reference medicine, its benefits and risks are taken as being the same as the reference medicine’s.

Why is Pheburane approved?

The Agency’s Committee for Medicinal Products for Human Use (CHMP) concluded that Pheburane has been shown to have comparable quality and to be bioequivalent to Ammonaps. Therefore, the CHMP’s view was that, as for Ammonaps, the benefit outweighs the identified risk. The Committee recommended that Pheburane be approved for use in the EU.

What measures are being taken to ensure the safe and effective use of Pheburane?

Safety information has been included in the summary of product characteristics and the package leaflet for Pheburane, including the appropriate precautions to be followed by healthcare professionals and patients.

Other information about Pheburane

The European Commission granted a marketing authorisation valid throughout the European Union for Pheburane on 31 July 2013.

For more information about treatment with Pheburane, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Name Language First published Last updated
Pheburane : EPAR - Summary for the public BG = bălgarski 2013-08-08  
Pheburane : EPAR - Summary for the public ES = español 2013-08-08  
Pheburane : EPAR - Summary for the public CS = čeština 2013-08-08  
Pheburane : EPAR - Summary for the public DA = dansk 2013-08-08  
Pheburane : EPAR - Summary for the public DE = Deutsch 2013-08-08  
Pheburane : EPAR - Summary for the public ET = eesti keel 2013-08-08  
Pheburane : EPAR - Summary for the public EL = elliniká 2013-08-08  
Pheburane : EPAR - Summary for the public EN = English 2013-08-08  
Pheburane : EPAR - Summary for the public FR = français 2013-08-08  
Pheburane : EPAR - Summary for the public IT = italiano 2013-08-08  
Pheburane : EPAR - Summary for the public LV = latviešu valoda 2013-08-08  
Pheburane : EPAR - Summary for the public LT = lietuvių kalba 2013-08-08  
Pheburane : EPAR - Summary for the public HU = magyar 2013-08-08  
Pheburane : EPAR - Summary for the public MT = Malti 2013-08-08  
Pheburane : EPAR - Summary for the public NL = Nederlands 2013-08-08  
Pheburane : EPAR - Summary for the public PL = polski 2013-08-08  
Pheburane : EPAR - Summary for the public PT = português 2013-08-08  
Pheburane : EPAR - Summary for the public RO = română 2013-08-08  
Pheburane : EPAR - Summary for the public SK = slovenčina 2013-08-08  
Pheburane : EPAR - Summary for the public SL = slovenščina 2013-08-08  
Pheburane : EPAR - Summary for the public FI = suomi 2013-08-08  
Pheburane : EPAR - Summary for the public SV = svenska 2013-08-08  

This EPAR was last updated on 29/09/2017 .

Authorisation details

Product details

Product details for Pheburane
NamePheburane
Agency product numberEMEA/H/C/002500
Active substance

sodium phenylbutyrate

International non-proprietary name (INN) or common name

sodium phenylbutyrate

Therapeutic area Carbamoyl-Phosphate Synthase I Deficiency Disease
Anatomical therapeutic chemical (ATC) code A16AX03

Publication details

Publication details for Pheburane
Marketing-authorisation holder

Lucane Pharma

Revision3
Date of issue of marketing authorisation valid throughout the European Union31/07/2013

Contact address:

Lucane Pharma
172 rue de Charonne
75011 Paris
France

Product information

Product information

20/07/2017  Pheburane -EMEA/H/C/002500 -IB/0016

Name Language First published Last updated
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29
Pheburane : EPAR - Product Information SV = svenska 2013-08-08 2017-09-29

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  
Pheburane : EPAR - All Authorised presentations SV = svenska 2013-08-08  

Pharmacotherapeutic group

Various alimentary-tract and metabolism products

Therapeutic indication

Treatment of chronic management of urea-cycle disorders.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Pheburane : EPAR - Procedural steps taken and scientific information after authorisation SV = svenska 2014-10-06 2017-09-29

Initial marketing-authorisation documents

Name Language First published Last updated
Pheburane : EPAR - Public assessment report SV = svenska 2013-08-08  
CHMP summary of positive opinion for Pheburane SV = svenska 2013-02-22  

Authorised

This medicine is approved for use in the European Union

More information on Pheburane

This product is no longer an orphan medicine. 

It was originally designated an orphan medicine for the following conditions:

Upon request of the marketing-authorisation holder, Pheburane has now been removed from the Community register of orphan medicinal products.