Crysvita

burosumab

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

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

What is Crysvita and what is it used for?

Crysvita is a medicine used for the treatment of X-linked hypophosphataemia, a hereditary disorder characterised by low levels of phosphate in the blood (hypophosphataemia). Phosphate is essential to build bones and teeth and to maintain their strength, so patients may develop rickets and other bone deformities and growth problems.

Crysvita can be used in children over 1 year of age and adolescents with growing skeletons. It is used if signs of bone disease are seen on X-rays.

Crysvita contains the active substance burosumab.

Because the number of patients with X-linked hypophosphataemia is low, the disease is considered ‘rare’, and Crysvita was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 October 2014.

How is Crysvita used?

The medicine can only be obtained with a prescription and treatment should be started by a doctor experienced in the management of patients with bone diseases caused by alterations in the body’s chemical processes.

Crysvita is given as an injection under the skin. The recommended dose at the start of treatment is 0.4 mg per kg of body weight, and then 0.8 mg/kg every two weeks to continue treatment. The dose is adjusted according to the patient’s phosphate levels in the blood. The maximum dose is 90 mg every two weeks.

For further information, see the package leaflet.

How does Crysvita work?

Phosphate levels are largely controlled by the kidneys, which either remove excess phosphate or reabsorb it into the bloodstream when needed. Patients with X-linked hypophosphataemia have abnormally high levels of a protein called FGF23 which causes the kidneys to stop reabsorbing phosphate into the bloodstream.

Crysvita is a monoclonal antibody (a type of protein) designed to recognise and attach to the FGF23 protein. By attaching to the FGF23 protein, the medicine blocks its activity, allowing the kidneys to reabsorb phosphate and restore normal levels of phosphate in the blood.

What benefits of Crysvita have been shown in studies?

Crysvita reduced the severity of rickets as shown in X-rays in children with X-linked hypophosphataemia.

The medicine was assessed in one main ongoing study in 52 children aged between 5 and 12 years. All the children received Crysvita either every two weeks or every four weeks. The main measure of effectiveness was a reduction in the severity of rickets (bone deformities) in the wrist and knee measured on a scale from 0 (normal) to 10 (severe). The average score before treatment was 1.9 points in those given Crysvita every 2 weeks, and this fell by 1.0 point after 64 weeks of treatment; in those given the medicine every 4 weeks the baseline score of 1.7 fell by 0.8 point. In addition, phosphate levels in the blood improved over time in both groups, particularly those given Crysvita every 2 weeks.

An additional ongoing study in 13 younger children shows that Crysvita is effective in those aged between 1 and 4 years.

What are the risks associated with Crysvita?

Side effects with Crysvita (which may affect more than 1 in 10 people) are injection site reactions (such as skin redness, itching, rash, pain and bruising), headache, pain in arms and legs, decreased vitamin D level, rash, toothache, tooth abscess, muscle pain and dizziness.

Oral phosphate and vitamin D medicines must not be used during treatment with Crysvita. Also, Crysvita must not be used in patients with high phosphate levels in the blood or in patients with severe kidney disease.

For the full list of restrictions with Crysvita see the package leaflet.

Why is Crysvita approved?

Crysvita was shown to reduce the severity of bone deformities in the wrist and knee and improve the level of phosphate in the blood of children with X-linked hypophosphataemia in one ongoing study, and further data are awaited. Its side effects seem manageable. The European Medicines Agency therefore decided that Crysvita’s benefits are greater than its risks and recommended that it be approved for use in the EU.

Crysvita has been given ‘conditional approval’. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary.

What information is still awaited for Crysvita?

Since Crysvita has been granted a conditional approval, the company that markets Crysvita will provide updated results from two ongoing studies in children aged between 5 and 12 years and between 1 and 4 years. The company is also required to conduct and submit the results of a study comparing Crysvita with oral phosphate and vitamin D in children aged between 1 and 12 years with X-linked hypophosphataemia.

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

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Crysvita have been included in the summary of product characteristics and the package leaflet.

Other information about Crysvita

The European Commission granted a conditional marketing authorisation valid throughout the European Union for Crysvita on 19 February 2018.

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

Name Language First published Last updated
Crysvita : EPAR - Summary for the public BG = bălgarski 2018-03-12  
Crysvita : EPAR - Summary for the public ES = español 2018-03-12  
Crysvita : EPAR - Summary for the public CS = čeština 2018-03-12  
Crysvita : EPAR - Summary for the public DA = dansk 2018-03-12  
Crysvita : EPAR - Summary for the public DE = Deutsch 2018-03-12  
Crysvita : EPAR - Summary for the public ET = eesti keel 2018-03-12  
Crysvita : EPAR - Summary for the public EL = elliniká 2018-03-12  
Crysvita : EPAR - Summary for the public EN = English 2018-03-12  
Crysvita : EPAR - Summary for the public FR = français 2018-03-12  
Crysvita : EPAR - Summary for the public IT = italiano 2018-03-12  
Crysvita : EPAR - Summary for the public LV = latviešu valoda 2018-03-12  
Crysvita : EPAR - Summary for the public LT = lietuvių kalba 2018-03-12  
Crysvita : EPAR - Summary for the public HU = magyar 2018-03-12  
Crysvita : EPAR - Summary for the public MT = Malti 2018-03-12  
Crysvita : EPAR - Summary for the public NL = Nederlands 2018-03-12  
Crysvita : EPAR - Summary for the public PL = polski 2018-03-12  
Crysvita : EPAR - Summary for the public PT = português 2018-03-12  
Crysvita : EPAR - Summary for the public RO = română 2018-03-12  
Crysvita : EPAR - Summary for the public SK = slovenčina 2018-03-12  
Crysvita : EPAR - Summary for the public SL = slovenščina 2018-03-12  
Crysvita : EPAR - Summary for the public FI = suomi 2018-03-12  
Crysvita : EPAR - Summary for the public SV = svenska 2018-03-12  
Crysvita : EPAR - Summary for the public HR = Hrvatski 2018-03-12  

This EPAR was last updated on 12/03/2018 .

Authorisation details

Product details

Product details for Crysvita
NameCrysvita
Agency product numberEMEA/H/C/004275
Active substance

burosumab

International non-proprietary name (INN) or common name

burosumab

Therapeutic area Hypophosphatemia, FamilialHypophosphatemic Rickets, X-Linked Dominant
Anatomical therapeutic chemical (ATC) code M05BX05
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Conditional Approval

Sometimes, the CHMP recommends that a medicine be given ‘conditional approval’. This happens when the Committee has based its positive opinion on data which, while not yet comprehensive, indicate that the medicine’s benefits outweigh its risks.

The company is given obligations to fulfil, such as the performance of further studies. The approval is renewed on a yearly basis until all obligations have been fulfilled, and is then converted from a conditional approval into a normal approval. Conditional approvals can only be granted for medicines that satisfy an ‘unmet medical need’, meaning the medicine is intended to be used for a disease or condition for which no treatment is readily available, and it is therefore important that patients have early access to the medicine concerned.

Publication details

Publication details for Crysvita
Marketing-authorisation holder

Kyowa Kirin Limited

Revision0
Date of issue of marketing authorisation valid throughout the European Union19/02/2018

Contact address:

Kyowa Kirin Limited
Galabank Business Park 
Galashiels TD1 1QH 
United Kingdom

Product information

Product information

19/02/2018  Crysvita -EMEA/H/C/004275

Name Language First published Last updated
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Product Information HR = Hrvatski 2018-03-12  

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
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  
Crysvita : EPAR - All Authorised presentations HR = Hrvatski 2018-03-12  

Pharmacotherapeutic group

Drugs for treatment of bone diseases

Therapeutic indication

Treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated

Initial marketing-authorisation documents

Name Language First published Last updated
Crysvita : Orphan maintenance assessment report HR = Hrvatski 2018-03-12  
Crysvita : EPAR - Public assessment report HR = Hrvatski 2018-03-12  
CHMP summary of positive opinion for Crysvita HR = Hrvatski 2017-12-15  

Authorised

This medicine is approved for use in the European Union

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