Overview

This medicine is now known as burosumab.

On 15 October 2014, orphan designation (EU/3/14/1351) was granted by the European Commission to NDA Group AB, Sweden, for recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 for the treatment of X-linked hypophosphataemia.

Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 for the treatment of X-linked hypophosphataemia has been authorised in the EU as Crysvita since 19 February 2018.

The sponsorship was transferred to Kyowa Kirin Holdings B.V., The Netherlands, in July 2018.

X-linked hypophosphataemia is a type of hereditary disorder characterised by low levels of phosphate in the blood (hypophosphatemia). Phosphate is a mineral essential to build bones and teeth and to maintain their strength. Phosphate levels are largely controlled by the kidneys, which eliminate excess phosphate in urine or reabsorb this mineral into the bloodstream when needed.

Patients with X-linked hypophosphataemia have high levels of a protein called fibroblast growth factor 23 (or FGF23). FGF23 signals the kidneys to stop reabsorbing phosphate into the bloodstream. If levels of FGF23 are high, the kidneys stop reabsorbing phosphate which is then eliminated from the body in the urine leading to low levels of phosphate. As a result, the disease causes delays in children's growth, bone pain and bone deformities (a disorder commonly known as 'rickets').

X-linked hypophosphataemia is a long-term debilitating condition due to bone deformities.

At the time of designation, X-linked hypophosphataemia affected between 0.002 and 0.04 in 10,000 people in the European Union (EU). This was equivalent to a total of between 100 to 2,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

At the time of designation, there were no satisfactory methods of treatment for X-linked hypophosphataemia in the EU. Patients with the disease were given phosphate by mouth to try to improve growth, and bone pain.

This medicine 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 is expected to 'neutralise' its activity leading to the kidneys being able to reabsorb phosphate and restore normal levels of phosphate in the blood. This is expected to improve the symptoms of the disease.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with X-linked hypophosphataemia were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for X-linked hypophosphatemia. Orphan designation of the medicine had been granted in the United States of America for X-linked hypophosphatemia.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 September 2014 recommending the granting of this designation.

  • the seriousness of the condition;
  • the existence of alternative methods of diagnosis, prevention or treatment;
  • either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/14/1351: Public summary of opinion on orphan designation: Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 for the treatment of X-linked hypophosphataemia

AdoptedReference Number: EMA/COMP/551233/2014

English (EN) (123.3 KB - PDF)

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Key facts

Active substance
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 (burosumab)
Medicine name
Crysvita
Intended use
Treatment of X-linked hypophosphataemia
Orphan designation status
Positive
EU designation number
EU/3/14/1351
Date of designation
Sponsor

Kyowa Kirin Holdings B.V.
Bloemlaan 2
2132NP Hoofddorp
The Netherlands
E-mail: medinfo@kyowakirin.com
The Netherlands

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Crysvita at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the Crysvita : Orphan maintenance assessment report (initial authorisation).

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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