EU/3/21/2475 - orphan designation for treatment of achondroplasia
Infigratinib
Orphan
Human
This medicine was designated as an orphan medicine for the treatment of achondroplasia in the European Union on 19 July 2021.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
Infigratinib belongs to a group of medicines called protein kinase inhibitors. It works by blocking enzymes known as protein kinases, particularly those that are part of receptors (targets) called fibroblast growth factor receptors (FGFRs). FGFRs are found on the surface of bone cells and are involved in the growth of bone cells. By blocking the tyrosine kinases in overactive FGFRs in patients with achondroplasia, infigratinib is expected to reduce the activity of FGFR3. This is expected to stimulate growth of bones, thereby improving the symptoms of the disease.
Based on description provided by sponsor
At the time of submission of the application for orphan designation:
More information on how potential new medicines are tested during their development is available on Authorisation of medicines.
Medicines intended for rare diseases can be granted an orphan designation during their development.
The orphan designation allows the developer to benefit from:
To qualify for orphan designation, a medicine must meet a number of criteria:
EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.
The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
For more information, see:
YES Pharmaceutical Development Services GmbH
Basler Strasse 7
Gonzenheim
61352 Bad Homburg
Hesse
Germany
E-mail: karl-heinz.loebel@pharmalex.com
Date | Update |
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August 2023 | The sponsor’s address was updated in August 2023. |
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: