Orphan designation: Infigratinib Treatment of achondroplasia, 19/07/2021 Positive

Overview

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: EU Clinical Trials Register ClinicalTrials.gov Expand section Collapse section How the medicine is expected to work 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 Stage of development at time of orphan designation At the time of submission of the application for orphan designation: The effects of the medicine had been evaluated in experimental models. Clinical trials with the medicine in patients with achondroplasia were ongoing. More information on how potential new medicines are tested during their development is available on Authorisation of medicines . About orphan designation Medicines intended for rare diseases can be granted an orphan designation during their development. The orphan designation allows the developer to benefit from: scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation; market exclusivity once the medicine is on the market. To qualify for orphan designation, a medicine must meet a number of criteria: it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development; there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or the medicine is of significant benefit to those affected by the condition. 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: Orphan designation: Overview Rare diseases, orphan medicines - Getting the facts straight Minutes of the COMP meeting 15-17 June 2021 Adopted Reference Number: EMA/COMP/359170/2021 English (EN) (551.28 KB - PDF) First published: 21/09/2021 View

Key facts

Active substance Infigratinib Intended use Treatment of achondroplasia Orphan designation status Positive EU designation number EU/3/21/2475 Date of designation 19/07/2021 Sponsor BridgeBio Pharma Europe B.V.

Update history

Date Update June 2026 The sponsorship was transferred to BridgeBio Pharma Europe B.V. August 2023 The sponsor's address was updated in August 2023.