EU/3/22/2697 - orphan designation for treatment of apolipoprotein L1-mediated kidney disease

Inaxaplin
OrphanHuman

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Overview

This medicine was designated as an orphan medicine for the treatment of apolipoprotein L1-mediated kidney disease in the European Union on 11 October 2022.

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:

This medicine belongs to the class of apolipoprotein L1 inhibitors.

Apolipoprotein L1-mediated kidney disease (AMKD) develops in individuals with G1 and G2 mutations to the APOL1 gene, which leads to errors in the APOL1 protein, which creates pores in membranes of certain kidney cells in patients with AMKD, leading to cell injury and death. The subsequent loss in kidney function results in a high level of proteins in urines (proteinuria) and worsening to end stage kidney disease.

Inaxaplin is an inhibitor of the apolipoprotein L1 protein (APOL1). By inhibiting APOL1 function in patients with AMKD, Inaxaplin is expected to reduce proteinuria and slow or stop the decline in kidney function, decrease the risk of end stage kidney disease and associated morbidity, hospitalisation and mortality.

Based on description provided by sponsor
 

At the time of submission of the application for orphan designation:

  • The effects of the medicine had been evaluated in experimental models.
  • No clinical trials with the medicine in patients with sickle cell disease had been started. 

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:

  • 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, if such a method exists, 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:

Minutes of the COMP meeting 6-8 September 2022

Adopted Reference Number: EMA/COMP/772376/2022

English (EN) (566.63 KB - PDF)

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Key facts

Active substance
Inaxaplin
Intended use
Treatment of apolipoprotein L1-mediated kidney disease
Orphan designation status
Positive
EU designation number
EU/3/22/2697
Date of designation
Sponsor

Vertex Pharmaceuticals (Ireland) Limited

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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