EU/3/22/2638 - orphan designation for treatment of methylmalonic acidaemia

modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671
OrphanHuman

Overview

This medicine was designated as an orphan medicine for the treatment of methylmalonic acidaemia in the European Union on 21 June 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:

Isolated methylmalonic acidemia is a serious, life-threatening inherited disorder in which the body cannot properly break down certain proteins and fats. It is caused by changes (mutations) in the gene that encodes an enzyme called methylmalonyl-CoA mutase (MUT), which plays a role in the breakdown of certain protein building blocks (amino acids) and fats. This medicine, also known as mRNA-3705, contains messenger RNA (mRNA; genetic material with instructions for producing a protein) that encodes a working version of MUT. In patients with MMA due to MUT deficiency, the medicine is anticipated to help the body produce a working version of the MUT enzyme in liver cells, allowing the proper breakdown of protein and fat molecules and thereby improving symptoms of the disease.

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.
  • Clinical trials with the medicine in patients with methylmalonic acidaemia were ongoing.

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:

Key facts

Active substance
modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671
Intended use
Treatment of methylmalonic acidaemia
Orphan designation status
Positive
EU designation number
EU/3/22/2638
Date of designation
Sponsor

Moderna Biotech Spain S.L.
Calle Del Principe De Vergara 132 Plt 12
28002 Madrid
Spain
Email: Luke.MirceaWillats@modernatx.com

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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