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This medicine was designated as an orphan medicine for the treatment of mucopolysaccharidosis type II (Hunter's syndrome) in the European Union on 20 August 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:
Patients with mucopolysaccharidosis type II (also known as Hunter’s syndrome) have mutations in the gene for the enzyme iduronate-2-sulfatase (IDS), which is responsible for breaking down and recycling complex glycosaminoglycans (GAGs).
The medicine consists of blood stem cells taken from the patient and modified using a virus to contain the gene for the missing IDS enzyme. When the stem cells are given back to the patient, these cells will produce the missing enzyme and so can break down GAGs, thereby helping to relieve symptoms of the disease.
The virus used to produce this medicine has been designed so that it cannot spread or cause disease in humans.
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 mucopolysaccharidosis type II (Hunter's syndrome) 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:
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:
University of Padua
Via Nicolo' Giustiniani 3
35128 Padova (PD)
Italy
Tel. +39 0498218030
E-mail: alessandra.biffi@unipd.it
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: