In October 2020 the European Medicines Agency (EMA) expressed positive opinion on the adoption and extension of indication for a number of treatments addressed to the paediatric population.
This is the case of Dupixent (dupilumab), for which on 15 October 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted an extension to the existing indication (dating back to 2002 and foreseeing an indication for adults and adolescents 12 years and older). CHMP released a positive opinion for the use of Dupixent also in children 6 to 11 years of age for the treatment of severe atopic dermatitis.
It is possible to read the Summary of CHMP opinion at this link.
A positive opinion recommending a change to the terms of the marketing authorisation has been released also for the medicine Humira (adalimumab). According to the existing indication (dating back to 1999), this medicinal product can be used for the treatment of several conditions in adults and children (rheumatoid arthritis, juvenile idiopathic arthritis, enthesitis-related arthritis, axial spondyloarthritis, Psoriatic arthritis, Psoriasis, Paediatric plaque psoriasis, Crohn’s disease, etc.). Moreover, with the new indication Humira is indicated also for the treatment of moderately to severely active ulcerative colitis in paediatric patients (from 6 years of age) who have had an inadequate response to conventional therapy. The Summary of CHMP opinion is available here.
Finally, CHMP has recommended granting a marketing authorisation also for two new paediatric treatments:
- Palforzia, a medicinal product by Aimmune Therapeutics Ireland Limited, indicated for the treatment of patients aged 4 to 17 years with a confirmed diagnosis of peanut allergy. Palforzia may be continued in patients 18 years of age and older. More information is available at this link.
- Libmeldy, the new gene therapy to treat children with metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is a gene therapy medicinal product, for which CD34+ haematopoietic stem and progenitor cells are collected either from the patient’s own bone marrow or mobilised peripheral blood. These cells are modified to insert a functional gene to produce the ARSA enzyme. When the modified cells are injected back into the patient as a one-time infusion, the cells are expected to start producing the ARSA enzyme that breaks down the build-up of sulfatides in the nerve cells and other cells of the patient’s body.
