First treatment recommended for rare progressive lung conditions in children and adolescents
Positive opinion for extension of indication from age of six
NewsHumanMedicines
EMA has recommended extending the indication of Ofev (nintedanib) to include the treatment of progressive fibrosing interstitial lung diseases (ILDs) in children and adolescents from the age of 6. Currently there are no approved therapies for these conditions in children.
Fibrosing ILDs encompass a group of complex and heterogeneous rare respiratory disorders that can be linked to many causes, including autoimmune conditions like systemic sclerosis. Fibrosing ILDs are characterised by the build-up of scarring in the lungs, which leads to breathing difficulties. Ofev is already authorised in the European Union for the treatment of fibrosing ILDs in adults.
The active substance in Ofev, nintedanib, blocks the activity of some enzymes known as tyrosine kinases. These enzymes are involved in the generation of scar tissue. By blocking these enzymes, nintedanib helps to reduce the formation of scarring in the lungs, preventing the symptoms of the disease from getting worse.
The medicine is available as capsules to be taken twice a day. A lower strength will be available for use in children. Considering the very small number of children with these conditions, which are estimated to affect less than 5 children per million in the EU, EMA’s human medicines committee (CHMP) based its recommendation to extend the use of Ofev on the results of a small paediatric clinical trial and extrapolation of data from adult trials. As only children whose conditions will progress and worsen should be treated with Ofev, the decision to start this treatment should involve a multidisciplinary team of specialists experienced in the diagnosis and treatment of fibrosing ILDs.
The safety of the medicine was studied in a randomised, double-blind, placebo-controlled, clinical trial involving 39 children and a subsequent trial following 33 of the same children and 15 new participants. Participants were followed for up to 124 weeks.
The safety profile of Ofev in children was comparable to that seen in adults. However, due to its mechanism of action, nintedanib may have longer-term effects on growth and tooth development in children and adolescents that are less relevant in adults. Paediatric patients should therefore be monitored for these potential effects through regular bone imaging and dental examinations while receiving treatment with Ofev. These potential effects are also to be further characterised through additional data collection post authorisation.
The opinion adopted by the CHMP is an intermediary step on Ofev’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide extension of the therapeutic indication. Once an extension has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.
Notes:
- The marketing authorisation holder for Ofev is Boehringer Ingelheim International GmbH.