Boehringer Ingelheim has won a positive European Medicines Agency (EMA) recommendation for blockbuster Ofev’s (nintedanib) use in children and adolescents with progressive fibrosing interstitial lung diseases (ILDs), after missing an expanded approval in the US earlier this year.
The positive opinion could lead to the first approval of a therapy for these conditions in children.
The recommendation is for the approval of Ofev in children and adolescents from six to 17 years old for the treatment of clinically significant, progressive fibrosing ILDs and in adolescents and children aged six years and older for the treatment of systemic sclerosis-associated interstitial lung disease (SSc-ILD), as per a 13 December press release.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) will now send its recommendation to the European Commission (EC) who will make the final decision on whether Ofev’s approval is extended.
Fibrosing ILDs comprise a group of complex and rare respiratory disorders that cause the lungs to become scarred. There are a range of environmental and genetic factors that can contribute to the development of one of the diseases, idiopathic pulmonary fibrosis (IPF) being the most common type.
Ofev works by blocking tyrosine kinases involved in the generation of scar tissue. These enzymes, present in VEGF, FGF and PDGF receptors in the lungs, normally activate several processes involved in the development and progression of fibrous tissue.
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Boehringer Ingelheim’s kinase inhibitor is approved for adults in Europe with chronic fibrosing ILDs which are progressive, IPF and systemic sclerosis associated with ILD.
Chronic fibrosing ILDs also make up the US Food and Drug Administration (FDA) label for Ofev, but a paediatric expansion was rejected by the agency in March this year. The drug generated global sales of $3.8bn for the German drugmaker in 2023, up 12.8% from the year before.
The EMA’s CHMP based its positive decision on a small paediatric clinical trial, along with data extrapolation from adult trials. The Phase III trial, called InPedILD (NCT05285982), was powered for safety and showed a weight-based dosing regimen that resulted in comparable exposure seen in adult patients.
The CHMP concurred that the safety profile was comparable in children to adults, though highlighted the possibilities of longer-term effects on growth and tooth development, which are more relevant in children and teenagers. Those taking the medication in the newly approved group will therefore be monitored for these potential effects through regular bone imaging and dental examinations.
The EMA also stated a reduced strength of the drug will be given to children due to its side effects, which include liver problems, bleeding problems, and nausea and diarrhoea, amongst others.
“Children battling these rare conditions are often overlooked within clinical trials due to the complexities of paediatric dosing and additional steps in the participant approval process, leaving a critical gap in our understanding and treatment options,” said Martin Beck, head of therapeutic area inflammation at Boehringer Ingelheim.
“The CHMP recommendation is a significant step forward in our commitment to addressing the unmet needs of all those affected by pulmonary fibrosis.”
