Boehringer Ingelheim has received a positive recommendation from the European Medicines Agency (EMA) for Ofev (nintedanib) to treat progressive fibrosing interstitial lung diseases (ILDs) in children and adolescents. This recommendation could lead to the first approved therapy for these conditions in the pediatric population. The EMA's Committee for Medicinal Products for Human Use (CHMP) will forward its recommendation to the European Commission (EC) for a final decision.
The recommendation covers children and adolescents aged 6 to 17 years with clinically significant, progressive fibrosing ILDs, and those aged six years and older with systemic sclerosis-associated interstitial lung disease (SSc-ILD). Ofev is already approved for adults in Europe with chronic fibrosing ILDs, idiopathic pulmonary fibrosis (IPF), and SSc-ILD.
Mechanism of Action
Ofev functions by inhibiting tyrosine kinases involved in the formation of scar tissue. Specifically, it targets enzymes present in VEGF, FGF, and PDGF receptors in the lungs, which are crucial in the development and progression of fibrous tissue. By blocking these kinases, Ofev aims to reduce the scarring associated with ILDs.
Clinical Trial Data
The CHMP's positive opinion was informed by data from the Phase III InPedILD trial (NCT05285982), a study powered for safety. The trial demonstrated that a weight-based dosing regimen of nintedanib in children resulted in drug exposure comparable to that observed in adult patients. The CHMP also noted that the safety profile in children was similar to that in adults, although it emphasized the potential for longer-term effects on growth and tooth development, necessitating regular monitoring through bone imaging and dental examinations.
Safety Considerations
The EMA has specified that a reduced strength of Ofev will be administered to children to mitigate side effects, including liver problems, bleeding issues, nausea, and diarrhea. These potential adverse effects will be closely monitored in the newly approved patient group.
Addressing Unmet Needs
According to Martin Beck, head of therapeutic area inflammation at Boehringer Ingelheim, children with rare conditions are often underrepresented in clinical trials due to complexities in pediatric dosing and participant approval. He stated that the CHMP recommendation is a significant step forward in addressing the unmet needs of individuals affected by pulmonary fibrosis.
Ofev generated global sales of $3.8 billion for Boehringer Ingelheim in 2023, representing a 12.8% increase from the previous year. While the FDA rejected a pediatric expansion for Ofev in the US earlier this year, the EMA's positive recommendation marks a significant milestone for the drug's potential use in treating young patients with progressive fibrosing ILDs.
