When The Medicine Maker first launched, I remember researching an eye-opening feature on pediatric drugs – or the lack thereof (you can read the issue here). Today, there is still a lack of drugs for children (especially biologics), but there are certainly more approvals coming through. So I was pleased to read about Regeneron’s Eylea (aflibercept) receiving FDA approval to treat the smallest patients with retinopathy of prematurity – a leading cause of childhood blindness worldwide. Previously, the only FDA-approved treatment was laser photocoagulation, which can be a stressful procedure for all patients – and children even more so.
Another recent biologic success for children is Takeda’s Takhzyro (lanadelumab-flyo), which now has FDA approval to prevent hereditary angioedema attacks in children over the age of two years old.
There’s been a great deal of focus during the last year from the FDA on pediatric medicines. In September, the agency outlined an ethical framework for including children in clinical trials. Also last year, the agency issued draft guidance on extrapolating data to accelerate drug development for children.
There is also activity in Europe, with the EMA last week summarizing action that has been taken to help support the development of medicines for children.
It’s fantastic to finally see progress in this important area. If you want to tell me about pediatric programs at your company, please let me know.
Until next time,
Stephanie Sutton, Editor, The Medicine Maker
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