Independent analysis of children’s medicines for pharma and biotech. About NextGenHealth
The science, policy and innovation shaping children’s healthcare.Read the latest
Treatment for children with tics has long relied on behavioural therapy and antipsychotics. Teva's ecopipam, under priority review at the FDA, would add a different mechanism, on evidence that needs careful reading.
Treatments
Deramiocel lost an advisory committee vote in July on its cardiac case. Capricor now pins its hopes on upper limb function, with an FDA target date of 22 November and a dispute over the statistics unresolved.
Medicines
The FDA has cleared a generic version of Firdapse, the Lambert-Eaton treatment labelled from age six. A patent settlement keeps it off the US market for more than eight years.
Medicines
The FDA has extended BioCryst's Orladeyo oral pellets to patients aged 12 and over, ten months after approving the formulation for younger children. It reverses the usual order of paediatric development.
Evidence
Lilly says half of the children who responded to induction with Omvoh were in clinical remission at one year. The SHINE-2 study had no control arm, so its result rests on a comparison with adult data.
Safety
A missing QT warning, particles in a medicine that protects children on cisplatin from hearing loss, an under-strength unlicensed liquid and a ketone strip shortage all touched paediatric care this month. None was a new pharmacological signal, and most were about product quality and information.
Medicines
The EMA has upheld its negative opinion on Omeros’s Yartemlea for transplant-associated thrombotic microangiopathy in adults and children from two years of age. The FDA approved the same drug for the same population in December 2025.
Policy
Two sales by Ultragenyx in a single week and a run of new FDA awards show how much the rare pediatric disease voucher is worth since Congress revived the programme in February.
Evidence
Rafael Holdings has asked the FDA to approve its cyclodextrin therapy for patients from birth. The phase 3 trial missed its main goal, so the case rests on a subgroup and a survival comparison with registry patients.
Medicines
The FDA has cleared a generic version of Firdapse, the Lambert-Eaton treatment labelled from age six. A patent settlement keeps it off the US market for more than eight years.
Medicines
The FDA has extended BioCryst's Orladeyo oral pellets to patients aged 12 and over, ten months after approving the formulation for younger children. It reverses the usual order of paediatric development.
Medicines
The EMA has upheld its negative opinion on Omeros’s Yartemlea for transplant-associated thrombotic microangiopathy in adults and children from two years of age. The FDA approved the same drug for the same population in December 2025.
Medicines
Orphelia Pharma's Kizfizo, an oral suspension of an old chemotherapy, has a positive CHMP opinion for malignant glioma from the age of one. Its first application, in neuroblastoma, was turned down last year.
Medicines
Europe's medicines committee has recommended Ascendis Pharma's once-weekly navepegritide for children with achondroplasia. If the European Commission agrees, it will compete with BioMarin's daily vosoritide.
Medicines
Novo Nordisk's once-weekly basal insulin can now be prescribed in the US to children aged 10 and over with type 2 diabetes. The paediatric evidence rests on adult trials and a single-dose study in 18 young people.
Medicines
Full phase 2 results presented at IDWeek show Pfizer’s next infant pneumococcal vaccine outperforming Prevnar 20 on serotype 3. The commercial stakes in the paediatric market are large, and phase 3 is already under way.
Medicines
A tablet that suits a 70kg adult is often useless for a toddler. Getting the dose, the taste and the ingredients right is a development programme in its own right.
Medicines
The liquid medicine is losing its monopoly. Flexible solid formats promise better stability, more accurate dosing and lower costs, especially outside rich countries.
Medicines
Children are not small adults, and dosing them by simple arithmetic from adult doses can go badly wrong. The science of getting it right starts with how the body matures.
Medicines
Preservatives, solvents and sweeteners rarely make headlines. In the youngest patients, they can matter as much as the active drug.
Medicines
The winter of 2022 exposed how thin the supply of basic paediatric antibiotics had become. The structural reasons have not gone away.
Medicines
Biosimilars can win approval for paediatric uses without paediatric trials. The bigger practical issue is often whether the right dose and device exist for a small child.
Treatments
Deramiocel lost an advisory committee vote in July on its cardiac case. Capricor now pins its hopes on upper limb function, with an FDA target date of 22 November and a dispute over the statistics unresolved.
Treatments
A month after US approval for patients aged 12 and over, Mirum Pharmaceuticals has reported that its oral ALK2 inhibitor slowed abnormal bone growth in children aged six to 11 with fibrodysplasia ossificans progressiva.
Treatments
Scholar Rock’s Isembyld is the first SMA therapy aimed at muscle rather than motor neurons. Its US approval came with a priority review voucher, and a European filing is back with the EMA.
Treatments
Many of the conditions that one-time genetic treatments target first appear in infancy. That puts children at the centre of the field’s scientific, commercial and ethical debates.
Treatments
The first CAR-T therapy approved anywhere was for children and young adults with leukaemia. That history still shapes how the field thinks about paediatric patients.
Treatments
In less than four years, a disease with no treatment gained three, each using a different technology. SMA has become a case study in how modality shapes access.
Treatments
Drugs that correct the underlying protein defect have changed cystic fibrosis. The next question is how early in life they should start.
Treatments
The first approved CRISPR-based medicine treats sickle cell disease from age 12. Extending it to younger children raises questions about conditioning, fertility and timing.
Treatments
Doctors have begun designing genetic medicines for one patient at a time. The science is moving faster than the regulatory and funding models built around it.
Evidence
Lilly says half of the children who responded to induction with Omvoh were in clinical remission at one year. The SHINE-2 study had no control arm, so its result rests on a comparison with adult data.
Evidence
Rafael Holdings has asked the FDA to approve its cyclodextrin therapy for patients from birth. The phase 3 trial missed its main goal, so the case rests on a subgroup and a survival comparison with registry patients.
Evidence
Idorsia has presented full phase 2 results for its orexin receptor antagonist in children and adolescents with insomnia. The trial was short and the effect modest, but it is a rare controlled study in a field that relies on off-label prescribing.
Evidence
The EMA has declined to extend gemtuzumab ozogamicin to children with newly diagnosed acute myeloid leukaemia, a use the FDA approved in 2020. The reasoning is a lesson in paediatric trial design.
Evidence
Regulators increasingly accept that adult efficacy data can carry over to children when the disease and the drug’s effect are similar enough. The argument is about how much.
Evidence
Small populations, cautious parents and dozens of trials chasing the same patients make recruitment the most common reason paediatric programmes slip.
Evidence
Health records, registries and claims data contain years of experience with medicines in children. Turning that into regulatory-grade evidence is harder than it looks.
Evidence
Physiologically based pharmacokinetic models simulate how a drug moves through a growing body. Regulators now see them in a large share of paediatric submissions.
Evidence
Children cannot fully consent, so research rules ask more of the people designing paediatric trials. Placebo arms are where those rules bite hardest.
Evidence
The babies in neonatal intensive care receive some of the most intensive drug treatment in medicine. Very little of it was ever tested in them.
Safety
A missing QT warning, particles in a medicine that protects children on cisplatin from hearing loss, an under-strength unlicensed liquid and a ketone strip shortage all touched paediatric care this month. None was a new pharmacological signal, and most were about product quality and information.
Safety
Rare adverse events rarely show up in trials of a few hundred children. Post-marketing surveillance has to do the heavy lifting.
Safety
A painkiller given to children for decades turned out to be dangerous for a genetically identifiable minority. The response remains a model for pharmacogenomic safety.
Safety
A medicine given to a child can affect growth, development and fertility years after the last dose. Trials end long before those effects would show.
Safety
Weight-based dosing, liquid formulations and adult-sized products combine to make children unusually vulnerable to medication errors.
Safety
The label is where paediatric evidence becomes usable at the bedside. US and EU rules now require results to reach it, even when the studies fail.
Safety
Second-generation antipsychotics are increasingly prescribed to children and adolescents. Their effects on weight and metabolism appear to be greater than in adults.
Conditions
Treatment for children with tics has long relied on behavioural therapy and antipsychotics. Teva's ecopipam, under priority review at the FDA, would add a different mechanism, on evidence that needs careful reading.
Conditions
Many rare conditions begin in childhood, so orphan drug incentives and paediatric rules increasingly pull in the same direction.
Conditions
Childhood cancers are biologically distinct from adult ones, and for years that let new cancer drugs bypass children entirely. The RACE for Children Act changed the rules.
Conditions
For decades, protection against respiratory syncytial virus was reserved for the highest-risk babies. Long-acting antibodies and maternal vaccines have changed that.
Conditions
Automated insulin delivery has transformed daily management. The bigger shift may be the first drug that delays type 1 diabetes before it starts.
Conditions
The drugs reshaping adult obesity care are approved for teenagers too. Their arrival has forced a rethink of how childhood obesity is treated, and raised questions trials cannot yet answer.
Conditions
No paediatric disease has tested the FDA’s accelerated approval pathway like Duchenne. The arguments over dystrophin, function and risk are far from settled.
Policy
Two sales by Ultragenyx in a single week and a run of new FDA awards show how much the rare pediatric disease voucher is worth since Congress revived the programme in February.
Policy
Two decades of incentives and mandates on both sides of the Atlantic have transformed paediatric drug development. They have not closed the gap.
Policy
Brussels’ overhaul of its medicines legislation folds the Paediatric Regulation into a single framework. The details will decide whether plans for children start earlier.
Policy
Since 2021 the MHRA has run its own paediatric regime for Great Britain. For most companies it means one more plan to agree, not a different philosophy.
Policy
A tradable right to faster FDA review has become one of the more valuable prizes in paediatric drug development. Whether it creates new medicines is still debated.
Policy
Health technology assessment was built around adult trials and adult lives. Children’s medicines strain almost every assumption it relies on.
Policy
Doctors routinely give children medicines that were never licensed for them. It is often the best option available, and that is precisely the problem.
