Paediatrics

The first patient has been dosed in a Phase III study evaluating a paediatric granulated formulation of leniolisib in children with activated phosphoinositide 3-kinase delta syndrome (APDS). Pharming’s investigational drug leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. At sites in the US, Japan, and the EU, the clinical trial will evaluate the […]

A label expansion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) has been approved in the European Union (EU). The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in […]

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The UK Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC), and Health Research Authority (HRA) have all approved the clinical trial application that was submitted by The University of Manchester in August 2022. In […]

The US Food and Drug Administration’s (FDA) Research to Accelerate Cures and Equity (RACE) for Children Act now requires all oncology drugs to be tested for paediatric indications ahead of approval. The EU regulation of the European Medicines Agency (EMA) is also being adapted to mirror the FDA’s regulations. In response, Charles River Laboratories is […]

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases. Stabilising calcium levels in the brain could therefore be a target for […]

The European Commission (EC) has granted marketing authorisation for Jazz Pharmaceuticals’ Enrylaze as part of a multi-agent regimen for acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LBL). The treatment is approved in adult and paediatric patients who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. Enrylaze, approved as Rylaze in the United States and […]

DDW’s Diana Spencer highlights five key clinical studies taking a new therapeutic approach to childhood cancers. Over 100 subtypes of childhood cancer exist today and it represents the most common cause of disease-related death in children. However, despite the fact that children’s response to adult cancer drugs is often poor, due to the challenges of […]

Talus Bio has received $4.3 million in new grants from the National Institutes for Health (NIH) and the Washington State Andy Hill CARE Fund to advance its paediatric cancer therapeutics. The Seattle-based company received two grants totalling $2.3M from the CARE Fund to fuel the discovery of new transcription factor inhibitors for rhabdomyosarcoma and neuroblastoma, […]

A new respiratory syncytial virus (RSV) vaccine candidate has achieved positive safety and immunogenicity results in a Phase I clinical study of RSV-naïve participants between the ages of six and 36 months. MV-012-968 is being developed by Meissa Vaccines as a needle-free, adjuvant-free vaccine to protect infants and toddlers from RSV. The study enrolled 79 […]

Justin Hay, Senior Director, Amy Cheung Senior Director of Integrated Drug Development, and Patrick Loebs Director, at Certara, examine recent changes in paediatric guidelines and how they affect drug development projects.   It has been a very busy period recently for paediatric drug development with the release of many guidance documents from two notable regulatory agencies. […]

What are benefits of drug repurposing and why do clinical drug trials so often fail? Diana Spencer summarises the key messages from the ‘Drug Repurposing: Challenges and Opportunities’ Symposium held at the 19th World Congress of Basic & Clinical Pharmacology (WCP) 2023. The Drug Repurposing symposium at WCP 2023 was chaired by Oscar Della Pasqua, Chair […]

The US Food and Drug Administration (FDA) has approved AstraZeneca’s Beyfortus (nirsevimab-alip) for the prevention of Respiratory Syncytial Virus (RSV) lower respiratory tract disease in neonates and infants born during or entering their first RSV season. The drug is also approved in children up to 24 months of age who remain vulnerable to severe RSV […]

CureSearch for Children’s Cancer has awarded a $1.5 million grant to Dr Patrick Grohar of Children’s Hospital of Philadelphia to develop a new targeted treatment approach for paediatric osteosarcoma, an aggressive form of bone cancer.    The award – the Fight Osteosarcoma Together Super Grant – is a joint funding award with St Baldrick’s Foundation, CureSearch, […]

Bloom Science, a clinical-stage, central nervous system (CNS) company focused on discovering and developing breakthrough therapeutics that target the Gut-Brain Axis, has completed enrolment in all four cohorts of healthy adult participants as part of its Phase I clinical trial.   Bloom is developing BL-001 for Dravet syndrome, a rare paediatric epilepsy that significantly impacts […]

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SiSaf’s small interfering RNA (siRNA) therapeutic for skeletal disorder Autosomal Dominant Osteopetrosis Type 2 (ADO2). In addition, due to the serious manifestations of this rare disease in children, SIS-101-ADO has been granted Rare Pediatric Disease Designation for the treatment of Autosomal Dominant […]

A combination of the PARP inhibitor olaparib (Lynparza) and the investigational ATR inhibitor ceralasertib showed clinical benefit in paediatric patients with solid tumours exhibiting DNA replication stress and/or DNA repair deficiencies, according to new data. The results from the Phase I portion of the Phase I/II AcSé-ESMART trial were presented at the AACR Annual Meeting […]