mecasermin
Regulatory sources consulted
Approved indications
- Long-term treatment of growth failure in children and adolescents aged 2–18 years with confirmed severe primary IGF-1 deficiency.
Contraindications
Absolute
- Active or suspected neoplasia, or a condition/history increasing neoplasia risk; hypersensitivity; do not use in neonates or premature infants because of benzyl alcohol.
Clinical warnings
- Major warning · Insulin-like hypoglycemia risk. Dose around food intake, monitor glucose initially, and avoid high-risk activities for 2–3 hours; have glucagon available with a history of severe hypoglycemia. — CIMA/AEMPS, ficha técnica 07402001
- Major warning · There is a risk of benign and malignant neoplasms; do not use off-label or exceed the dose. Permanently stop if a neoplasm develops. — CIMA/AEMPS, ficha técnica 07402001
- Major warning · Perform funduscopic examination before and periodically during treatment because of intracranial-hypertension risk. Monitor tonsillar/adenoidal hypertrophy, snoring, apnea, and otitis due to lymphoid-tissue growth. — CIMA/AEMPS, ficha técnica 07402001
- Major warning · Evaluate limping or hip/knee pain for possible slipped capital femoral epiphysis or osteonecrosis, and monitor scoliosis progression during rapid growth. — CIMA/AEMPS, ficha técnica 07402001
Drug interactions
- HighInsulin or other glucose-lowering medicines
Mechanism: Hypoglycemic effects may be additive.
Recommendation: Glucose-lowering therapy may need reduction; monitor glucose closely.
CIMA/AEMPS, ficha técnica 07402001https://cima.aemps.es/cima/dochtml/ft/07402001/FT_07402001.html
Adverse events
Common (≥1%)
Hypoglycemia · Headache · Vomiting · Injection-site hypertrophy · Otitis media
Rare but serious
Benign or malignant neoplasia · Intracranial hypertension · Anaphylaxis or angioedema · Hypoglycemic seizure
Pregnancy and lactation
Require a negative pregnancy test and effective contraception. It should not be used during pregnancy, and breastfeeding is not recommended.
Recent literature (PubMed)
Mecasermin, a recombinant analogue of insulin‑like growth factor 1 (IGF‑1), is under investigation as a potential therapy for Rett syndrome (RTT), a neurodevelopmental disorder resulting from mutations in the MECP2 gene. In this systematic review, we assessed the impact of mecasermin on the full spectrum of RTT severity by screening relevant clinical studies identified through MeSH‑based database queries. Evidence indicates that IGF‑1 administration may help preserve social engagement and cognitive function in RTT, although autonomic control and behavioral outcomes have been inconsistent, and electroencephalographic alterations display considerable heterogeneity. Moreover, transcriptomic analyses have uncovered discrete gene‑expression signatures in molecularly defined patient subgroups, suggesting that genetic background modulates therapeutic response. These findings highlight the promise of mecasermin for ameliorating specific RTT features while underscoring the necessity of larger, rigorously designed trials to refine treatment regimens and implement stratified, patient‑specific approaches. CADTH recommends that Increlex should be reimbursed by public drug plans for the treatment of treatment of growth failure in children and adolescents from 2 to 18 years with confirmed severe primary insulin-like growth factor-1 deficiency (SPIGFD) if certain conditions are met. WHICH PATIENTS ARE ELIGIBLE FOR COVERAGE? Increlex should only be covered to treat patients who are at least 2 years of age with confirmed diagnosis of SPIGFD and in whom epiphyseal growth plates have not yet closed. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Increlex should only be reimbursed if prescribed by a pediatric endocrinologist, if it is not prescribed in combination with recombinant growth hormone treatment, and the price of Increlex is reduced. WHY DID CADTH MAKE THIS RECOMMENDATION? Evidence from 1 clinical trial demonstrated that Increlex increases height velocity in children with open epi
Autism spectrum disorder (ASD), a heterogeneous group of neurodevelopmental disorders, is characterized by social impairment and repetitive and stereotypic behaviors. Because of the lack of approved laboratory diagnostic markers and effective therapeutic medications, it is one of the most challenging diseases. Therefore, it is urgent to explore potential diagnosis markers or therapeutic targets. Insulin-like growth factor 1 (IGF-1) is a neurotrophic growth factor that enhances brain development. IGF-1 levels in body fluids are lower in preschool children with ASD than in typically developing children, which may serve as a potential diagnostic marker. In various ASD models associated with genetic or environmental exposure, IGF-1 treatment can improve core symptoms or pathological changes, including neuronal development, neural cell survival, balance of synaptic excitation and inhibition, neuroimmunology, and oxidative stress status. In March 2023 an IGF-1 derivative was approved as the first drug for treating Rett syndrome, an ASD-related neurodevelopmental disorder, to improve fundamental symptoms such as social communication. Thus, in this review, we present accumulating evidence of altered IGF-1 levels in ASD patients and the possible mechanisms, as well as evidence that IGF-1 treatment improves the pathophysiology in various ASD models. IGF-1 has the potential to be an early diagnosis marker and an effective therapeutic for ASD.