In November, the European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorization for Ascendis Pharma’s lonapegsomatropin (TransCon hGH), a long-acting once-weekly, transiently pegylated somatropin that in the body releases somatropin, indicated for growth failure in children and adolescents aged from 3 years...
Rapid uptake of long-acting growth hormones (GH) is set to shake up the growth hormone deficiency (GHD) space in the three major markets (3MM): the US, Germany, and Japan. These therapies are projected to capture more than 90% of market share in this region by 2030 due to their potential to improve patient convenience and...
New App Helps Parents Identify Treatable Childhood Growth Disorders Earlier
A mobile phone app can place the accurate measurement of children’s height in the hands of parents and caregivers. Preliminary data presented at the Society for Endocrinology annual conference in Edinburgh suggests that the app could reliably identify treatable growth disorders much earlier, with significant improvements in child health. Childhood growth is a strong indicator...
Baseline IGF-1 and Stimulated GH Can Predict Pediatric GHD Severity, Response to Treatment
Baseline insulin-like growth factor-1 (IGF-1) and stimulated peak growth hormone response can serve as predictors for how severe growth hormone deficiency is in pediatric patients as well as how these patients will respond to different treatments, according to a study recently published in the Journal of the Endocrine Society. Researchers led by Michael O. Thorner,...
The investigational long-acting, once-weekly prodrug lonapegsomatropin (TransCon hGH) may be more beneficial to treatment-naïve pediatric patients with growth hormone deficiency (GHD) than daily somatropin of equivalent weekly dose, according to a paper recently published in The Journal of Clinical Endocrinology & Metabolism. Researchers led by Aimee D. Shu, MD, of Ascendis Pharma (who funded the...
From ENDO2021: Phase 3 Trial Results Show Patient Preference for First Oral Therapy for Acromegaly
Data from a Phase 3 trial presented this week at ENDO2021 showed that octreotide capsules improved clinical symptoms and other patient-reported outcomes compared to long-acting injectable somatostatin receptor ligands (iSRLs) in patients with acromegaly. The data went on to show that 90% of patients receiving octreotide capsules elected to enroll in the open-label extension (OLE)...
Pharmaceutical Companies Team Up to Develop Acromegaly Treatment
Two pharmaceutical companies will collaborate to optimize and evaluate growth hormone receptor antagonists to select a candidate for clinical development as a potential treatment for acromegaly Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and metabolic diseases, and PeptiDream Inc., a public Kanagawa-based biopharmaceutical company, today announced a research collaboration...
Digenic Variants in Fibroblast Growth Factor 21 Signaling Linked to Severe Insulin Resistance
Variants in genes that mediate fibroblast growth factor 21 – a hormone secreted by the liver that acts to regulate the body’s response to starvation – have for the first time been linked to severe insulin resistance, according to a paper recently published in the Journal of the Endocrine Society. Researchers led by Stephen Stone,...
