Article
Endocrinology & Metabolism
Lars Savendahl, Tadej Battelino, Michael Hojby Rasmussen, Meryl Brod, Paul Saenger, Reiko Horikawa
Summary: This study evaluated the efficacy, safety, and tolerability of once-weekly somapacitan treatment for GH deficiency in children over a three-year period. The results showed sustained efficacy in height-related outcomes with similar safety and tolerability to daily GH treatment.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2022)
Article
Endocrinology & Metabolism
Lars Savendahl, Tadej Battelino, Michael Hojby Rasmussen, Meryl Brod, Sebastian Rohrich, Paul Saenger, Reiko Horikawa
Summary: This study aimed to assess the efficacy and safety of somapacitan in treating growth hormone deficiency (GHD) in children, as well as the burden of treatment. The results showed that weekly injections of somapacitan could reduce the burden for patients and their parents/guardians compared to daily GH injections.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2023)
Article
Pharmacology & Pharmacy
Ruoqian Cheng, Qian Zhao, Guoping Zhong, Jiong Xu, Zhangqian Zheng, Li Xi, Miaoying Zhang, Jinwen Ni, Pei Hu, Feihong Luo, Wei Lu
Summary: This study established a PopPK/PD model and found that longer intervals or higher doses (e.g., 0.3 mg/kg/10 days) of PEG-rhGH may be promising for treating children with growth hormone deficiency, beyond the approved 0.2 mg/kg/week dose.
EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
(2022)
Article
Endocrinology & Metabolism
Bradley S. Miller, Joanne C. Blair, Michael Hojby Rasmussen, Aristides Maniatis, Rasmus Juul Kildemoes, Jun Mori, Michel Polak, Rikke Beck Bang, Volker Boettcher, Stefano Stagi, Reiko Horikawa
Summary: Similar efficacy and safety were demonstrated for Somapacitan compared to daily GH over 52 weeks of treatment in children with GH deficiency.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2022)
Article
Endocrinology & Metabolism
Bradley S. Miller, Joanne C. Blair, Michael Hojby Rasmussen, Aristides Maniatis, Jun Mori, Volker Boettcher, Ho-Seong Kim, Rikke Beck Bang, Michel Polak, Reiko Horikawa
Summary: This study evaluated the efficacy and tolerability of somapacitan in children with growth hormone deficiency (GHD) for 2 years and after switching from daily growth hormone treatment. The results showed that somapacitan was effective and well-tolerated during the 2-year period, and patients and caregivers preferred once-weekly somapacitan treatment.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2023)
Article
Endocrinology & Metabolism
Yan Liang, Cai Zhang, Haiyan Wei, Hongwei Du, Gaixiu Zhang, Yu Yang, Hua Zhang, Haihong Gong, Pin Li, Fuying Song, Zhuangjian Xu, Ruoyi He, Weidong Zhou, Heng Zheng, Li Sun, Xiaoping Luo
Summary: This study investigated the pharmacokinetics and pharmacodynamics of Y-shape branched PEGylated recombinant human growth hormone (YPEG-rhGH) in children with growth hormone deficiency (GHD). The results suggest that YPEG-rhGH is suitable for once-weekly treatment, and a dose of 120-140 mu g/kg/week provides similar efficacy and safety compared to daily rhGH 35 mu g/kg/day.
FRONTIERS IN ENDOCRINOLOGY
(2022)
Article
Medicine, General & Internal
Ichiro Abe, Kaori Takeshita, Mai Nagata, Yuya Fujita, Kentaro Ochi, Midori Koga, Tadachika Kudo, Hideaki Shimada, Makiko Abe, Shigeaki Mukoubara, Kunihisa Kobayashi
Summary: This real-world pilot study evaluated the metabolic and endocrinological effects of switching from daily growth hormone (GH) replacement therapy to weekly GH replacement therapy in patients with adult growth hormone deficiency (AGHD). The results suggest that weekly GH replacement therapy may have more benefits compared to daily therapy.
Review
Endocrinology & Metabolism
Kevin C. J. Yuen, Bradley S. Miller, Cesar L. Boguszewski, Andrew R. Hoffman
Summary: Long-acting Growth Hormone (LAGH) analogs have been developed to reduce injection frequency and improve patient acceptance and therapeutic flexibility. However, potential clinical issues such as unphysiological GH profile, differing molecular structures, dose initiation, therapeutic monitoring, side-effect incidence, and long-term safety may arise with these analogs.
FRONTIERS IN ENDOCRINOLOGY
(2021)
Article
Endocrinology & Metabolism
Yutaka Takahashi, Beverly M. K. Biller, Hidenori Fukuoka, Ken K. Y. Ho, Michael Hojby Rasmussen, Navid Nedjatian, Claus Svaerke, Kevin C. J. Yuen, Gudmundur Johannsson
Summary: This study investigated the impact of long-acting growth hormone somapacitan on glucose metabolism in adult growth hormone deficiency patients. The results showed that somapacitan did not adversely affect glucose metabolism compared to daily growth hormone over 86 weeks.
Review
Infectious Diseases
Gauri G. Rao, Cornelia B. Landersdorfer
Summary: The use of pharmacokinetic/pharmacodynamic approaches is crucial in optimizing dosage regimens of anti-infectives to maximize efficacy, minimize toxicity and resistance, and ensure a high likelihood of therapeutic success. This involves well-designed preclinical studies, mathematical-model-based analysis, in-silico simulations, and the integration with population pharmacokinetic modelling to consider interindividual variability and predict treatment outcomes in patients. The importance of this approach is highlighted through the example of Polymyxin B, showcasing the necessity of pharmacokinetic/pharmacodynamic modelling in conjunction with experimentation for successful translation to clinical practice.
INTERNATIONAL JOURNAL OF ANTIMICROBIAL AGENTS
(2021)
Review
Pediatrics
Margaret Steiner Grillo, Jacklyn Frank, Paul Saenger
Summary: Since 1957, the treatment of growth hormone deficiency has been continuously improved. Currently, there are four long-acting growth hormone formulations available, which have been shown to have comparable safety and tolerability to daily somatropin injections and can improve patient adherence.
FRONTIERS IN PEDIATRICS
(2023)
Article
Engineering, Biomedical
Zhaoying Yang, Qiongqiong Yao, Like Gong, Fan Zhang, Jiawei Sun, Yuanzi Sun, Weiping Gao
Summary: This study reports a method of genetically fusing recombinant human growth hormone (rhGH) to a thermosensitive elastin-like polypeptide (ELP), resulting in the formation of rhGH-ELP fusion protein. The fusion protein exhibits enhanced stability and sustained release, allowing for the promotion of linear growth and development in rats. This approach has the potential to reduce the frequency of rhGH administration and improve patient convenience.
ADVANCED HEALTHCARE MATERIALS
(2023)
Review
Endocrinology & Metabolism
Bradley S. Miller
Summary: Novel long-acting growth hormone (LAGH) therapies have shown promising efficacy and safety in children with pediatric growth hormone deficiency (PGHD), potentially improving patient adherence and quality of life. Clinicians need to carefully select suitable candidates for LAGH therapy and monitor treatment progress accordingly.
FRONTIERS IN ENDOCRINOLOGY
(2022)
Article
Pharmacology & Pharmacy
M. David Paton
Summary: Growth hormone deficiency (GHD) is a condition where the body does not produce enough growth hormone, leading to various physiological problems in adult patients. Traditionally, GHD has been treated with daily subcutaneous injections of growth hormone, which can be burdensome for patients. However, a new long-acting human growth hormone analog, Somapacitan, allows for once-weekly subcutaneous injections, making it the first weekly injection therapy for adult GHD compared to other daily injection therapies.
Article
Pharmacology & Pharmacy
Hans Helleberg, Mads Bjelke, Birgitte Bentz Damholt, Palle Jacob Pedersen, Michael Hojby Rasmussen
Summary: Somapacitan is a reversible growth hormone derivative used for the treatment of GH deficiency. In healthy males, the drug is primarily excreted through urine, with metabolites fully degraded and eliminated. The study demonstrates that it is safe and well tolerated.
EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
(2021)