Status of long-acting-growth hormone preparations — 2015

Höybye, Charlotte; Cohen, Pinchas; Hoffman, Andrew R.; Ross, R. J. M.; Biller, Beverly M. K.; Christiansen, Jens Sandahl

doi:10.1016/j.ghir.2015.07.004

Cited by 61 publications

(48 citation statements)

References 53 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Newer therapeutics include bioequivalent GH products and a combined GH/IGF-I product [265] . The most sought-after intervention possibility continues to be an effective sustained-release GH preparation that will reduce the required frequency of injections [266,267] . Likewise, some studies have suggested potential benefit of treating patients with GH who have non-FDA-approved indications, such as Crohn disease, cystic fibrosis, and glucocorticoid-dependent patients, and some have advocated further expanding the potential patient population by relaxing the height criteria for ISS and PIGFD; these topics are beyond the scope of this document to review.…”

Section: Discussionmentioning

confidence: 99%

Guidelines for Growth Hormone and Insulin-Like Growth Factor-I Treatment in Children and Adolescents: Growth Hormone Deficiency, Idiopathic Short Stature, and Primary Insulin-Like Growth Factor-I Deficiency

DiVall²,

et al. 2016

View full text Add to dashboard Cite

Background/Aims: On behalf of the Drug and Therapeutics, and Ethics Committees of the Pediatric Endocrine Society, we sought to update the guidelines published in 2003 on the use of growth hormone (GH). Because idiopathic short stature (ISS) remains a controversial indication, and diagnostic challenges often blur the distinction between ISS, GH deficiency (GHD), and primary IGF-I deficiency (PIGFD), we focused on these three diagnoses, thereby adding recombinant IGF-I therapy to the GH guidelines for the first time. Methods: This guideline was developed following the GRADE approach (Grading of Recommendations, Assessment, Development, and Evaluation). Results: This guideline provides recommendations for the clinical management of children and adolescents with growth failure from GHD, ISS, or PIGFD using the best available evidence. Conclusion: The taskforce suggests that the recommendations be applied in clinical practice with consideration of the evolving literature and the risks and benefits to each individual patient. In many instances, careful review highlights areas that need further research.

show abstract

Section: Discussionmentioning

confidence: 99%

Guidelines for Growth Hormone and Insulin-Like Growth Factor-I Treatment in Children and Adolescents: Growth Hormone Deficiency, Idiopathic Short Stature, and Primary Insulin-Like Growth Factor-I Deficiency

DiVall²,

et al. 2016

View full text Add to dashboard Cite

show abstract

“…These included pediatric and adult endocrinologists, basic scientists, regulatory scientists from the FDA and EMA, and scientists from the pharmaceutical industry. A review of the current status of LAGH development was published before the meeting (9). A planning committee of the GRS comprising adult and pediatric endocrinologists from academic institutions determined the agenda, selected speakers to summarize key relevant topics, and formulated the questions for discussion at this workshop.…”

Section: Methodsmentioning

confidence: 99%

Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations

Christiansen

Backeljauw

Bidlingmaier

et al. 2016

European Journal of Endocrinology

Self Cite

116

147

View full text Add to dashboard Cite

ObjectiveThe Growth Hormone (GH) Research Society (GRS) convened a workshop to address important issues regarding trial design, efficacy, and safety of long-acting growth hormone preparations (LAGH).ParticipantsA closed meeting of 55 international scientists with expertise in GH, including pediatric and adult endocrinologists, basic scientists, regulatory scientists, and participants from the pharmaceutical industry.EvidenceCurrent literature was reviewed for gaps in knowledge. Expert opinion was used to suggest studies required to address potential safety and efficacy issues.Consensus processFollowing plenary presentations summarizing the literature, breakout groups discussed questions framed by the planning committee. Attendees reconvened after each breakout session to share group reports. A writing team compiled the breakout session reports into a draft document that was discussed and revised in an open forum on the concluding day. This was edited further and then circulated to attendees from academic institutions for review after the meeting. Participants from pharmaceutical companies did not participate in the planning, writing, or in the discussions and text revision on the final day of the workshop. Scientists from industry and regulatory agencies reviewed the manuscript to identify any factual errors.ConclusionsLAGH compounds may represent an advance over daily GH injections because of increased convenience and differing phamacodynamic properties, providing the potential for improved adherence and outcomes. Better methods to assess adherence must be developed and validated. Long-term surveillance registries that include assessment of efficacy, cost-benefit, disease burden, quality of life, and safety are essential for understanding the impact of sustained exposure to LAGH preparations.

show abstract

“…Nutropin Depot, the first long-acting rhGH formulation, was withdrawn from the market in 2004 because of manufacturing issues (1, 2, 3). Subsequently, a few novel long-acting rhGH preparations such as NNC126-0083, LB03002, PHA-794428 and VRS-317 have been developed and evaluated (3, 4, 5, 6, 7, 8, 9, 10, 11, 12). Among them, further developments for PHA-794428 was stopped due to high incidences of injection-site lipoatrophy after first injection (3, 10).…”

Section: Introductionmentioning

confidence: 99%

“…Subsequently, a few novel long-acting rhGH preparations such as NNC126-0083, LB03002, PHA-794428 and VRS-317 have been developed and evaluated (3, 4, 5, 6, 7, 8, 9, 10, 11, 12). Among them, further developments for PHA-794428 was stopped due to high incidences of injection-site lipoatrophy after first injection (3, 10). Additionally, the efficacy and safety of long-acting rhGH preparations such as ACP-001, NNbib195-0092, GX-H9, HM10560A, MOD-4023 and VRS-317 in adults and children are currently being evaluated by ongoing phase II or III clinical studies (13).…”

Section: Introductionmentioning

confidence: 99%

Long-acting PEGylated recombinant human growth hormone (Jintrolong) for children with growth hormone deficiency: phase II and phase III multicenter, randomized studies

Luo

Hou

et al. 2017

European Journal of Endocrinology

View full text Add to dashboard Cite

ObjectiveWe assessed the efficacy and safety of a weekly pegylated human growth hormone (PEG-rhGH) (Jintrolong) vs daily rhGH for children with growth hormone deficiency (GHD).DesignPhase II and III, multicenter, open-label, randomized controlled trials.Methods108 and 343 children with treatment-naive GHD from 6 hospitals in China were enrolled in the phase II and III studies respectively. Patients in the phase II study were randomized 1:1:1 to weekly Jintrolong (0.1 mg/kg/week PEG-rhGH complex), weekly Jintrolong (0.2 mg/kg/week PEG-rhGH complex) or daily rhGH (0.25 mg/kg/week) for 25 weeks. Patients in the phase III study were randomized in a 2:1 ratio to weekly Jintrolong (0.2 mg/kg/week) or daily rhGH (0.25 mg/kg/week) for 25 weeks. The primary endpoint for both studies was height velocity (HV) increase at the end of treatment. Other growth-related parameters, safety and compliance were also monitored.ResultsThe phase II study established the preliminary efficacy, safety and recommended dose of Jintrolong PEG-rhGH. In the phase III study, we demonstrated significantly greater HV increases in patients receiving Jintrolong treatment (from 2.26 ± 0.87 cm/year to 13.41 ± 3.72 cm/year) vs daily rhGH (from 2.25 ± 0.82 cm/year to 12.55 ± 2.99 cm/year) at the end of treatment (P < 0.05). Additionally, significantly greater improvement in the height standard deviation scores was associated with Jintrolong throughout the treatment (P < 0.05). Adverse event rates and treatment compliance were comparable between the two groups.ConclusionJintrolong PEG-rhGH at a dose of 0.2 mg/kg/week for 25 weeks is effective and safe for GHD treatment and is non-inferior to daily rhGH.

show abstract

Status of long-acting-growth hormone preparations — 2015

Cited by 61 publications

References 53 publications

Guidelines for Growth Hormone and Insulin-Like Growth Factor-I Treatment in Children and Adolescents: Growth Hormone Deficiency, Idiopathic Short Stature, and Primary Insulin-Like Growth Factor-I Deficiency

Guidelines for Growth Hormone and Insulin-Like Growth Factor-I Treatment in Children and Adolescents: Growth Hormone Deficiency, Idiopathic Short Stature, and Primary Insulin-Like Growth Factor-I Deficiency

Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations

Long-acting PEGylated recombinant human growth hormone (Jintrolong) for children with growth hormone deficiency: phase II and phase III multicenter, randomized studies

Contact Info

Product

Resources

About