transcon hgh hGH - Transconcnp hGH TransCon hGH: A Novel Approach to Growth Hormone Deficiency Treatment

Transconpth TransCon hGH, also known by its active ingredient lonapegsomatropin, represents a significant advancement in the treatment of growth hormone deficiency (GHD)作者：AK Maniatis·2021·被引用次数：4—Background: Once-weeklyTransCon hGH(lonapegsomatropin) is an investigational long-acting prodrug of somatropin in development for GHD.. This innovative long-acting prodrug of somatropin is designed for once-weekly administration, offering a more convenient and potentially more effective therapeutic option compared to traditional daily injections. The development of TransCon hGH by Ascendis Pharma signifies a commitment to improving the lives of individuals with GHD, from children to adults.

Understanding Growth Hormone Deficiency (GHD)

Growth hormone (GH) is a crucial hormone produced by the pituitary gland, vital for growth and development in children and for maintaining various metabolic functions in adults. When the body doesn't produce enough GH, it leads to GHD, characterized by impaired growth in children and metabolic disturbances in adults. Traditional treatments involve daily injections of recombinant human growth hormone (hGH), which, while effective, can be burdensome for patients and their caregivers.

The TransCon hGH Technology: A Paradigm Shift

The core innovation behind TransCon hGH lies in its proprietary TransCon™ technology. This technology enables the creation of a prodrug that, once administered, is designed to release the active, unmodified somatropin (identical to endogenous hGH) at a predictable rate over a week.FDA Accepts Ascendis Pharma's Supplemental Biologics ... Unlike other sustained-release formulations, TransCon hGH’s approach ensures that the active hormone is released without any chemical modifications, aiming to provide a therapeutic profile similar to daily injections but with a significantly reduced dosing frequency.A Phase 3 Trial ofTransCon hGH(lonapegsomatropin) for the Treatment for Pediatric Growth Hormone Deficiency (GHD). Status: Active, no longer recruiting. Phase ... This sustained release mechanism is key to its efficacy and improved patient experience.

Clinical Efficacy and Safety of TransCon hGH

Extensive clinical trials have demonstrated the safety and efficacy of TransCon hGH across different patient populations.2024年12月12日—...TransCon™hGHfor the Treatment of Adults with Growth Hormone Deficiency ... human growth hormone (hGH) in adults with GHD. The trial ...

* Pediatric Growth Hormone Deficiency (GHD): Studies, such as the NCT03305016 trial, have evaluated the safety, tolerability, and efficacy of once-weekly TransCon hGH in children with GHD2019年9月19日—Results: Baseline demographics were similar between the two groups. AHV at 52 weeks was 11.2 cm/y for theTransCon hGHgroup vs. 10.3 cm/y for .... These trials have shown comparable efficacy to daily hGH therapy, with positive outcomes in terms of height velocity (AHV). For instance, a 52-week trial comparing once-weekly TransCon hGH to daily hGH in pediatric patients showed an AHV of 11.2 cm/year for the TransCon hGH group versus 10.3 cm/year for the daily hGH group作者：S Hu·2025·被引用次数：1—Human growth hormone (hGH) is a protein hormone that is secreted by ... For example, Lonapegsomatropin [15], the first prodrug utilizingTransCon.... Furthermore, TransCon hGH has demonstrated an anti-hGH immunogenic profile comparable to daily hGH, suggesting reduced potential for antibody formation that could impede treatment effectiveness.Pediatric phase 2 data demonstrate that TransCon hGH ... The New InsiGHTS Trial has provided topline data showing comparable safety and endocrine benefits to daily treatments at week 26.

* Adult Growth Hormone Deficiency (GHD): The efficacy and safety of TransCon hGH have also been established for adults with GHD. The FDA has approved lonapegsomatropin (marketed as Skytrofa) for the treatment of adults with GHD. Clinical studies, including those presented in Phase 3 trials, have shown that TransCon hGH can lead to significant improvements in body composition, such as a reduction in trunk fat and an increase in total body lean mass.NCT03305016 | A Safety, Tolerability and Efficacy Study of ... For example, at Week 38, TransCon hGH demonstrated a statistically significant reduction from baseline in trunk fat and an increase in total body lean mass compared to placebo. Long-term extension trials, like the ENLIGHTEN trial, are further investigating the sustained benefits of TransCon hGH in adults. Ascendis Pharma has actively pursued FDA label expansion for adult growth hormone deficiency, submitting supplemental Biologics License Applications (sBLAs) to the FDA for TransCon hGH for this indication.

* Combination Therapy: Promising research is also exploring the potential of combining TransCon hGH with other therapies. For instance, the combination of TransCon hGH with TransCon CNP has shown potential in improving linear growth and body proportionality in conditions like achondroplasia.

Safety Considerations

The safety results from various clinical trials indicate that TransCon hGH is generally safe and well-tolerated. Studies have reported no discontinuations related to the study drug, and the observed safety profile has been comparable to existing hGH therapies. The focus on delivering unmodified hGH at a predictable rate over one week is designed to optimize therapeutic outcomes while minimizing risks.

Regulatory Milestones and Future Directions

TransCon hGH has achieved significant regulatory milestones. It was first approved by the FDA in 2021 as a treatment for pediatric GHDPhase 3 Clinical Trial in Adult Growth Hormone Deficiency .... Subsequent approvals and ongoing submissions for adult GHD highlight its expanding therapeutic utility.2024年12月12日—...TransCon™hGHfor the Treatment of Adults with Growth Hormone Deficiency ... human growth hormone (hGH) in adults with GHD. The trial ... Ongoing research and clinical trials, including long-term extension studies and investigations into combination therapies, continue to shape the future of TransCon hGH as a leading treatment option for GHD. The development of TransCon hGH exemplifies the progress in peptide therapy and protein based therapy for rare diseases and endocrine disorders.A 52 week trial ofTransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy.TransCon hGHwill be given once-a-week, ... Ascendis Pharma remains at the forefront of this innovation, aiming to provide improved treatment paradigms for patients worldwide. The continued exploration of TransCon CNP alongside TransCon hGH signifies a comprehensive approach to addressing complex growth disordersTransCon hGH Aims for FDA Label Expansion for Adult .... The ultimate goal is to provide a transformative treatment that enhances the quality of life for individuals living with growth hormone deficiency.