Ngenla (somatrogon)

To treat growth failure due to inadequate secretion of endogenous growth hormone Drug Trials Snapshot

FDA Approval: 6/27/2023

Research Synopsis

Related articles

Research articles about Ngenla (somatrogon)

Ngenla (somatrogon)

Somatrogon: First Approval.

London, UK

2 hours ago

1 Received

  • - Somatrogon (NGENLA) is a long-acting human growth hormone being developed by Pfizer and OPKO Health to treat growth hormone deficiency in both kids and adults.
  • - It is given as a once-weekly subcutaneous injection, making it easier to manage than traditional once-daily growth hormone treatments, while still being effective.
  • - The drug received its first approval in Canada in October 2021 for treating pediatric patients with growth failure caused by low growth hormone levels.

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Spotlight on Lonapegsomatropin Once-Weekly Injection and Its Potential in the Treatment of Growth Hormone Deficiency in Pediatric Patients.

London, UK

2 hours ago

1 Received

  • Lonapegsomatropin is a long-acting growth hormone therapy approved in August 2021 for treating pediatric growth hormone deficiency, featuring a time-release mechanism allowing for weekly injections.
  • Clinical trials showed that lonapegsomatropin led to greater height velocity in children compared to daily GH, demonstrating its efficacy without serious side effects.
  • The treatment is offered in prefilled cartridges with varying doses and requires a special delivery device, and it may enhance patient adherence and quality of life while prompting new considerations for clinicians in monitoring and dosage adjustment.

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Efficacy and Safety of Once-Weekly Somatrogon Compared with Once-Daily Somatropin (Genotropin®) in Japanese Children with Pediatric Growth Hormone Deficiency: Results from a Randomized Phase 3 Study.

London, UK

2 hours ago

1 Received

  • Somatrogon, a long-acting recombinant human growth hormone, is being tested as a once-weekly treatment for growth hormone deficiency (GHD) in children, compared to the daily treatment of Genotropin in a phase 3 study involving Japanese children.
  • The study randomized 44 prepubertal children to either somatrogon or Genotropin for 12 months, measuring outcomes such as annualized height velocity (HV) at the end of the trial.
  • Results showed that somatrogon had a higher mean HV (9.65 cm/year) compared to Genotropin (7.87 cm/year), with both treatments showing similar safety profiles and mild-to-moderate side effects, although som

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What do we do now that the long-acting growth hormone is here?

London, UK

2 hours ago

1 Received

  • Recent phase III clinical trials show that once-weekly long-acting growth hormone therapies (lonapegsomatropin, somatrogon, and somapacitan) provide similar growth rates and safety as daily growth hormone injections in children with growth hormone deficiency.
  • Lonapegsomatropin was FDA approved in August 2021, while somatrogon received approval in late 2021 and somapacitan was approved for adults in August 2020, with its pediatric trial demonstrating effectiveness as well.
  • New long-acting growth hormone products may enhance patient adherence and overall wellbeing, but further long-term studies are needed to evaluate their effectiveness, safety, and how they compare to daily injections.

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Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study.

London, UK

2 hours ago

1 Received

  • Somatrogon is a long-acting growth hormone treatment designed for children with growth hormone deficiency, administered once a week compared to a daily option (Somatropin).
  • The study compared the treatment burden felt by patients and caregivers during 12-week periods of each injection regimen, using standardized questionnaires to measure overall life interference.
  • Results showed that once-weekly somatrogon significantly reduced treatment burden (lower life interference scores) and was associated with greater convenience and satisfaction than the daily somatropin injections.

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Somatrogon for growth hormone deficiency.

London, UK

2 hours ago

1 Received

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Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period.

London, UK

2 hours ago

1 Received

  • Impaired cilial signaling in the MC4R pathway is linked to obesity in patients with Bardet-Biedl and Alström syndromes, prompting a study on the weight-loss effects of setmelanotide in these groups.
  • The study was a multicenter, double-blind, placebo-controlled trial involving 38 patients over 14 weeks, followed by 52 weeks of open-label treatment, assessing the impact of setmelanotide on body weight.
  • Results indicated that 32.3% of patients aged 12 years or older with Bardet-Biedl syndrome experienced at least a 10% weight reduction after 52 weeks on setmelanotide, with skin hyperpigmentation and injection

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Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results.

London, UK

2 hours ago

1 Received

  • Bardet-Biedl syndrome is a rare genetic disorder causing severe obesity and hyperphagia, and its impact on quality of life has not been well studied.
  • In a Phase 3 trial of 52 weeks of setmelanotide treatment, both children and adults showed significant improvements in health-related quality of life, as measured by the PedsQL for kids and the IWQOL-Lite for adults.
  • The results indicated that after treatment, children had a mean improvement of +11.2 in PedsQL scores, while adults had a mean improvement of +12.0 in IWQOL-Lite scores, with notable correlations found between quality of life and changes in body weight and BMI.

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An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment.

London, UK

2 hours ago

1 Received

  • Somatrogon, a long-acting human growth hormone, is tested as a weekly treatment for children with growth hormone deficiency in a 12-month study compared to daily GH (Genotropin).
  • The study involved 53 prepubertal children, who were divided into groups receiving different doses of somatrogon or Genotropin, and many continued in a long-term safety and efficacy evaluation.
  • Results showed significant improvements in annual height velocity and height standard deviation scores over the treatment period, with most side effects being mild and unrelated to the medication.

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Outcomes in children of women with type 2 diabetes exposed to metformin versus placebo during pregnancy (MiTy Kids): a 24-month follow-up of the MiTy randomised controlled trial.

London, UK

2 hours ago

1 Received

  • Metformin is commonly used during pregnancy in women with type 2 diabetes, but its long-term effects on children are still being studied, specifically regarding their body fat and growth patterns up to 24 months of age.
  • The MiTy Kids follow-up study included 283 children and found no significant differences in average body mass index (BMI) or skinfold thickness between those exposed to metformin and those who received a placebo during pregnancy.
  • Despite the overall similarity in growth trajectories, boys in the metformin group showed higher BMI rates between 6 and 24 months, indicating the need for further research on how metformin may affect male child development.

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Efficacy, safety, quality of life, adherence and cost-effectiveness of long-acting growth hormone replacement therapy compared to daily growth hormone in children with growth hormone deficiency: A systematic review and meta-analysis.

London, UK

2 hours ago

1 Received

  • The study compared the efficacy, safety, adherence, quality of life (QoL), and cost-effectiveness of long-acting growth hormone (LAGH) versus daily growth hormone (GH) in treating growth hormone deficiency (GHD) in children, analyzing data from 16 efficacy/safety studies, 8 adherence studies, and 2 QoL studies.
  • Results showed no significant differences in height velocity or other efficacy and safety outcomes between LAGH and daily GH formulations, although most studies included had some risk of bias.
  • The authors highlighted the need for future high-quality studies to confirm their findings, as well as more real-world data on adherence and QoL, and

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Burden of hyperphagia and obesity in Bardet-Biedl syndrome: a multicountry survey.

London, UK

2 hours ago

1 Received

  • Bardet-Biedl syndrome (BBS) significantly impacts both patients and their caregivers, particularly through hyperphagia, which contributes to early-onset obesity and various emotional and physical challenges.
  • The CAREgiver Burden in BBS (CARE-BBS) study surveyed 242 adult caregivers, revealing common behaviors like food negotiation and overnight food-seeking in patients, as well as notable negative effects on mood, sleep, and school attendance.
  • Results indicated that obesity greatly harms aspects of life such as physical comfort, body esteem, and social interaction, with a substantial portion of patients missing school frequently due to their symptoms.

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Caregiver burden in Bardet-Biedl syndrome: findings from the CARE-BBS study.

London, UK

2 hours ago

1 Received

  • Bardet-Biedl syndrome (BBS) is a rare genetic disorder characterized by obesity and uncontrollable hunger (hyperphagia), significantly affecting the lives of caregivers.
  • A survey conducted across the US, UK, Canada, and Germany involved 242 caregivers, revealing extensive caregiver burden that impacts their mood, sleep, relationships, and work productivity due to the demands of caring for individuals with BBS.
  • Caregivers reported high personal and family strain along with substantial financial costs, indicating the need for more effective weight management strategies and support resources for those affected by BBS.

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Somatrogon-ghla.

London, UK

2 hours ago

1 Received

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Cost-effectiveness and cost-utility analysis of somatrogon once-weekly injections vs. daily growth hormone injection for treating paediatric growth hormone deficiency in Ireland.

London, UK

2 hours ago

1 Received

  • Pediatric growth hormone deficiency (pGHD) results in growth failure due to insufficient growth hormone production, and the standard treatment with daily injections of recombinant human GH (somatropin) often suffers from poor patient adherence.
  • A new treatment, somatrogon, which requires once-weekly injections, has shown similar effectiveness and reduces treatment burden compared to somatropin in Phase 3 clinical trials.
  • A cost-effectiveness analysis from an Irish healthcare perspective indicates that somatrogon offers greater height gain, improved quality of life, and significant cost savings compared to daily growth hormone injections.

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Cost-effectiveness of somatrogon in the Spanish pediatric population with growth hormone deficiency.

London, UK

2 hours ago

1 Received

  • The study aims to assess the cost-effectiveness of weekly somatrogon versus daily growth hormone treatments in young children in Spain with growth hormone deficiency.
  • A Markov model was used to simulate health outcomes and costs from the perspective of the Spanish National Health System, focusing on untreated prepubertal children with GHD.
  • Results indicated that somatrogon is cost-effective, providing a good quality-adjusted life year (QALY) gain at a cost of €19,259 per QALY, and has a 61% probability of being the preferred treatment option at a willingness-to-pay threshold of €25,000 per QALY gained.

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Physician experience with once-weekly somatrogon versus once-daily rhGH regimen in pediatric patients with growth hormone deficiency: a cross-sectional survey of physicians from the global phase 3 study.

London, UK

2 hours ago

1 Received

  • The study evaluates physicians' preferences and experiences with once-weekly somatrogon, a long-acting growth hormone, compared to the standard once-daily rhGH for treating pediatric growth hormone deficiency (pGHD).
  • Survey results showed that 75% of pediatric endocrinologists preferred somatrogon, citing its convenience and lower burden on patients and caregivers.
  • A majority found both treatment regimens equally effective for long-term growth outcomes, but significantly more were "very satisfied" with somatrogon.

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Somatrogon injection for the treatment of pediatric growth hormone deficiency with comparison to other LAGH products.

London, UK

2 hours ago

1 Received

  • Somatrogon (NGENLA™) is a long-acting growth hormone therapy approved for treating pediatric growth hormone deficiency in multiple countries, allowing for administration just once a week.
  • Clinical trials showed that somatrogon was effective in increasing height without the concerning side effects typically associated with daily GH injections, matching the growth rates of other treatments.
  • New long-acting growth hormone products like somatrogon could enhance patient adherence to treatment and improve overall quality of life, but ongoing studies are required to assess their long-term effectiveness and safety.

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Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin.

London, UK

2 hours ago

1 Received

  • The study aimed to compare the effectiveness and safety of once-weekly somatrogon versus once-daily somatropin in treating pediatric growth hormone deficiency (pGHD) over 12 months, focusing on height velocity and health-related quality of life (HRQoL).
  • Researchers administered the QoLISSY questionnaire to assess changes in HRQoL among treatment-naïve, prepubertal children, with caregivers' input for younger children and self-reports from older children.
  • Results showed similar improvements in HRQoL in both treatment groups after 12 months, with children often reporting better HRQoL than their parents perceived, indicating that children's perspectives are crucial in assessing treatment outcomes.

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