Pediatric Growth Hormone Deficiency - Pipeline Insight, 2025

This “Pediatric Growth Hormone Deficiency - Pipeline Insight, 2025” report provides comprehensive insights about 2+ companies and 3+ pipeline drugs in Pediatric Growth Hormone Deficiency pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Pediatric Growth Hormone Deficiency: Understanding

Pediatric Growth Hormone Deficiency: Overview

Pediatric growth hormone deficiency (GHD) is a condition characterized by insufficient secretion of growth hormone from the anterior pituitary gland, leading to impaired growth and short stature in children. This deficiency can be congenital, resulting from genetic mutations or structural abnormalities of the pituitary gland, or acquired later in life due to factors such as brain injury, tumors, or infections. Children with GHD typically present with normal body proportions but exhibit significantly reduced growth rates compared to their peers, often falling below the fifth percentile for height. The diagnosis of GHD involves a combination of clinical assessment, including monitoring growth patterns and auxological measurements, along with biochemical tests to evaluate growth hormone levels and exclude other potential causes of growth delay.

Magnetic resonance imaging (MRI) of the brain may also be performed to detect any pituitary or hypothalamic abnormalities. Treatment primarily consists of recombinant human growth hormone therapy, which has been shown to effectively enhance growth and improve final adult height outcomes when started early in life. Early diagnosis and intervention are crucial for optimizing growth potential and overall health in affected children, as untreated GHD can lead to long-term developmental and psychological consequences. With appropriate treatment, many children with GHD can achieve near-normal height and lead healthy, active lives.

The epidemiology of pediatric growth hormone deficiency (GHD) suggests that it is a relatively rare condition, with an estimated prevalence ranging from 1 in 3,500 to 1 in 10,000 children. Isolated GHD, which accounts for most cases, occurs in approximately 1 in 4,000 to 1 in 10,000 children and is often idiopathic. However, around 25% of cases can be attributed to identifiable causes, such as genetic mutations or structural abnormalities of the pituitary gland. GHD can also present as part of broader pituitary hormone deficiencies or occur independently. Diagnosis typically occurs during childhood when growth patterns deviate significantly from age-appropriate norms, prompting concerns about short stature. Factors such as sex, age at diagnosis, and the underlying cause of GHD can influence the clinical presentation, severity, and management of the condition. Early detection and timely intervention are essential for optimizing growth outcomes, as well as overall health and development, in affected children..

The diagnosis of pediatric growth hormone deficiency (GHD) is a comprehensive process that combines clinical evaluation, biochemical testing, and imaging studies. Initial assessment includes tracking growth patterns through auxologic measurements, where height and weight are plotted on growth charts to assess growth velocity and identify any significant deviations. A notable drop in height velocity, particularly below -2 standard deviations for age, often signals the need for further investigation. Biochemical tests, including the measurement of insulin-like growth factor 1 (IGF-1) and IGF binding protein 3 (IGFBP-3), provide valuable insight but are not definitive on their own. Provocative growth hormone testing, which assesses GH secretion through stimulation tests such as insulin tolerance or glucagon stimulation, is typically required to confirm the diagnosis.

The treatment and management of pediatric growth hormone deficiency (GHD) primarily involve the administration of recombinant human growth hormone (rhGH) to stimulate growth and improve overall health outcomes. The standard treatment protocol includes daily subcutaneous injections of rhGH, with dosages typically ranging from 0.03 to 0.05 mg/kg per day, initiated once a diagnosis is confirmed through clinical evaluation and biochemical testing, including growth hormone stimulation tests. RhGH therapy has been shown to significantly enhance growth velocity, especially in the first year of treatment, where children may experience height increases of 10 to 12 cm/year. Treatment usually continues until the child reaches an acceptable adult height or growth velocity falls below 2.5 cm/year, which typically occurs when the epiphyseal growth plates close during late puberty.

'Pediatric Growth Hormone Deficiency- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Pediatric Growth Hormone Deficiency pipeline landscape is provided which includes the disease overview and Pediatric Growth Hormone Deficiency treatment guidelines. The assessment part of the report embraces, in depth Pediatric Growth Hormone Deficiency commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Pediatric Growth Hormone Deficiency collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Pediatric Growth Hormone Deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Pediatric Growth Hormone Deficiency.

Pediatric Growth Hormone Deficiency Emerging Drugs Chapters

This segment of the Pediatric Growth Hormone Deficiency report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Pediatric Growth Hormone Deficiency Emerging Drugs

TJ101: TJ Biopharma Co., Ltd.

TJ101 is one of the long-acting hGH products, which uses hyFc fusion protein technology to increase the half-life in the body, and it is administered once a week or twice-monthly. It is a biobetter product that significantly increases the convenience of administration for patients compared to the conventional short-acting hGH administered daily. It is a long-acting growth hormone preparation that combines a platform that has non-cytolytic properties because it uses a sequence that does not have the ability to induce cytotoxicity among the sites of IgG4 and IgD that exist in vivo and a human growth hormone secreted from the anterior pituitary. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Pediatric Growth Hormone Deficiency.

SCO-240: SCOHIA PHARMA, Inc.

SCO-240 is a once-daily oral medication that provides an alternative to growth hormone injections. SCO-240 selectively antagonizes somatostatin receptor type 5 (SSTR5). SCO-240 stimulates a robust growth hormone (GH) secretion without affecting the secretion of other pituitary hormones. Collectively, SCO-240-mediated SSTR5 antagonism is a novel oral therapy for GH-related disorders, including pediatric GH deficiency (PGHD). Currently, the drug is in Phase I stage of its clinical trial for the treatment of Pediatric Growth Hormone Deficiency.

Pediatric Growth Hormone Deficiency: Therapeutic Assessment

This segment of the report provides insights about the different Pediatric Growth Hormone Deficiency drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Pediatric Growth Hormone Deficiency

• There are approx. 2+ key companies which are developing the therapies for Pediatric Growth Hormone Deficiency. The companies which have their Pediatric Growth Hormone Deficiency drug candidates in the most advanced stage, i.e. Phase III include, TJ Biopharma Co., Ltd

Phases

The report covers around 3+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Pediatric Growth Hormone Deficiency pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Pediatric Growth Hormone Deficiency: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Pediatric Growth Hormone Deficiency therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Pediatric Growth Hormone Deficiency drugs.

Pediatric Growth Hormone Deficiency Report Insights

• Pediatric Growth Hormone Deficiency Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Pediatric Growth Hormone Deficiency Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Pediatric Growth Hormone Deficiency drugs?
• How many Pediatric Growth Hormone Deficiency drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pediatric Growth Hormone Deficiency?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pediatric Growth Hormone Deficiency therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Pediatric Growth Hormone Deficiency and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• TJ Biopharma Co., Ltd.

Lumos Pharma.

SCOHIA PHARMA, Inc.

Key Products

• TJ101
• LUM-201
• SCO-240

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