Pediatric Growth Hormone Deficiency Market

• Pediatric Growth Hormone Deficiency (PGHD) is a significant endocrine disorder characterized by insufficient production of growth hormone (GH) from the pituitary gland, leading to notably slow growth and short stature in children. 
• In the United States, PGHD is estimated to affect approximately 1 in 4,000 to 1 in 10,000 children, with both congenital and acquired forms observed.
• The market is anticipated to witness a substantial positive shift owing to better uptake of existing drugs, the expected market launch of therapies, and raised awareness.
• The approved therapies for PGHD are as follow- SKYTROFA,GENOTROPIN, and NGENLA.
• The United States accounts for the largest market size of PGHD, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
• GHD pipeline is not so robust but possesses potential drug i.e. LUM-201.
• In October 2024, Lumos Pharma, announced that updated analyses of data from the company’s Phase II OraGrowtH210 and OraGrowtH212 clinical trials had been accepted for oral presentation at the 62nd Annual European Society for Paediatric Endocrinology Meeting, or ESPE 2024, which was to be held November 16-18, 2024, in UK.
• In August 2024, Lumos Pharma had made substantial progress in finalizing its proposal for a Phase III double-blinded, placebo-controlled trial with a 2:1 randomization involving about 150 subjects. It expected to finalize design details with the FDA in the fourth quarter while prudently managing cash resources and evaluating strategic opportunities to initiate the trial in the second quarter of 2025.

DelveInsight’s "Pediatric Growth Hormone Deficiency – Market Insight, Epidemiology, and Market Forecast – 2034" report delivers an in-depth understanding of the Pediatric Growth Hormone Deficiency historical and forecasted epidemiology as well as the Pediatric Growth Hormone Deficiency market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Pediatric Growth Hormone Deficiency market report offers an in-depth analysis of current treatment practices, market share of individual therapies, and the projected market size from 2020 to 2034, segmented across seven major markets. Additionally, the report examines treatment algorithms and unmet medical needs, identifying key opportunities and assessing the market's underlying potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Pediatric Growth Hormone Deficiency: Disease Understanding and Treatment Algorithm

Pediatric Growth Hormone Deficiency Overview

Pediatric Growth Hormone Deficiency (PGHD) represents a significant health concern, impacting children's physical development and overall well-being. This condition occurs when the pituitary gland fails to produce adequate growth hormone (GH), leading to symptoms such as short stature and delayed growth. Key risk factors include congenital abnormalities, acquired conditions such as tumors or trauma, and genetic mutations. As awareness of PGHD grows, the demand for effective treatment strategies is increasing, particularly as early intervention is crucial for optimizing growth potential.

The diagnosis of PGHD involves a comprehensive clinical evaluation supported by specific diagnostic tests. A critical component of the diagnostic process is auxology, which involves plotting the child’s height and weight on growth charts to evaluate growth over time. Blood tests are essential for measuring levels of insulin-like growth factor 1 (IGF-1) and IGF binding protein 3 (IGFBP-3), providing valuable information when interpreted alongside clinical findings. Provocative growth hormone testing may also be conducted to stimulate GH secretion, although this method has limitations due to variability based on age and body composition. Neuroimaging, particularly magnetic resonance imaging (MRI), is performed to assess for structural abnormalities in the pituitary gland or hypothalamus if GHD is suspected. Additionally, it is crucial to rule out other potential causes of short stature, including thyroid disorders and genetic syndromes, through further blood tests and possibly genetic testing. 

Further details related to diagnosis will be provided in the report…

Pediatric Growth Hormone Deficiency Treatment

The treatment of PGHD is essential for promoting healthy growth and preventing long-term complications. The primary approach involves the immediate initiation of hormone replacement therapy, specifically through daily subcutaneous injections of recombinant human growth hormone (rhGH), commonly known as somatropin. This therapy should begin as soon as the diagnosis is confirmed to optimize growth potential. Recent advancements have introduced long-acting formulations, such as NGENLA (somatrogon-ghla), which allow for weekly injections, thereby improving adherence to treatment. Alongside rhGH therapy, it is crucial to address any underlying conditions that may contribute to growth issues, such as thyroid hormone deficiencies or other hormonal imbalances. Supportive care may also include counseling for families to help them manage treatment logistics and expectations. As treatment continues, healthcare providers will closely monitor height velocity and adjust dosages accordingly, ensuring that children achieve their target adult height. Timely and effective management of PGHD is vital not only for physical development but also for enhancing overall quality of life and psychological well-being in affected children. 

Further details related to treatment will be provided in the report…

Pediatric Growth Hormone Deficiency Epidemiology

The PGHD epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the total prevalent cases of pghd, diagnosed prevalent cases of pghd, risk factors specific cases of pghd, type-specific cases of pghd, gender specific cases of pghd, and total treated cases of pghd in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• According to findings, among children with short stature, GHD happens in approximately 1:4,000 to 1:10,000 cases.
• It is usually idiopathic, but about 25% of patients have an identifiable etiology.
• As per the analysis, the prevalence rate of children in Italy with GHD was 8.62–9.44/10,000.

Drug Class Insight

The drug chapter segment of the Pediatric Growth Hormone Deficiency report encloses a detailed analysis of the emerging drugs. The drug chapter also helps understand the Pediatric Growth Hormone Deficiency clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Marketed Drugs

SKYTROFA: Ascendis Pharma

Ascendis Pharma's SKYTROFA (lonapegsomatropin-tcgd) is a groundbreaking treatment PGHD, approved by the FDA in 2021. It is notable for being the first and only once-weekly growth hormone therapy, which significantly reduces the burden of daily injections that have been the standard for over 30 years.

SKYTROFA is indicated for children aged one year and older who weigh at least 11.5 kg and have growth failure due to inadequate endogenous growth hormone secretion. Clinical studies demonstrated that children using SKYTROFA experienced an average growth rate of 11.2 cm per year, compared to 10.3 cm per year with daily somatropin, showcasing its effectiveness.

The drug utilizes Ascendis' innovative TransCon technology, allowing for a controlled release of somatropin over time, which enhances patient compliance and convenience. SKYTROFA is administered via a specially designed auto-injector, which simplifies the injection process and can be stored at room temperature for up to six months. As Ascendis Pharma continues to expand its market presence, SKYTROFA represents a significant advancement in the treatment of PGHD.

NGENLA (somatrogon-ghla): Pfizer

Somatrogon-ghla, a human growth hormone analog, is a fusion protein. It binds to the GH receptor and initiates a signal transduction cascade culminating in changes in growth and metabolism. Somatrogon-ghla binding leads to activation of the STAT5b signaling pathway and increases the serum concentration of Insulin-like Growth Factor (IGF-1). GH and IGF-1 stimulate metabolic changes, linear growth, and enhance growth velocity in pediatric patients with GHD. 

In June 2023, Pfizer announced that the US FDA approval of NGENLA for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone.

Detailed therapy assessment will be provided in the final report.

Emerging Drugs

LUM-201: Lumos Pharma

LUM-201, also known as ibutamoren, is an investigational oral growth hormone (GH) secretagogue developed by Lumos Pharma for the treatment of Pediatric Growth Hormone Deficiency (PGHD).

LUM-201 functions as a GH secretagogue receptor agonist, stimulating the secretion of growth hormone from the pituitary gland. Unlike traditional treatments that require daily or weekly injections of recombinant human growth hormone (rhGH), LUM-201 is administered orally once daily, which may enhance patient compliance and comfort. The OraGrowtH210 and OraGrowtH212 trials are Phase II clinical studies conducted by Lumos Pharma to evaluate the efficacy and safety of LUM-201, an oral treatment for Pediatric Growth Hormone Deficiency (PGHD).

LUM-201 is currently being evaluated in multiple Phase II clinical studies in Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan Drug Designation in both the US and EU.

Detailed therapy assessment will be provided in the final report.

Pediatric Growth Hormone Deficiency Market Outlook

The market outlook for Pediatric Growth Hormone Deficiency (PGHD) is increasingly shaped by the growing recognition of the need for effective treatment strategies and early intervention. With a global prevalence that significantly impacts various demographics, particularly children with congenital conditions or those affected by brain trauma, there is a pressing demand for innovative therapeutic solutions. Current treatment protocols primarily emphasize hormone replacement therapy, specifically through recombinant human growth hormone (rhGH) injections, which are essential for promoting normal growth and development. The recent introduction of long-acting formulations, such as NGENLA (somatrogon-ghla), allows for weekly dosing instead of daily injections, enhancing patient adherence and convenience for families.

Detailed market assessment will be provided in the final report.

KOL- Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the Pediatric Growth Hormone Deficiency evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Gastroenterologists, Geriatricians, Pelvic Floor Physical Therapists, Surgeons and others.

DelveInsight’s analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as the nfectious Diseases Society of America (IDSA), American Academy of Neurology (AAN), Neurocritical Care Society (NCS), Mount Sinai Health System etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Pediatric Growth Hormone Deficiency market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of Pediatric Growth Hormone Deficiency, explaining its causes, signs, symptoms, pathogenesis, and currently used therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• A detailed review of the Pediatric Growth Hormone Deficiency market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and KOL views, patient journey, and treatment preferences that help shape and drive the Pediatric Growth Hormone Deficiency market.

Pediatric Growth Hormone Deficiency Report Insights

• Patient Population
• Therapeutic Approaches
• Pediatric Growth Hormone Deficiency Pipeline Analysis
• Pediatric Growth Hormone Deficiency Market Size and Trends
• Existing and Future Market Opportunity 

Pediatric Growth Hormone Deficiency Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage 
• Pediatric Growth Hormone Deficiency Epidemiology Segmentation
• Key Market Forecast Assumptions

Pediatric Growth Hormone Deficiency Report Assessment

• Current Treatment Practices
• Unmet Needs
• Market Attractiveness
• Qualitative Analysis (SWOT)

FAQs

• What was the Pediatric Growth Hormone Deficiency market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like by 2034? What are the contributing factors for this growth?
• What can be the future treatment paradigm for Pediatric Growth Hormone Deficiency?
• What are the disease risks, burdens, and unmet needs of Pediatric Growth Hormone Deficiency? What will be the growth opportunities across the 7MM concerning the patient population with Pediatric Growth Hormone Deficiency?

• What are the current options for the treatment of Pediatric Growth Hormone Deficiency? What are the current guidelines for treating Pediatric Growth Hormone Deficiency in the 7MM?

• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies? 
• What is the patient share in Pediatric Growth Hormone Deficiency?

Reasons to Buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the Pediatric Growth Hormone Deficiency market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong players in the market will help devise strategies to help get ahead of competitors.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.