Achondroplasia - Market Insight, Epidemiology and Market Forecast - 2032

Key Highlights

• The diagnosed prevalence of achondroplasia has been increasing in the US due to improved understanding among healthcare professionals, advances in diagnosis, besides an increasing population.
• Advances in genetic testing, such as chorionic villus sampling, new-generation sequencing, and amniocentesis, allow for the prenatal diagnosis of achondroplasia.
• Management of achondroplasia is symptomatic and requires a multidisciplinary approach involving pharmacological and nonpharmacological interventions. The current treatment landscape has only one approved therapy in the US and Europe, VOXZOGO (vosoritide), while in Japan, growth hormone has also been authorized for the management of achondroplasia; however, its long-term benefits are controversial.
• Other therapies like statins, anti-infectives, antihistamines, CNP analogs, etc, are also used to manage symptoms and aid surgical procedures of achondroplasia. Further, surgical therapies like adenotonsillectomy, suboccipital decompression, tracheostomy, laminectomy, and physical therapy play a vital role in managing several complications of achondroplasia.
• One of the major concerns in understanding the achondroplasia market is the paucity of evidence to validate interventions used in the daily management of achondroplasia.
• There is a lack of curative therapy; however, the presence of clinical and diagnostic guidelines, recommendations, and consensus statements have enabled informed treatment and management, the American Academy of Pediatric's Health Supervision for Children with Achondroplasia, International Consensus Statement on the Diagnosis, Multidisciplinary Management, and Lifelong Care of Individuals with Achondroplasia, and Japanese Society for Pediatric Endocrinology's Clinical Practice Guidelines for Achondroplasia are few among them. However, the detailed basis for recommendations and the existing uncertainties are yet to be explored.
• Achondroplasia is associated with poor quality of life, substantial economic burdens, negative stereotypes, and social challenges.
• In 2022, the US had the largest market size of achondroplasia among the 7MM, accounting for approximately USD 111.85 million, expected to increase further by 2032.
• The approved drug is VOXZOGO (vosoritide), a C-type natriuretic peptide (CNP) analog, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. The drug is indicated to increase linear growth in children with achondroplasia having open epiphyses.
• Although much has been learned about the disorder, limited pharmacological therapies are available, and surgeries have the risk of morbidity. There is an urgent need for an effective therapy that can restore skeletal growth and improve patients' quality of life.
• Ongoing research into achondroplasia has led to the discovery of potential therapies that might alleviate some of the symptoms and complications associated with the condition. Emerging therapies like Ascendis Pharma's TransCon CNP (navepegritide) and QED Therapeutics (BridgeBio)/Novartis' infigratinib (BBP-831/BGJ398) have the potential to quench the thirst for effective therapies. The achondroplasia market will change during the forecast period due to the expected approval of these emerging therapies, leading to the entry of new players in the treatment landscape.

This “Achondroplasia - Market Insights, Epidemiology, and Market Forecast - 2032” report delivers an in-depth understanding of achondroplasia, historical and forecasted epidemiology, as well as the achondroplasia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The achondroplasia market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM achondroplasia market size from 2019 to 2032. The report also covers achondroplasia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered

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

Study Period: 2019-2032

Achondroplasia Understanding and Treatment Algorithm

Achondroplasia Overview

Achondroplasia is a rare genetic bone growth disorder that results in marked short stature (dwarfism) due to a mutation in the ?broblast growth factor receptor 3 (FGFR3) gene. The mutation leads to a gain-of-function of the FGFR3 gene, which slows down the formation of bone in the cartilage of the growth plate and impairs growth in almost all bones in the body.

It is inherited in an autosomal dominant pattern, and the gene is fully penetrant. The risk to offspring of an achondroplastic individual of inheriting a mutated copy of the FGFR3 gene is 50%. When both parents are affected, their offspring have a one in four (25%) chance of having normal stature, a one in two (50%) chance of having achondroplasia (heterozygous), and a one in four (25%) chance of homozygous achondroplasia. Moreover, the homozygous form is usually incompatible with life, resulting in early neonatal death from respiratory insufficiency due to a small thoracic cage and neurologic deficits from cervicomedullary stenosis. However, the condition occurs in over 80% of cases due to sporadic or de novo mutation. Thus, a child with achondroplasia can be born to healthy parents without a family history.

Achondroplasia Diagnosis

It is often diagnosed through physical examination and confirmed with genetic testing. The clinical and radiological features of achondroplasia are well-characterized and diagnostically helpful in neonates and young infants. Prenatal diagnosis is possible through genetic testing, allowing parents to make informed decisions about their child's healthcare. Further, differential diagnosis is done to rule out similar conditions like hypochondroplasia, pseudoachondroplasia, thanatophoric dysplasia, and others.

Further details related to country-based variations are provided in the report.

Achondroplasia Treatment

Management of achondroplasia requires a multidisciplinary approach involving an interprofessional team. There are no curative treatments for achondroplasia; however, treatment aims to relieve complications and provide symptomatic relief.

The current treatment landscape has only one approved therapy in the US and Europe, VOXZOGO (vosoritide), which is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. It is a C-type natriuretic peptide (CNP) analog, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

In Japan, recombinant human growth hormone (r-hGH) therapy and VOXZOGO (vosoritide) are authorized for treating achondroplasia. However, the long-term benefits of r-hGh are controversial, and treatment with growth hormone does not largely affect the height of a person with achondroplasia.

Achondroplasia Epidemiology

As the market is derived using a patient-based model, the achondroplasia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total diagnosed prevalent cases of achondroplasia and gender-specific diagnosed prevalent cases of achondroplasia in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032.

• In 2022, the total diagnosed prevalent cases of achondroplasia were estimated to be approximately 28,613 cases in the 7MM. These cases are expected to decrease by 2032.
• Among the 7MM, the United States accounted for nearly 51% of the total diagnosed prevalent cases of achondroplasia in 2022. These cases are expected to increase during the study period (2019-2032).
• As per the analysis, EU4 and the UK accounted for around 10,923 diagnosed prevalent cases of achondroplasia in 2022. These cases will change during the study period (2019-2032).

Among EU4 and the UK, France accounted for the highest cases of achondroplasia representing nearly 35% of the cases, followed by Germany, while Spain had the least cases in 2022.

According to the publisher's epidemiology model estimates, achondroplasia exhibits a significant female preponderance than males in EU4 and the UK. Of the total cases, nearly 46% were males and 54% were females.

• In 2022, among the 7MM, Japan had the third-highest cases of achondroplasia, contributing approximately 11% to the total cases. Around 44% of males and 56% of females were diagnosed with achondroplasia in 2022.

Achondroplasia Drug Chapters

The drug chapter segment of the achondroplasia report encloses a detailed analysis of achondroplasia-marketed drugs and mid to late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the achondroplasia clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Marketed Drugs

VOXZOGO (vosoritide): BioMarin Pharmaceutical/Chugai Pharmaceutical

VOXZOGO (vosoritide) is a CNP analog that increases linear growth in pediatric patients with achondroplasia with open epiphyses. In achondroplasia patients, the FGFR3 gene, which controls growth, is permanently turned on, hindering the normal formation of bones and resulting in shorter-than-normal bones. VOXZOGO binds to the receptor called natriuretic peptide receptor-B (NPR-B) and antagonizes FGFR3 downstream signaling by inhibiting the extracellular signal-regulated kinases 1 and 2 (ERK1/2) in the mitogen-activated protein kinase (MAPK) pathway and accelerate fibrosarcoma serine/threonine (RAF-1). The drug was approved in 2021 the US and Europe, while in Japan, it was approved in 2022.

The recommended dosage of VOXZOGO is based on the patient's actual body weight and is administered by subcutaneous injection (SC) once daily. The concentration of vosoritide is to be reconstituted before usage, depending upon the body weight, 0.4 mg vial and 0.56 mg vial to 0.8 mg/mL, while the concentration of 1.2 mg vial is reconstituted to 2 mg/mL.

BioMarin received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in Europe to expand the use of VOXZOGO (vosoritide) to treat children aged 4 months and older with achondroplasia late in 2023. However, a final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the fourth quarter of 2023 based on positive results from global Phase II and ongoing extension study.

BioMarin also plans to begin enrollment in the pivotal program with VOXZOGO to treat children with hypochondroplasia. Moreover, they are also preparing to initiate two additional clinical programs in 2024 with VOXZOGO, one in idiopathic short stature and one in genetic short stature conditions (BioMarin, 2023c).

Additionally, VOXZOGO is being tested in Phase I/II for safety and efficacy data of growth deficits in Morquio syndrome (mucopolysaccharidosis IVA/ MPS-IV) and Phase II trial for short stature in Turner Syndrome.

Emerging Drugs

TransCon CNP (navepegritide): Ascendis Pharma

TransCon CNP is a novel long-acting CNP prodrug that comes in a convenient once-weekly SC dose and is intended to offer therapeutic amounts of continuous CNP exposure. It is being developed for the treatment of children with achondroplasia. TransCon CNP is made to effectively shield CNP from neutral endopeptidase degradation in the blood compartment and SC tissue. It also reduces CNP binding to the natriuretic peptide receptor (NPR-B) receptor in the cardiovascular system, preventing hypotension, and minimizes CNP binding to the NPR-C receptor to decrease clearance. Lastly, it releases unmodified CNP, which is small enough to penetrate growth plates effectively.

The drug is undergoing a pivotal Phase II/III ApproaCH trial, with expected results in the second half of 2024. The drug is also being investigated in two other trials, ACcomplisH and AttaCH. The 1-year follow-up data from AComplisH OLE is expected in the fourth quarter of 2023.

Further, the company has filed an IND amendment with the FDA to initiate reACHin, a Phase II trial, designed to evaluate the safety, tolerability, and efficacy of 100 µg/kg of TransCon CNP once weekly for 52 weeks in infants of age 0-2 years with achondroplasia. Moreover, the company expects to submit an IND application in the fourth quarter of 2023 for COACH, a combination trial evaluating TransCon CNP and TransCon hGH in children with achondroplasia.

Note: Detailed emerging therapies assessment will be provided in the final report.

Drug Class Insights

Achondroplasia is a genetic disorder characterized by abnormal bone development, resulting in dwarfism and distinct physical features. It follows an autosomal dominant inheritance pattern, where a single copy of the mutated gene is sufficient to cause the condition. It is associated with increased mortality in early childhood, otolaryngology problems later in childhood, and increased risk of obesity into adulthood. Affected individuals can also develop joint laxity, thoracolumbar kyphosis (TLK), and spinal stenosis that may progress and contribute to morbidity as an adult. Management of achondroplasia involves an interprofessional team approach, and anticipatory care is essential. Statins, CNP analogs, growth hormones, and antihistamines are different classes of medications used to manage complications and symptoms of achondroplasia.

C-type natriuretic peptide (CNP) antagonizes FGFR3 downstream signaling by inhibiting the mitogen-activated protein kinase (MAPK) pathway. VOXZOGO (vosoritide) is the only US FDA-approved CNP analog therapy to increase linear growth in children with achondroplasia and open growth plates.

Besides pharmacological treatment, several surgical options are used to manage the different complications of achondroplasia; macrocephaly, a common complication of achondroplasia in children, is surgically corrected by endoscopic third ventriculostomy, ventricular shunt, or posterior fossa decompression.

Achondroplasia Market Outlook

The adequate management of achondroplasia involves a multidisciplinary approach; due to the different comorbidities of individuals, different care approaches are required; several specialists like pediatrics, endocrinologists, medical geneticists, ENT specialists, orthopedics, neurosurgeons, and others are needed. A few treatment guidelines and recommendations have been developed that aid in the informed evaluation and management of achondroplasia, including the American Academy of Pediatric's Health Supervision for Children with Achondroplasia, International Consensus Statement on the Diagnosis, Multidisciplinary Management, and Lifelong Care of Individuals with Achondroplasia, and Japanese Society for Pediatric Endocrinology's Clinical Practice Guidelines for Achondroplasia.

The current treatment landscape of achondroplasia lacks curative therapies and involves both pharmacological and nonpharmacological options. Different treatment is directed against the specific issues encountered in achondroplasia. Pharmacological intervention includes treatment with CNP analogs and other symptomatic medications for managing complications. Further, growth hormone therapy may be considered as a treatment option for achondroplasia; however, its long-term benefits are controversial and are only authorized in Japan.

The treatment of achondroplasia in Europe and Japan is similar to that of the US. Europe also has only one approved drug, VOXZOGO, which was authorized before its approval in the US. In August 2021, the EMA approved the treatment of children with achondroplasia starting at the age of 2 years until the closure of growth plates, which occurs after puberty. On the contrary, in Japan, the drug was approved in 2022 for treating achondroplasia in children of all ages whose growth plates are not closed. Earlier, recombinant human growth (r-hGH) was the only approved therapy for achondroplasia in Japan. r-hGH therapy was initially approved for Turner syndrome associated with GHD, and later, the indications for r-hGH treatment expanded to include other growth disorders involving normal GH secretion, including short stature in children with chronic renal failure and achondroplasia/hypochondroplasia in 1997.

The current market has been segmented into VOXZOGO (vosoritide) and other therapies, including statins, antihistamines, CNPs, growth hormones, etc. These are the major drugs covered in the forecast model.

Key players Ascendis Pharma's TransCon CNP (navepegritide), QED Therapeutics (BridgeBio) and Novartis' infigratinib (BBP-831/BGJ398), Sanofi‘s SAR442501, and RIBOMIC's RBM-007 are evaluating their lead candidates in different stages of clinical development. They aim to investigate their products to treat achondroplasia.

• The total market size of achondroplasia in the 7MM was approximately USD 197.9 million in 2022 and is projected to increase during the forecast period (2023-2032).
• The market size of achondroplasia in the US was approximately USD 111.8 million in 2022, which is anticipated to increase due to the increasing awareness of the disease and the launch of the emerging therapy.
• The total market size of achondroplasia in EU4 and the UK was calculated to be approximately USD 70.6 million in 2022, which was nearly 36% of the total market revenue for the 7MM.
• According to the publisher's estimates, among EU4 and the UK, France accounted for the highest market with approximately USD 25.1 million in 2022, Germany with approximately USD 17.6 million in the respective year, and Spain accounted for the lowest market in 2022.
• According to the publisher's analysis, in the US, BioMarin Pharmaceutical and Chugai Pharmaceutical's VOXZOGO (vosoritide) had the highest market share among all the therapies in 2022, with a revenue of approximately USD 87.3 million, followed by other therapies that include statins, antihistamines, growth hormone, etc.
• In 2022, the market size of achondroplasia in Japan was nearly 8% of the total market size in the 7MM, with a revenue of approximately USD 15.5 million.
• Among the emerging therapies, Ascendis Pharma's TransCon CNP (navepegritide), a novel long-acting CNP prodrug, is being developed as a convenient, once-weekly SC dose. It effectively shields CNP from neutral endopeptidase degradation in the blood compartment and SC tissues. It is expected to enter the US market by 2025 and is projected to generate a revenue of USD 18.6 million in its launch year; the drug is predicted to peak in the 7th year.

Achondroplasia Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2019-2032. For example, QED Therapeutics (BridgeBio) and Novartis' Infigratinib (BBP-831/BGJ398), a FGFR 1-3-selective tyrosine kinase inhibitor, is expected to enter the US market by 2026 and is projected to have a medium uptake during the forecast period.

Further detailed analysis of emerging therapies drug uptake in the report.

Achondroplasia Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging therapies for achondroplasia.

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 achondroplasia's evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Medical Professionals, Professors, Directors, and Others.

This analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers like the University of Wisconsin School of Medicine and Public Health, University of California, University Medical Center Hamburg-Eppendorf, University of Genova, St. George's University of London, and the University of Tokyo were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or achondroplasia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Physician's view

According to our primary research analysis, though there was a lack of approved therapies before 2021, it was found that several off-label medications, including statins, antihistamines, and growth hormones, were used for the management of complications and symptoms of achondroplasia. A few studies suggested that statins could correct the degraded cartilage in chondrogenically differentiated thanatophoric dysplasia type I and achondroplasia-induced pluripotent stem cells. Further, treatment of achondroplasia model mice with statin led to a significant recovery of bone growth. Meclizine, an antihistamine, is usually used to relieve the symptoms of travel sickness or dizziness and has an additional mechanism to block the ERK1/2-MAPK pathway, which inhibits the activity of FGFR3. It is being used to treat achondroplasia in children; however, its efficacy has not yet been established. Recombinant somatotropin is a symptomatic treatment method for short stature in achondroplasia. It aims to enhance the patients' growth via direct action or through the effect of insulin-like growth factor-1 on chondrocyte proliferation. However, the market regime changed in 2021 after the approval of VOXZOGO (vosoritide), a CNP analog, and is being used to promote endochondral bone growth. However, there is an urgent need for therapies that restore skeletal growth and improve quality of life by significantly reducing the burden of complications.

The current pipeline contains CNP analog, monoclonal antibody, peptide aptamers, and a small molecule that targets different pathways in achondroplasia. The entry of these drugs will provide different options relating to patient-specific needs. Further, the entry of these molecules will end the monopoly of VOXZOGO (vosoritide).

Qualitative Analysis

The publisher performs Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. 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.

Conjoint Analysis analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in achondroplasia trials, efficacy scores are according to the change from baseline in annualized height/growth velocity (cm/year).

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed. It sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers' concerns about budget impact. The high cost of rare disease drugs usually has a limited effect on the budget due to the small number of eligible patients being prescribed the drug. The US FDA has approved several rare disease therapies in recent years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products independently.

The reimbursement challenges related to medical care and treatment for individuals with achondroplasia can be significant as it often requires specialized medical attention, covering the costs of diagnosis, treatment, and ongoing care. Health insurance plans may not fully cover limited coverage of some medical treatments, therapies, and devices specific to achondroplasia. This can result in high out-of-pocket expenses for families seeking the best care for their loved ones. Moreover, it requires specialized care from healthcare providers with expertise. Finding and accessing such specialists may be challenging, and the associated costs may not always be fully reimbursed by insurance.

Many individuals may also require orthopedic interventions, such as limb lengthening or corrective surgeries. These surgeries can be expensive, and reimbursement for these specialized procedures can be an obstacle. Furthermore, GH therapy is used to help children grow taller and costs a lot. In addition, individuals with achondroplasia may benefit from counseling or psychological support to address issues related to self-esteem, body image, and social challenges. Mental health services may have reimbursement limitations.

The recently approved BioMarin's VOXZOGO co-pay assistance program is running in the US. With other initiatives, including VOXZOGO reimbursement in Italy (AIFA), France's HAS Transparency Committee Summary, and others.

VOXZOGO Co-Pay Assistance Program

The program covers up to USD 17,000 annually for eligible families in co-pay assistance. It includes all VOXZOGO (vosoritide) co-pay costs up to the annual maximum. Additionally, eligible families may pay as little as USD 0 for VOXZOGO prescriptions.

The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenarios, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of achondroplasia, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the achondroplasia 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 expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM achondroplasia market.

Achondroplasia Report Insights

• Patient Population
• Therapeutic Approaches
• Achondroplasia Pipeline Analysis
• Achondroplasia Market Size and Trends
• Existing and Future Market Opportunity

Achondroplasia Report Key Strengths

• Ten years Forecast
• The 7MM Coverage
• Achondroplasia Epidemiology Segmentation
• Key Cross Competition
• Attribute Analysis
• Drugs Uptake and Key Market Forecast Assumptions

Achondroplasia Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

Key Questions Answered

Market Insights

• What was the total market size of achondroplasia, the market size of achondroplasia by therapies, and market share (%) distribution in 2019, and what would it look like by 2032?
• What are the contributing factors for this growth?
• How will TransCon CNP (navepegritide) and infigratinib (BBP-831/BGJ398 affect the treatment paradigm of achondroplasia?
• How will VOXZOGO (vosoritide) compete with upcoming products and off-label therapies?
• Which drug is going to be the largest contributor by 2032?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights

• What are the disease risks, burdens, and unmet needs of achondroplasia?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to achondroplasia?
• What is the historical and forecasted achondroplasia patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?
• Out of the countries mentioned above, which country would have the highest diagnosed prevalent achondroplasia population during the forecast period (2023-2032)?
• What factors are contributing to the growth of achondroplasia cases?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the treatment of achondroplasia?
• What are the current clinical and treatment guidelines for treating achondroplasia?
• How many companies are developing therapies for the treatment of achondroplasia?
• How many emerging therapies are in the mid-stage and late stage of development for treating achondroplasia?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• What is the cost burden of current treatment on the patient?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the accessibility issues of approved therapy in the US?
• What is the 7MM historical and forecasted market of achondroplasia?