軟骨無形成症市場 - 市場の洞察、疫学、市場予測：2034年

軟骨無形成症の市場規模は予測期間中に年間平均成長率（CAGR）が上昇すると予測されます。市場収益の成長は、主に遺伝子治療の進歩、認知度と診断率の向上、研究開発投資の増加別もので、治療選択肢の改善と患者の転帰改善につながります。2023年に米国で診断された軟骨無形成症の有病者数は約14,500人に上ると推定されています。

軟骨無形成症は、線維芽細胞増殖因子受容体3（FGFR3）遺伝子の変異により、著しい低身長（小人症）となるまれな遺伝性骨成長障害です。この変異はFGFR3遺伝子の機能獲得につながり、成長板の軟骨における骨の形成を遅らせ、全身のほぼすべての骨の成長を障害します。

常染色体優性遺伝で、遺伝子は完全に浸透しています。しかし、80％以上の症例で、散発性またはde novo変異により発症します。したがって、軟骨無形成症の子供は、家族歴のない健康な両親から生まれることもあります。

軟骨無形成症は、低身長、突出した額（前頭部隆起）と扁平な鼻梁（陥没）を持つ大きな頭部（大頭症）、短い腕と脚、突出した腹部と臀部（背骨の内方への湾曲による）、伸展時に「三叉」または三又になる指を持つ短い手などの特徴的な特徴を有します。さらに、軟骨無形成症のほとんどの女性は自然に約4フィート2インチ（128cm）に成長し、男性は約4フィート4インチ（134cm）に成長します。

2021年11月、米国FDAは軟骨無形成症に対する初の治療を承認しました。VOXZOGO（vosoritide）という薬剤は、この疾患のアンメット・メディカル・ニーズを達成したため、加速承認経路を通じて承認されました。

治療法の進歩にもかかわらず、軟骨無形成症の治療にはいくつかのアンメットニーズが残っています。これらの課題には、効果的な標的治療の開発、専門的治療へのアクセスの改善、早期診断の強化、患者とその家族が直面する身体的・精神的・社会的課題に対処するための包括的支援体制などが含まれます。

当レポートでは、主要7ヶ国における軟骨無形成症市場について調査し、市場の概要とともに、疫学、患者動向、新たな治療法、2034年までの市場規模予測、および医療のアンメットニーズなどを提供しています。

目次

第1章 重要な洞察

第2章 レポートのイントロダクション

第3章 軟骨無形成症市場概要

第4章 軟骨無形成症の疫学と市場の調査手法

第5章 軟骨無形成症のエグゼクティブサマリー

第6章 重要な出来事

第7章 軟骨無形成症の疾患背景と概要

• イントロダクション
• 軟骨無形成症の原因
• 軟骨無形成症のリスク要因
• 軟骨無形成症の兆候と症状
• 診断
• 軟骨無形成症の治療と管理

第8章 疫学と患者人口

• 主な調査結果
• 前提と根拠：主要7ヶ国
• 主要7ヶ国における軟骨無形成症の診断された有病者総数
• 米国
• EU4ヶ国と英国
• 日本

第9章 患者動向

第10章 上市済み治療法

第11章 新たな治療法

第12章 軟骨無形成症：市場分析

• 主な調査結果
• 主要な市場予測の前提条件
• 市場見通し
• コンジョイント分析
• 主要7ヶ国における軟骨無形成症の市場規模
• 主要7ヶ国における軟骨無形成症の治療別市場規模
• 米国
• EU4ヶ国と英国
• 日本

第13章 主要オピニオンリーダーの見解

第14章 SWOT分析

第15章 アンメットニーズ

第16章 市場アクセスと償還

• 米国
• EU4ヶ国と英国
• 日本

第17章 付録

第18章 レポートの調査手法

第19章 DelveInsightのサービス内容

第20章 免責事項

List of Tables

• Table 1: Summary of Achondroplasia Market and Epidemiology (2020-2034)
• Table 2: Key Events
• Table 3: Total Diagnosed Prevalent Cases of Achondroplasia in the 7MM (2020-2034)
• Table 4: Total Diagnosed Prevalent Cases of Achondroplasia in the US (2020-2034)
• Table 5: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in the US (2020-2034)
• Table 6: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in the US (2020-2034)
• Table 7: Total Diagnosed Prevalent Cases of Achondroplasia in EU4 and the UK (2020-2034)
• Table 8: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in EU4 and the UK (2020-2034)
• Table 9: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in EU4 and the UK (2020-2034)
• Table 10: Total Diagnosed Prevalent Cases of Achondroplasia in Germany (2020-2034)
• Table 11: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Germany (2020-2034)
• Table 12: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Germany (2020-2034)
• Table 13: Total Diagnosed Prevalent Cases of Achondroplasia in France (2020-2034)
• Table 14: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in France (2020-2034)
• Table 15: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in France (2020-2034)
• Table 16: Total Diagnosed Prevalent Cases of Achondroplasia in Italy (2020-2034)
• Table 17: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Italy (2020-2034)
• Table 18: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Italy (2020-2034)
• Table 19: Total Diagnosed Prevalent Cases of Achondroplasia in Spain (2020-2034)
• Table 20: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Spain (2020-2034)
• Table 21: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Spain (2020-2034)
• Table 22: Total Diagnosed Prevalent Cases of Achondroplasia in the UK (2020-2034)
• Table 23: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in the UK (2020-2034)
• Table 24: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in the UK (2020-2034)
• Table 25: Total Diagnosed Prevalent Cases of Achondroplasia in Japan (2020-2034)
• Table 26: Gender-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Japan (2020-2034)
• Table 27: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Japan (2020-2034)
• Table 28: Comparison of Emerging Drugs
• Table 29: RBM-007, Clinical Trial Description, 2024
• Table 30: Low dose infigratinib, Clinical Trial Description, 2024
• Table 31: Key Market Forecast Assumptions for RBM-007
• Table 32: Key Market Forecast Assumptions for Low dose infigratinib
• Table 33: Total Achondroplasia Market Size in the 7MM, in USD million (2020-2034)
• Table 34: Achondroplasia Market Size by Therapies in the 7MM, in USD million (2020-2034)
• Table 35: Total Achondroplasia Market Size in the US, in USD million (2020-2034)
• Table 36: Achondroplasia Market Size by Therapies in the US, in USD million (2020-2034)
• Table 37: Total Achondroplasia Market Size in EU4 and the UK, in USD million (2020-2034)
• Table 38: Achondroplasia Market Size by Therapies in EU4 and the UK, in USD million (2020-2034)
• Table 39: Total Achondroplasia Market Size in Germany, in USD million (2020-2034)
• Table 40: Achondroplasia Market Size by Therapies in Germany, in USD million (2020-2034)
• Table 41: Total Achondroplasia Market Size in France, in USD million (2020-2034)
• Table 42: Achondroplasia Market Size by Therapies in France, in USD million (2020-2034)
• Table 43: Total Achondroplasia Market Size in Italy, in USD million (2020-2034)
• Table 44: Achondroplasia Market Size by Therapies in Italy, in USD million (2020-2034)
• Table 45: Total Achondroplasia Market Size in Spain, in USD million (2020-2034)
• Table 46: Achondroplasia Market Size by Therapies in Spain, in USD million (2020-2034)
• Table 47: Total Achondroplasia Market Size in the UK, in USD million (2020-2034)
• Table 48: Achondroplasia Market Size by Therapies in the UK, in USD million (2020-2034)
• Table 49: Total Achondroplasia Market Size in Japan, in USD million (2020-2034)
• Table 50: Achondroplasia Market Size by Therapies in Japan, in USD million (2020-2034)

List of Figures

• Figure 1: Total Diagnosed Prevalent Cases of Achondroplasia in the 7MM (2020-2034)
• Figure 2: Total Diagnosed Prevalent Cases of Achondroplasia in the US (2020-2034)
• Figure 3: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in the US (2020-2034)
• Figure 4: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in the US (2020-2034)
• Figure 5: Total Diagnosed Prevalent Cases of Achondroplasia in EU4 and the UK (2020-2034)
• Figure 6: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in EU4 and the UK (2020-2034)
• Figure 7: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in EU4 and the UK (2020-2034)
• Figure 8: Total Diagnosed Prevalent Cases of Achondroplasia in Germany (2020-2034)
• Figure 9: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in Germany (2020-2034)
• Figure 10: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Germany (2020-2034)
• Figure 11: Total Diagnosed Prevalent Cases of Achondroplasia in France (2020-2034)
• Figure 12: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in France (2020-2034)
• Figure 13: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in France (2020-2034)
• Figure 14: Total Diagnosed Prevalent Cases of Achondroplasia in Italy (2020-2034)
• Figure 15: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in Italy (2020-2034)
• Figure 16: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Italy (2020-2034)
• Figure 17: Total Diagnosed Prevalent Cases of Achondroplasia in Spain (2020-2034)
• Figure 18: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in Spain (2020-2034)
• Figure 19: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Spain (2020-2034)
• Figure 20: Total Diagnosed Prevalent Cases of Achondroplasia in the UK (2020-2034)
• Figure 21: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in the UK (2020-2034)
• Figure 22: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in the UK (2020-2034)
• Figure 23: Total Diagnosed Prevalent Cases of Achondroplasia in Japan (2020-2034)
• Figure 24: Gender-specific Diagnosed Prevalent Cases of Achondroplasia in the Japan (2020-2034)
• Figure 25: Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia in Japan (2020-2034)
• Figure 26: Patient Journey
• Figure 27: Total Achondroplasia Market Size in the 7MM, in USD million (2020-2034)
• Figure 28: Achondroplasia Market Size by Therapies in the 7MM, in USD million (2020-2034)
• Figure 29: Total Achondroplasia Market Size in the US, in USD million (2020-2034)
• Figure 30: Achondroplasia Market Size by Therapies in the US, in USD million (2020-2034)
• Figure 31: Total Achondroplasia Market Size in EU4 and the UK, in USD million (2020-2034)
• Figure 32: Achondroplasia Market Size by Therapies in EU4 and the UK, in USD million (2020-2034)
• Figure 33: Total Achondroplasia Market Size in Germany, in USD million (2020-2034)
• Figure 34: Achondroplasia Market Size by Therapies in Germany, in USD million (2020-2034)
• Figure 35: Total Achondroplasia Market Size in France, in USD million (2020-2034)
• Figure 36: Achondroplasia Market Size by Therapies in France, in USD million (2020-2034)
• Figure 37: Total Achondroplasia Market Size in Italy, in USD million (2020-2034)
• Figure 38: Achondroplasia Market Size by Therapies in Italy, in USD million (2020-2034)
• Figure 39: Total Achondroplasia Market Size in Spain, in USD million (2020-2034)
• Figure 40: Achondroplasia Market Size by Therapies in Spain, in USD million (2020-2034)
• Figure 41: Total Achondroplasia Market Size in the UK, in USD million (2020-2034)
• Figure 42: Achondroplasia Market Size by Therapies in the UK, in USD million (2020-2034)
• Figure 43: Total Achondroplasia Market Size in Japan, in USD million (2020-2034)
• Figure 44: Achondroplasia Market Size by Therapies in Japan, in USD million (2020-2034)
• Figure 45: SWOT Analysis
• Figure 46: Unmet Needs

Key Highlights:

• The achondroplasia market size is projected to experience a rise in Compound Annual Growth Rate (CAGR) during the forecast period. Market revenue growth is mainly due to advancements in genetic therapies, increased awareness and diagnosis rates, and a rise in research and development investments, which will lead to improved treatment options and better patient outcomes.
• DelveInsight estimates that the US accounted for nearly 14,500 diagnosed prevalent cases of achondroplasia in 2023.
• The dynamics of the Achondroplasia therapeutics market are anticipated to change in the coming years owing to the improvement in novel treatment options, which will increase healthcare spending worldwide. Key players in the treatment and prevention landscape are BioMarin Pharmaceuticals, Tyra Biosciences, Inc., RIBOMIC Inc., Bridge Bio Inc., Sanofi, and others.
• In November 2021, the US FDA approved the first treatment for Achondroplasia. The drug VOXZOGO (vosoritide) was approved through an accelerated approval pathway because it accomplished the disease's unmet medical need.
• Despite advancements in treatment options, several unmet needs persist in the management of Achondroplasia. These include the development of effective, targeted therapies, improved access to specialized care, enhanced early diagnosis, and comprehensive support systems to address the physical, emotional, and social challenges faced by patients and their families. DelveInsight's comprehensive report titled "Achondroplasia - Market Insights, Epidemiology, and Market Forecast - 2034" offers a detailed analysis of Achondroplasia. The report presents historical and projected epidemiological data covering Total Diagnosed Prevalent Cases of Achondroplasia, Age-specific Diagnosed Prevalent Cases of Achondroplasia, and Gender-specific Diagnosed Prevalent Cases of Achondroplasia In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.
• The Achondroplasia market report analyzes the existing treatment practices and unmet medical requirements in Achondroplasia. It evaluates the Achondroplasia market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.
• Achondroplasia prevalence is estimated at 1 in 15,000 to 40,000 live births, making it one of the most common skeletal dysplasias.

Achondroplasia Treatment Market

Achondroplasia is a rare genetic bone growth disorder that results in marked short stature (dwarfism) due to a mutation in the fibroblast 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. 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.

The disorder is characterized by distinctive features including short stature, a considerable head (macrocephaly) with a prominent forehead (frontal bossing) and flat (depressed) nasal bridge, short arms, and legs; prominent abdomen and buttocks (due to an inward curve of the spine), and short hands with fingers that assume a "trident" or three-pronged position during extension. Further, most females with achondroplasia naturally grow to approximately 4 feet 2 inches (128 cm), while males to about 4 feet 4 inches (134 cm).

Achondroplasia Diagnosis and Treatment Algorithm

Achondroplasia is typically diagnosed through clinical evaluation, genetic testing, and radiographic imaging. Clinicians assess physical characteristics such as disproportionate short stature, shortened limbs, and distinctive facial features. Prenatal ultrasound can often detect signs of Achondroplasia by the second trimester, identifying abnormalities in limb length and head size. Genetic testing confirms the diagnosis by identifying mutations in the FGFR3 gene, responsible for this condition. Postnatal diagnosis involves detailed physical examination and X-rays to observe characteristic skeletal abnormalities, such as shortening of the long bones, spinal stenosis, and specific features of the pelvis and skull.

While there is no cure for Achondroplasia, treatment focuses on managing symptoms and complications. Multidisciplinary care is essential, involving pediatricians, geneticists, orthopedists, neurologists, and physical therapists. Growth hormone therapy has shown some effectiveness in increasing height, though it is not a standard treatment. Surgical interventions may be necessary for severe skeletal abnormalities, such as limb lengthening procedures or surgeries to address spinal stenosis and other complications. Supportive therapies, including physical therapy and occupational therapy, help improve mobility and daily functioning. Additionally, new treatments targeting the underlying genetic mutation, such as C-type natriuretic peptide analogs, are under investigation and show promise in potentially altering disease progression. Regular monitoring and early intervention are crucial to optimize outcomes and improve the quality of life for individuals with Achondroplasia.

Achondroplasia Epidemiology

The epidemiology section on the Achondroplasia epidemiology segment offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the data with relevant tables and graphs, offering a clear and concise view of the incidence of Achondroplasia. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

Key Findings

• The total diagnosed prevalent cases of Achondroplasia in Japan and the US were ~18,000 in 2023, and they are expected to rise in the future.
• In the US, achondroplasia affects a slightly higher percentage of females (51.2%) compared to males (48.8%), possibly due to genetic or biological factors contributing to the marginally more significant occurrence among females.
• Japan had nearly 3000 total diagnosed prevalent cases of Achondroplasia in 2023.
• According to the epidemiology model, the age-specific cases are divided into nine groups: 0-4, 5-10, 11-15, 16-20, 21-30, 31-40, 41-50, 51-60, and >60. Based on DelveInsight's estimates, the people in the age group 5-10 years of age were affected the most in the US, accounting for approximately 4000 cases in 2023.
• Achondroplasia prevalence is estimated at 1 in 15,000 to 40,000 live births, making it one of the most common skeletal dysplasias.

Achondroplasia Market Outlook

Achondroplasia, a common form of dwarfism caused by a genetic mutation, has historically been managed through supportive care and surgical interventions. However, recent advancements in medical science have introduced promising pharmacological treatments. Traditional management includes orthopedic surgeries to correct bone deformities, alleviate spinal stenosis, and enhance mobility. These procedures, though effective in addressing specific complications, do not alter the underlying growth deficiency.

The introduction of pharmacological treatments marks a significant shift in the management of achondroplasia. Vosoritide, a daily injectable drug, has emerged as a breakthrough therapy. Vosoritide is an analog of C-type natriuretic peptide (CNP) that targets the overactive fibroblast growth factor receptor 3 (FGFR3) pathway, which is responsible for inhibiting bone growth in individuals with achondroplasia. Clinical trials have demonstrated that vosoritide significantly improves annual growth velocity in children with achondroplasia, offering a potential means to enhance final adult height and reduce complications associated with disproportionate growth.

Another promising treatment under investigation is transition CNP, a prodrug designed to provide sustained CNP release, which aims to reduce the frequency of dosing while maintaining efficacy. Early studies indicate that transCon CNP has the potential to improve growth outcomes and quality of life for patients with achondroplasia.

Beyond these pharmacological approaches, gene therapy is an area of active research. Scientists are exploring methods to correct the underlying FGFR3 mutation at the genetic level, potentially offering a one-time cure for achondroplasia. Although still in the experimental stages, gene therapy holds immense promise for transforming the future landscape of achondroplasia treatment market.

Overall, the evolution of treatment options for achondroplasia from purely supportive measures to innovative pharmacological and genetic therapies offers new hope for improving the lives of individuals with this condition. These advancements not only target the symptoms but also address the root cause of achondroplasia, paving the way for more effective and comprehensive management.

With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, the Achondroplasia therapeutics market in the 7MM is projected to grow significantly during the study period 2020-2034.

Achondroplasia Drug Chapters

Marketed Achondroplasia Drugs

VOXZOGO (vosoritide): BioMarin Pharmaceuticals

VOXZOGO (vosoritide) by BioMarin Pharmaceuticals is a groundbreaking treatment for achondroplasia. It works by mimicking C-type natriuretic peptide (CNP), which counteracts the overactive FGFR3 pathway that impairs bone growth. Administered via daily subcutaneous injections, Voxzogo enhances growth velocity in children with achondroplasia. It received FDA approval in November 2021 and EMA approval in August 2021, making it the first pharmacological treatment approved explicitly for this genetic condition.

Emerging Achondroplasia Drugs

RBM-007: RIBOMIC Inc.

RBM-007, developed by RIBOMIC Inc., is an RNA aptamer targeting fibroblast growth factor 2 (FGF2) for the treatment of achondroplasia. Its mechanism of action involves inhibiting FGF2, thereby promoting bone growth and reducing the effects of the FGFR3 mutation responsible for achondroplasia. RBM-007 is administered via subcutaneous injection. It is currently in Phase IIa clinical trials, assessing its safety and preliminary efficacy in affected individuals.

SAR-442501: Sanofi

SAR-442501, developed by Sanofi, is a novel treatment for achondroplasia. It functions as a selective antagonist of the fibroblast growth factor receptor 3 (FGFR3), which is implicated in inhibiting bone growth in achondroplasia. By blocking this receptor, SAR-442501 aims to enhance skeletal growth and improve height outcomes. The drug is administered via subcutaneous injection. The upreACH Phase II study of SAR442501 in pediatric patients with achondroplasia will remain ongoing but is not expected to advance further.

Low dose infigratinib: BridgeBio Inc.

Infigratinib is an oral small molecule in development for the treatment of FGFR-driven conditions, including achondroplasia, a bone growth disorder in children. Over-activating FGFR3 mutations drive downstream MAPK and STAT1 signaling that aberrates growth plate development, thereby causing disproportionate short stature and severe health complications stemming from cranial and spinal issues. Low-dose infigratinib has the potential to help children due to its direct inhibition of the mutant FGFR3 receptor. BridgeBio initiated PROPEL 3, a Phase III clinical trial studying the efficacy and safety of infigratinib in children with achondroplasia. Both the US FDA and the EU EMA indicated the trial design for PROPEL 3 would be acceptable as a registrational study to support a marketing application for the treatment of children with achondroplasia.

Achondroplasia Market Segmentation

DelveInsight's Achondroplasia Market Insights, Epidemiology, and Market Forecast-2034 report provides a detailed outlook of the current and future Achondroplasia market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Achondroplasia Market Size by Countries

The Achondroplasia market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2023, the United States held a significant share of the overall 7MM (Seven Major Markets) Achondroplasia market, primarily attributed to the elevated cost of the available treatments and the increasing sensitivity toward pneumococcal infections. This dominance is projected to persist, especially with the potential early introduction of new products.

Achondroplasia Market Size by Therapies

Achondroplasia Market Size by Therapies is categorized into current and emerging markets for the study period 2020-2034. One of the emerging drugs anticipated to launch during the forecast period is low-dose infigratinib, which is in BridgeBio Inc.'s developmental pipeline.

Achondroplasia Drugs Uptake

The Achondroplasia drugs market is growing due to rising prevalence, with increasing demand for treatments targeting the genetic disorder's symptoms. This section focuses on the sales uptake of potential Achondroplasia drugs that have recently been launched or are anticipated to be launched in the Achondroplasia market between 2020 and 2034. It estimates the market penetration of Achondroplasia drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the Achondroplasia market.

Achondroplasia Market Access and Reimbursement

DelveInsight's 'Achondroplasia Market Insights, Epidemiology, and Market Forecast-2034' report provides a descriptive overview of the market access and reimbursement scenario for Achondroplasia.

This section includes a detailed analysis of each therapy's country-wise healthcare system, enlightening market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current Achondroplasia market trends and fill gaps in secondary findings, we interview KOLs and SMEs working in the Achondroplasia domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns for Achondroplasia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market of Achondroplasia unmet needs.

Achondroplasia: KOL Insights

DelveInsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as Greenberg Center for Skeletal Dysplasias, Department of Genetic Medicine, Johns Hopkins University, Baltimore, USA; McGovern Medical School, University of Texas Health, Houston, TX, USA; Department of Pediatrics, IRCCS Istituto Giannina Gaslini, Genoa, Italy; Instituto de Investigacion Biomedica de Malaga-IBIMA, Hospital Universitario Virgen de la Victoria, Malaga, Spain; Department of Medical Genetics, Tokyo Medical University, Tokyo Japan; and others.

"Achondroplasia occurs in approximately 1 in 25,000 births globally. Although treatments focus on managing symptoms, there is a need for research into therapies that address the genetic basis of the condition."

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the Achondroplasia therapeutics market, utilizing various Competitive Intelligence tools such as SWOT analysis, Conjoint Analysis, and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

The emerging Achondroplasia therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the Achondroplasia market.

Achondroplasia Pipeline Development Activities

The report offers an analysis of different Achondroplasia clincial trials within Phase II and III stages and examines companies involved in developing targeted therapeutics for Achondroplasia. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, patent details, and other aspects of emerging Achondroplasia.

Achondroplasia Market Report Insights

• Achondroplasia Patient Population
• Therapeutic Approaches
• Achondroplasia Pipeline Analysis
• Achondroplasia Market Size and Trends
• Achondroplasia Market Opportunities
• Impact of Upcoming Therapies

Achondroplasia Market Report Key Strengths

• 11 years Forecast
• The 7MM Coverage
• Achondroplasia Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Achondroplasia Market
• Achondroplasia Drugs Uptake

Achondroplasia Market Report Assessment

• Achondroplasia Current Treatment Practices
• Achondroplasia Unmet Needs
• Achondroplasia Pipeline Product Profiles
• Achondroplasia Market Attractiveness
• Achondroplasia Market Drivers
• Achondroplasia Market Barriers

Key Questions:

• How common is Achondroplasia?
• What are the key findings of Achondroplasia epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020-2034)?
• What are the currently available treatments for Achondroplasia?
• What are the disease risk, burden, and unmet needs of Achondroplasia?
• At what CAGR is the Achondroplasia market and its epidemiology is expected to grow in the 7MM during the forecast period (2024-2034)?
• How would the unmet needs impact the Achondroplasia market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of Achondroplasia in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• Which country will have the highest number of patients among EU4 and the UK during the forecast period (2020-2034)?
• How many companies are currently developing therapies for the treatment of Achondroplasia?

Table of Contents

1. Key Insights

2. Report Introduction

3. Achondroplasia Market Overview

• 3.1. Market Share (%) Distribution of Achondroplasia by Class in 2020
• 3.2. Market Share (%) Distribution of Achondroplasia by Class in 2034

4. Methodology of Achondroplasia Epidemiology and Market

5. Executive Summary of Achondroplasia

6. Key Events

7. Disease Background and Overview of Achondroplasia

• 7.1. Introduction
• 7.2. Causes of Achondroplasia
• 7.3. Risk factors of Achondroplasia
• 7.4. Signs and Symptoms of Achondroplasia
• 7.5. Diagnosis
  • 7.5.1. Diagnostic Algorithm
  • 7.5.2. Diagnostic Guidelines
• 7.6. Treatment and Management of Achondroplasia
  • 7.6.1. Treatment Algorithm
  • 7.6.2. Treatment and Management Guidelines

8. Epidemiology and Patient Population

• 8.1. Key Findings
• 8.2. Assumptions and Rationale: The 7MM
  • 8.2.1. Total Diagnosed Prevalent Cases of Achondroplasia
  • 8.2.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
  • 8.2.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
• 8.3. Total Diagnosed Prevalent Cases of Achondroplasia in the 7MM
• 8.4. The United States
  • 8.4.1. Total Diagnosed Prevalent Cases of Achondroplasia
  • 8.4.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
  • 8.4.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
• 8.5. EU4 and the UK
  • 8.5.1. Germany
    • 8.5.1.1. Total Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.1.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.1.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
  • 8.5.2. France
    • 8.5.2.1. Total Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.2.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.2.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
  • 8.5.3. Italy
    • 8.5.3.1. Total Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.3.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.3.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
  • 8.5.4. Spain
    • 8.5.4.1. Total Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.4.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.4.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
  • 8.5.5. The United Kingdom
    • 8.5.5.1. Total Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.5.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
    • 8.5.5.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia
• 8.6. Japan
  • 8.6.1. Total Diagnosed Prevalent Cases of Achondroplasia
  • 8.6.2. Gender-specific Diagnosed Prevalent Cases of Achondroplasia
  • 8.6.3. Age-specific Cases Diagnosed Prevalent Cases of Achondroplasia

9. Patient Journey

10. Marketed Therapies

• 10.1. VOXZOGO (vosoritide): BioMarin Pharmaceuticals
  • 10.1.1. Product Description
  • 10.1.2. Other Developmental Activities
  • 10.1.3. Clinical Development
  • 10.1.4. Clinical Trials Information
  • 10.1.5. Safety and Efficacy
  • 10.1.6. Product Profile

11. Emerging Therapies

• 11.1. Key Cross Competition
• 11.2. RBM-007: RIBOMIC Inc.
  • 11.2.1. Product Description
  • 11.2.2. Other Developmental Activities
  • 11.2.3. Clinical Development
  • 11.2.4. Clinical Trials Information
  • 11.2.5. Safety and Efficacy
  • 11.2.6. Product Profile
  • 11.2.7. Analysts' Views
• 11.3. Low dose infigratinib: BridgeBio Inc.
  • 11.3.1. Product Description
  • 11.3.2. Other Developmental Activities
  • 11.3.3. Clinical Development
  • 11.3.4. Clinical Trials Information
  • 11.3.5. Safety and Efficacy
  • 11.3.6. Product Profile
  • 11.3.7. Analysts' Views

12. Achondroplasia: Market Analysis

• 12.1. Key Findings
• 12.2. Key Market Forecast Assumptions
• 12.3. Market Outlook
• 12.4. Conjoint Analysis
• 12.5. Market Size of Achondroplasia in the 7MM
• 12.6. Total Market Size of Achondroplasia by Therapies in the 7MM
• 12.7. The US
  • 12.7.1. Total Market Size of Achondroplasia
  • 12.7.2. Market Size of Achondroplasia by Therapies in the US
• 12.8. EU4 and the UK
  • 12.8.1. Germany
    • 12.8.1.1. Total Market Size of Achondroplasia
    • 12.8.1.2. Market Size of Achondroplasia by Therapies
  • 12.8.2. France
    • 12.8.2.1. Total Market Size of Achondroplasia
    • 12.8.2.2. Market Size of Achondroplasia by Therapies
  • 12.8.3. Italy
    • 12.8.3.1. Total Market Size of Achondroplasia
    • 12.8.3.2. Market Size of Achondroplasia by Therapies
  • 12.8.4. Spain
    • 12.8.4.1. Total Market Size of Achondroplasia
    • 12.8.4.2. Market Size of Achondroplasia by Therapies
  • 12.8.5. The UK
    • 12.8.5.1. Total Market Size of Achondroplasia
    • 12.8.5.2. Market Size of Achondroplasia by Therapies
• 12.9. Japan
  • 12.9.1. Total Market Size of Achondroplasia
  • 12.9.2. Market Size of Achondroplasia by Therapies in Japan

13. Key Opinion Leaders' Views

14. SWOT Analysis

15. Unmet needs

16. Market Access and Reimbursement

• 16.1. The United States
  • 16.1.1. Centre for Medicare and Medicaid Services (CMS)
• 16.2. In EU4 and the UK
  • 16.2.1. Germany
  • 16.2.2. France
  • 16.2.3. Italy
  • 16.2.4. Spain
  • 16.2.5. The United Kingdom
• 16.3. Japan
  • 16.3.1. MHLW

17. Appendix

• 17.1. Bibliography
• 17.2. Acronyms and Abbreviations

18. Report Methodology

19. DelveInsight Capabilities

20. Disclaimer