查看购物车
  • Traditional Chinese
  • English
  • Japanese
  • Korean
苯丙酮尿症治疗的全球市场-2022-2029
市场调查报告书
商品编码
1140769

苯丙酮尿症治疗的全球市场-2022-2029

Global Phenylketonuria Treatment Market - 2022-2029
出版日期: | 出版商: DataM Intelligence | 英文 200 Pages | 商品交期: 约2个工作天内

价格

※ 本网页内容可能与最新版本有所差异。详细情况请与我们联繫。

简介目录

市场概况

据估计，苯丙酮尿症治疗市场在预测期内（2022-2029 年）的复合年增长率将达到 9.46%。

苯丙酮尿症是一种罕见的遗传性疾病，其中氨基酸苯丙氨酸在体内积累。PKU 是由有助于产生苯丙氨酸降解□的基因突变引起的。血液中过量的苯丙氨酸会导致癫痫发作、精神疾病和其他未经治疗的疾病。PKU 无法治愈，但药物可以预防智力障碍和其他健康问题。PKU 的治疗包括坚持限制含有苯丙氨酸的食物的 PKU 饮食。

市场动态

由于对苯丙酮尿症新干预措施的研究增加等多种因素，全球苯丙酮尿症治疗市场正在增长。

市场参与者进行的治疗苯丙酮尿症的临床试验预计将推动市场增长。

BioMarin Pharmaceutical Inc. 将研究 BMN 307（一种腺相关病毒载体介导的人苯丙氨酸羟化□基因转移）在血浆 Phe 水平 >600 mol/L 的苯丙酮尿症患者中的安全性和有效性。1/2 开放标籤，进行了剂量递增研究。PTC Therapeutics 还通过测量血液 Phe 水平从基线到第 5 周和第 6 週的变化来评估 PTC923 降低苯丙酮尿症患者血液苯丙氨酸水平的效果（即，每次治疗还进行了临床试验以评估剂量（平均值2 周双盲治疗），证实 PTC923 可有效降低苯丙酮尿症患者的血 Phe 水平。因此，根据上述描述，预计将在预测期内推动市场。

低意识和高成本预计将阻碍全球苯丙酮尿症治疗市场的增长。

缺乏对罕见病的认识、PKU 治疗的高成本、不规则的报销政策以及缺乏对治疗方案的依从性是预计在预测期内阻碍市场增长的一些因素。

行业分析

全球苯丙酮尿症治疗市场报告根据波特五力、创新、新产品发布、定价和管道分析等各种行业因素对市场进行了深入分析。

COVID-19 的影响分析。

COVID-19 大流行对医疗保健系统和市场产生了适度的影响。苯丙酮尿症 (PKU) 的神经认知结果和预后与终生苯丙氨酸 (Phe) 水平和治疗依从性直接相关。PKU 患者不得不调整饮食以适应新环境。年轻患者可能接触较少（饮食严格遵循饮食计划，无论情况如何），但青少年和成年患者表现出更好的代谢控制，从而履行了他们留在家中的义务。强烈反映。这可归因于多种因素，包括更容易获得远程咨询。儘管如此，我们的研究结果表明流行病和环境如何显着影响 PKU 治疗依从性，以及消除距离障碍如何改善和优化代谢依从性。综上所述，市场受到影响，预计随着经济活动的恢復，市场将出现早期拉动。

细分分析

在苯丙酮尿症治疗市场中，预计膳食补充剂部分将在整个预测期间（2022-2029 年）占据最大的市场份额。

膳食补充剂市场预计将在 2021 年占据主导地位。这部分的增长是有益的，因为饮食限制了含有苯丙氨酸的食物。这意味着饮食必须低蛋白质，PKU 患者与医生和营养师密切合作，在减少苯丙氨酸摄入的同时保持健康的食物平衡。您还应该跟踪当天饮食中苯丙氨酸的含量，并了解您的苯丙氨酸水平。此外，患有 PKU 的儿童必须服用 PKU 配方以保持他们获得足够的蛋白质。含有除苯丙氨酸外的所有必需氨基酸。健康食品商店还提供低蛋白、对 PKU 友好的食品。例如，患有 PKU 的婴儿是母乳喂养的。一般来说，您还应该服用一种特殊的粉状配方，称为洛芬酸。一旦婴儿长大到可以吃固体食物，就应该避免富含蛋白质的食物。因此，从上述描述来看，这种市场细分预计将在预测期内占最大的市场份额。

区域分析

北美地区占全球苯丙酮尿症治疗市场最大的市场份额。

到 2021 年，北美将占据最高的收入份额。苯丙酮尿症患病率的上升和苯丙酮尿症治疗饮食的高度采用、政府对正在进行的临床研究的支持以及该地区市场参与者推出的产品预计将在预测期内增加。例如，据说 PKU 会影响美国 10,000 到 15,000 人中的 1 人。大多数 PKU 在出生后不久通过新生儿筛查被诊断出来，并立即开始治疗。美国在治疗苯丙酮尿症的饮食流行方面也处于领先地位。该国的患者服用许多 PKU 物质，包括必需和非必需氨基酸。

Palynziq (Pegvariase-pqpz) 也被 FDA 批准用于患有 PKU 的成年人。Palynziq 是一种可注射的□疗法，适用于儘管现有药物疗法但血液苯丙氨酸水平不受控制的患者。BioMarin Pharmaceutical Inc. 生产 Palynziq。因此，从上述陈述来看，预计北美地区将在预测期内占据最大的市场份额。

竞争格局

苯丙酮尿症治疗市场的主要参与者是 BioMarin、SOM Innovation Biotech SL、Homology Medicines, Inc.、Par Pharmaceutical、PTC Therapeutics 和 Synlogic, Inc.。主要参与者正在采取各种策略，例如产品发布、併购、合作伙伴和联盟，以促进全球苯丙酮尿症治疗市场的增长。例如，2021 年 12 月 16 日，BioMarin 和 Skyline Therapeutics 结成战略联盟，开发针对心血管疾病的新型基因疗法。此外，2022 年 6 月 13 日，美国食品药品监督管理局 (FDA) 批准了 Homology Medicines, Inc. 的 pheNIX 基因治疗临床试验，其研究药物 HMI-102 用于成人苯丙酮尿症 (PKU)。抓住。

BioMarin Pharmaceutical Inc.

概述

BioMarin, Inc. 是一家美国生物技术公司，总部位于加利福尼亚州圣拉斐尔。我们在世界各地设有办事处，包括美国、南美、亚洲和欧洲。□替代药物是 BioMarin 的主要业务和研究重点 (ERT)。通过製造 Laronidase，BioMarin 成为第一家提供 I 型粘多醣贮积症 (MPS I) 治疗的公司（Aldurazyme，由 Genzyme Corporation 商业化）。它也是第一家开发治疗苯丙酮尿症（PKU）药物的公司。

产品介绍

KUVAN（沙丙蝶呤二盐酸盐）：KUVAN（沙丙蝶呤二盐酸盐）口服片剂和口服溶液粉可降低成人和 1 个月及以上患有苯丙酮尿症 (PKU) 的儿童的血液 Phe 水平。它是一种处方药，用于与限制 Phe 的饮食一起使用。

全球苯丙酮尿症治疗市场报告将提供对大约 40 多个市场数据表、45 多个数字和 200 页（大约）的访问。

目录

  • 调查方法
  • 调查目的和范围

第二章市场定义和概述

第三章执行摘要

第四章市场动态

  • 市场影响因素
    • 促进者
      • 市场参与者进行的治疗苯丙酮尿症的临床试验预计将推动市场增长。
    • 抑製剂
      • 对罕见病的低认识预计将阻碍市场增长
      • 治疗费用高
    • 机会
    • 影响分析

第五章行业分析

  • 波特五力分析
  • 供应链分析
  • 监管分析

第 6 章 COVID-19 分析

  • COVID-19 的市场分析
    • COVID-19 之前的市场情景
    • COVID-19 的当前市场情景
    • COVID-19 后或未来情景
  • COVID-19 中的价格动态
  • 供需谱
  • 大流行期间与市场相关的政府努力
  • 製造商的战略举措
  • 概括

第 7 章 按类型

  • 高苯丙氨酸血症
  • 轻度北大
  • 中等或突变
  • 经典北大
  • 其他

第 8 章 按治疗类型

  • 药物治疗
    • 关
    • 聚乙二醇化□
    • 帕林泽克
    • CNSA-001
    • SYNB1618
    • 其他
  • 膳食补充剂
  • 其他

第 9 章 按行政途径

  • 口服
  • 肠外

第10章按地区

  • 北美
    • 美国
    • 加拿大
    • 墨西哥
  • 欧洲
    • 德国
    • 英国
    • 法国
    • 意大利
    • 西班牙
    • 欧洲其他地区
  • 南美洲
    • 巴西
    • 阿根廷
    • 其他南美地区
  • 亚太地区
    • 中国
    • 印度
    • 日本
    • 澳大利亚
    • 亚太其他地区
  • 中东/非洲

第 11 章 竞争格局

  • 主要发展和战略
  • 公司股份分析
  • 产品基准

第十二章公司简介

  • BioMarin
    • 公司简介
    • 产品组合和描述
    • 主要亮点
    • 财务摘要
  • SOM Innovation Biotech SL
  • Homology Medicines, Inc.
  • Par Pharmaceutical
  • PTC Therapeutics
  • Synlogic, Inc.

第 13 章 数据管理

简介目录
Product Code: DMPH2150

Market Overview

Phenylketonuria Treatment Market is estimated to reach at a CAGR of 9.46% during the forecast period (2022-2029).

Phenylketonuria is a rare hereditary condition in which the body accumulates the amino acid phenylalanine. PKU is caused by a mutation in a gene that aids in the production of the phenylalanine-degrading enzyme. Seizures, mental illnesses, and other untreated disorders can result from an excess of phenylalanine in the blood. Although there is no cure for PKU, medication can help to prevent intellectual deficits and other health issues. PKU is treated by adhering to a PKU diet that restricts foods containing phenylalanine.

Market Dynamics

The global phenylketonuria treatment market is growing due to several factors, such as the rising research on new interventions for phenylketonuria.

Clinical trials conducted by the market players to treat phenylketonuria are expected to drive market growth.

BioMarin Pharmaceutical Inc. conducted a Phase 1/2 Open-Label, Dose Escalation research in Phenylketonuria patients with Plasma Phe Levels > 600 mol/L to investigate the safety and efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase. Moreover, PTC Therapeutics also conducted a clinical trial to assess the efficacy of PTC923 in lowering blood phenylalanine (Phe) levels in Phenylketonuria patients, as evaluated by the mean change in blood Phe levels from baseline to Weeks 5 and 6. (that is, the average of each respective treatment dose 2-week period of double-blind treatment). Thus, the market is expected to drive in the forecast period from the above statements.

The lack of awareness and high cost are expected to hinder global phenylketonuria treatment market growth.

Lack of awareness regarding the rare disease, the high cost of PKU treatment, irregular reimbursement policies, and lack of treatment regimen compliance are some factors expected to hamper the market growth in the forecast period.

Industry analysis.

The global phenylketonuria treatment market report will provide an in-depth analysis of the market based on various industry factors such as porter`s five forces, innovations, new product launches, pricing, and pipeline analysis.

COVID-19 Impact Analysis.

The COVID-19 pandemic has moderately impacted healthcare systems and the market. Neurocognitive outcome and phenylketonuria (PKU) prognosis are directly related to lifelong phenylalanine (Phe) levels and treatment adherence. PKU patients had to adjust their dietary restrictions to the new environment. While younger patients may have been less exposed (meals strictly according to diet plan regardless of setting), adolescent and adult patients strongly reflected the obligation to stay home by showing better metabolic control. Multiple factors could have contributed to this, including the availability of teleconsultancy, which allowed for easier connections. Still, findings show how the pandemic and the environment can significantly impact PKU treatment adherence and how removing distance barriers can improve and optimize metabolic compliance. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of economic activities.

Segment Analysis

The dietary supplement segment is expected to hold the largest market share in the phenylketonuria treatment market throughout the forecast period (2022-2029).

The dietary supplement segment is expected to dominate in 2021. The segment's growth is beneficial because diet limits foods with phenylalanine. It means the diet must be low in protein, and people with PKU work closely with a doctor or nutritionist to maintain a healthy food balance while decreasing their phenylalanine intake. They must also keep track of the amount of phenylalanine in the meals they eat throughout the day to track their phenylalanine levels. Moreover, Children with PKU must take PKU formula to ensure they continue obtaining adequate protein. Except for phenylalanine, it contains all of the essential amino acids. At specialty health stores, you can also get low-protein, PKU-friendly foods. For instance, Breast milk is given to infants with PKU. They generally need to take a particular formula called Lofenalac as well. When your infant is old enough to eat solid foods, you should avoid giving them protein-rich foods. Thus, the market segment is expected to hold the largest market share in the forecast period from the above statements.

Geographical Analysis

The North American region holds the largest market share in the global phenylketonuria treatment market.

In 2021, North America accounted for the highest revenue share. The increasing prevalence of phenylketonuria and higher adoption of phenylketonuria treatment-based diets, government support for ongoing clinical research and product launches by the market players in the region are expected to boost in the forecast period. For instance, PKU affects 1 in 10,000 to 15,000 babies in the United States. Most instances of PKU are diagnosed by newborn screening shortly after birth, and treatment is started immediately. Moreover, In terms of phenylketonuria treatment-based diet adoption, the United States is a leader. Patients in the country have taken a lot of PKU substances, including essential and non-essential amino acids.

Palynziq (pegvaliase-pqpz) has also been approved by the Food and Drug Administration (FDA) for adults with PKU. Palynziq is an injectable enzyme therapy for patients with uncontrolled blood phenylalanine levels despite existing medication. BioMarin Pharmaceutical Inc. manufactures Palynziq. Thus, the North American region is expected to hold the largest market share in the forecast period from the above statements.

Competitive Landscape

Major key players in the phenylketonuria treatment market are BioMarin, SOM Innovation Biotech SL, Homology Medicines, Inc., Par Pharmaceutical, PTC Therapeutics, and Synlogic, Inc. The key players are adopting various strategies such as product launches, mergers & acquisitions, partnerships, and collaborations, contributing to the growth of the Phenylketonuria Treatment market globally. For instance, on Dec 16, 2021, BioMarin and Skyline Therapeutics entered a strategic collaboration to develop novel gene therapies for cardiovascular diseases. Also, on June 13, 2022, the U.S. Food and Drug Administration (FDA) lifted the clinical hold previously placed on the pheNIX gene therapy clinical trial with investigational HMI-102 for adults with phenylketonuria (PKU) of Homology Medicines, Inc.

BioMarin Pharmaceutical Inc.

Overview:

BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities worldwide, including in the United States, South America, Asia, and Europe. Enzyme replacement medicines are BioMarin's main business and research focus (ERTs). By manufacturing laronidase, BioMarin was the first company to deliver therapies for mucopolysaccharidosis type I (MPS I) (Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first business to develop phenylketonuria treatments (PKU).

Product Portfolio:

KUVAN (sapropterin dihydrochloride): KUVAN (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in adults and children over one month of age with a certain type of Phenylketonuria (PKU). It is to be used along with a Phe-restricted diet.

The global phenylketonuria treatment market report would provide access to approximately 40+ market data tables, 45+ figures, and in the range of 200 (approximate) pages.

Table of Contents

1. Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Market Definition and Overview

3. Executive Summary

4. Market Dynamics

  • 4.1. Market Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Clinical Trials Conducted by The Market Players to Treat Phenylketonuria Are Expected to Drive Market Growth.
    • 4.1.2. Restraints
      • 4.1.2.1. Lack Of Awareness Regarding the Rare Disease Is Expected to Hamper Market Growth.
      • 4.1.2.2. High Treatment Cost
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Industry Analysis

  • 5.1. Porter's Five Forces Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Regulatory Analysis

6. COVID-19 Analysis

  • 6.1. Analysis of Covid-19 on the Market
    • 6.1.1. Before COVID-19 Market Scenario
    • 6.1.2. Present COVID-19 Market Scenario
    • 6.1.3. After COVID-19 or Future Scenario
  • 6.2. Pricing Dynamics Amid Covid-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During Pandemic
  • 6.5. Manufacturer's Strategic Initiatives
  • 6.6. Conclusion

7. By Type

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type Segment
    • 7.1.2. Market Attractiveness Index, By Type Segment
  • 7.2. Hyperphenylalaninemia
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis, US$ Million, 2021-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 7.3. Mild PKU
  • 7.4. Moderate or variant
  • 7.5. Classic PKU
  • 7.6. Others

8. By Treatment Type

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type Segment
    • 8.1.2. Market Attractiveness Index, By Treatment Type Segment
  • 8.2. Medications
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis, US$ Million, 2021-2029 and Y-o-Y Growth Analysis (%), 2021-2029
      • 8.2.2.1. Kuvan
      • 8.2.2.2. Pegvaliase
      • 8.2.2.3. Palynziq
      • 8.2.2.4. CNSA-001
      • 8.2.2.5. SYNB1618
      • 8.2.2.6. Others
  • 8.3. Dietary Supplement
  • 8.4. Others

9. By Route of Administration

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration Segment
    • 9.1.2. Market Attractiveness Index, By Route of Administration Segment
  • 9.2. Oral
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis, US$ Million, 2021-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 9.3. Parenteral

10. By Region

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis, US$ Million, 2021-2029 and Y-o-Y Growth Analysis (%), 2021-2029, By Region
    • 10.1.2. Market Attractiveness Index, By Region
  • 10.2. North America
    • 10.2.1. Introduction
    • 10.2.2. Key Region-Specific Dynamics
    • 10.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 10.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 10.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 10.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.2.6.1. The U.S.
      • 10.2.6.2. Canada
      • 10.2.6.3. Mexico
  • 10.3. Europe
    • 10.3.1. Introduction
    • 10.3.2. Key Region-Specific Dynamics
    • 10.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 10.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 10.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 10.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.3.6.1. Germany
      • 10.3.6.2. The U.K.
      • 10.3.6.3. France
      • 10.3.6.4. Italy
      • 10.3.6.5. Spain
      • 10.3.6.6. Rest of Europe
  • 10.4. South America
    • 10.4.1. Introduction
    • 10.4.2. Key Region-Specific Dynamics
    • 10.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 10.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 10.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 10.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.4.6.1. Brazil
      • 10.4.6.2. Argentina
      • 10.4.6.3. Rest of South America
  • 10.5. Asia Pacific
    • 10.5.1. Introduction
    • 10.5.2. Key Region-Specific Dynamics
    • 10.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 10.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 10.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 10.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.5.6.1. China
      • 10.5.6.2. India
      • 10.5.6.3. Japan
      • 10.5.6.4. Australia
      • 10.5.6.5. Rest of Asia Pacific
  • 10.6. Middle East and Africa
    • 10.6.1. Introduction
    • 10.6.2. Key Region-Specific Dynamics
    • 10.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 10.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 10.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration

11. Competitive Landscape

  • 11.1. Key Developments and Strategies
  • 11.2. Company Share Analysis
  • 11.3. Product Benchmarking

12. Company Profiles

  • 12.1. BioMarin
    • 12.1.1. Company Overview
    • 12.1.2. Product Portfolio and Description
    • 12.1.3. Key Highlights
    • 12.1.4. Financial Overview
  • 12.2. SOM Innovation Biotech SL
  • 12.3. Homology Medicines, Inc.
  • 12.4. Par Pharmaceutical
  • 12.5. PTC Therapeutics
  • 12.6. Synlogic, Inc.

LIST NOT EXHAUSTIVE

13. DataM

  • 13.1. Appendix
  • 13.2. About Us and Services
  • 13.3. Contact Us