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苯丙酮尿症治療的全球市場-2022-2029
市場調查報告書
商品編碼
1140769

苯丙酮尿症治療的全球市場-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: 約2個工作天內

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簡介目錄

市場概況

據估計，苯丙酮尿症治療市場在預測期內（2022-2029 年）的複合年增長率將達到 9.46%。

苯丙酮尿症是一種罕見的遺傳性疾病，其中氨基酸苯丙氨酸在體內積累。PKU 是由有助於產生苯丙氨酸降解□的基因突變引起的。血液中過量的苯丙氨酸會導致癲癇發作、精神疾病和其他未經治療的疾病。PKU 無法治愈，但藥物可以預防智力障礙和其他健康問題。PKU 的治療包括堅持限制含有苯丙氨酸的食物的 PKU 飲食。

市場動態

由於對苯丙酮尿症新干預措施的研究增加等多種因素，全球苯丙酮尿症治療市場正在增長。

市場參與者進行的治療苯丙酮尿症的臨床試驗預計將推動市場增長。

BioMarin Pharmaceutical Inc. 將研究 BMN 307（一種腺相關病毒載體介導的人苯丙氨酸羥化□基因轉移）在血漿 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 開放標籤，進行了劑量遞增研究。PTC Therapeutics 還通過測量血液 Phe 水平從基線到第 5 周和第 6 週的變化來評估 PTC923 降低苯丙酮尿症患者血液苯丙氨酸水平的效果（即，每次治療還進行了臨床試驗以評估劑量（平均值2 周雙盲治療），證實 PTC923 可有效降低苯丙酮尿症患者的血 Phe 水平。因此，根據上述描述，預計將在預測期內推動市場。

低意識和高成本預計將阻礙全球苯丙酮尿症治療市場的增長。

缺乏對罕見病的認識、PKU 治療的高成本、不規則的報銷政策以及缺乏對治療方案的依從性是預計在預測期內阻礙市場增長的一些因素。

行業分析

全球苯丙酮尿症治療市場報告根據波特五力、創新、新產品發布、定價和管道分析等各種行業因素對市場進行了深入分析。

COVID-19 的影響分析。

COVID-19 大流行對醫療保健系統和市場產生了適度的影響。苯丙酮尿症 (PKU) 的神經認知結果和預後與終生苯丙氨酸 (Phe) 水平和治療依從性直接相關。PKU 患者不得不調整飲食以適應新環境。年輕患者可能接觸較少（飲食嚴格遵循飲食計劃，無論情況如何），但青少年和成年患者表現出更好的代謝控制，從而履行了他們留在家中的義務。強烈反映。這可歸因於多種因素，包括更容易獲得遠程諮詢。儘管如此，我們的研究結果表明流行病和環境如何顯著影響 PKU 治療依從性，以及消除距離障礙如何改善和優化代謝依從性。綜上所述，市場受到影響，預計隨著經濟活動的恢復，市場將出現早期拉動。

細分分析

在苯丙酮尿症治療市場中，預計膳食補充劑部分將在整個預測期間（2022-2029 年）佔據最大的市場份額。

膳食補充劑市場預計將在 2021 年佔據主導地位。這部分的增長是有益的，因為飲食限制了含有苯丙氨酸的食物。這意味著飲食必須低蛋白質，PKU 患者與醫生和營養師密切合作，在減少苯丙氨酸攝入的同時保持健康的食物平衡。您還應該跟蹤當天飲食中苯丙氨酸的含量，並了解您的苯丙氨酸水平。此外，患有 PKU 的兒童必須服用 PKU 配方以保持他們獲得足夠的蛋白質。含有除苯丙氨酸外的所有必需氨基酸。健康食品商店還提供低蛋白、對 PKU 友好的食品。例如，患有 PKU 的嬰兒是母乳喂養的。一般來說，您還應該服用一種特殊的粉狀配方，稱為洛芬酸。一旦嬰兒長大到可以吃固體食物，就應該避免富含蛋白質的食物。因此，從上述描述來看，這種市場細分預計將在預測期內佔最大的市場份額。

區域分析

北美地區佔全球苯丙酮尿症治療市場最大的市場份額。

到 2021 年，北美將佔據最高的收入份額。苯丙酮尿症患病率的上升和苯丙酮尿症治療飲食的高度採用、政府對正在進行的臨床研究的支持以及該地區市場參與者推出的產品預計將在預測期內增加。例如，據說 PKU 會影響美國 10,000 到 15,000 人中的 1 人。大多數 PKU 在出生後不久通過新生兒篩查被診斷出來，並立即開始治療。美國在治療苯丙酮尿症的飲食流行方面也處於領先地位。該國的患者服用許多 PKU 物質，包括必需和非必需氨基酸。

Palynziq (Pegvariase-pqpz) 也被 FDA 批准用於患有 PKU 的成年人。Palynziq 是一種可注射的□療法，適用於儘管現有藥物療法但血液苯丙氨酸水平不受控制的患者。BioMarin Pharmaceutical Inc. 生產 Palynziq。因此，從上述陳述來看，預計北美地區將在預測期內佔據最大的市場份額。

競爭格局

苯丙酮尿症治療市場的主要參與者是 BioMarin、SOM Innovation Biotech SL、Homology Medicines, Inc.、Par Pharmaceutical、PTC Therapeutics 和 Synlogic, Inc.。主要參與者正在採取各種策略，例如產品發布、併購、合作夥伴和聯盟，以促進全球苯丙酮尿症治療市場的增長。例如，2021 年 12 月 16 日，BioMarin 和 Skyline Therapeutics 結成戰略聯盟，開發針對心血管疾病的新型基因療法。此外，2022 年 6 月 13 日，美國食品藥品監督管理局 (FDA) 批准了 Homology Medicines, Inc. 的 pheNIX 基因治療臨床試驗，其研究藥物 HMI-102 用於成人苯丙酮尿症 (PKU)。抓住。

BioMarin Pharmaceutical Inc.

概述

BioMarin, Inc. 是一家美國生物技術公司，總部位於加利福尼亞州聖拉斐爾。我們在世界各地設有辦事處，包括美國、南美、亞洲和歐洲。□替代藥物是 BioMarin 的主要業務和研究重點 (ERT)。通過製造 Laronidase，BioMarin 成為第一家提供 I 型粘多醣貯積症 (MPS I) 治療的公司（Aldurazyme，由 Genzyme Corporation 商業化）。它也是第一家開發治療苯丙酮尿症（PKU）藥物的公司。

產品介紹

KUVAN（沙丙蝶呤二鹽酸鹽）：KUVAN（沙丙蝶呤二鹽酸鹽）口服片劑和口服溶液粉可降低成人和 1 個月及以上患有苯丙酮尿症 (PKU) 的兒童的血液 Phe 水平。它是一種處方藥，用於與限制 Phe 的飲食一起使用。

全球苯丙酮尿症治療市場報告將提供對大約 40 多個市場數據表、45 多個數字和 200 頁（大約）的訪問。

目錄

  • 調查方法
  • 調查目的和範圍

第二章市場定義和概述

第三章執行摘要

第四章市場動態

  • 市場影響因素
    • 促進者
      • 市場參與者進行的治療苯丙酮尿症的臨床試驗預計將推動市場增長。
    • 抑製劑
      • 對罕見病的低認識預計將阻礙市場增長
      • 治療費用高
    • 機會
    • 影響分析

第五章行業分析

  • 波特五力分析
  • 供應鏈分析
  • 監管分析

第 6 章 COVID-19 分析

  • COVID-19 的市場分析
    • COVID-19 之前的市場情景
    • COVID-19 的當前市場情景
    • COVID-19 後或未來情景
  • COVID-19 中的價格動態
  • 供需譜
  • 大流行期間與市場相關的政府努力
  • 製造商的戰略舉措
  • 概括

第 7 章 按類型

  • 高苯丙氨酸血症
  • 輕度北大
  • 中等或突變
  • 經典北大
  • 其他

第 8 章 按治療類型

  • 藥物治療
    • 關
    • 聚乙二醇化□
    • 帕林澤克
    • CNSA-001
    • SYNB1618
    • 其他
  • 膳食補充劑
  • 其他

第 9 章 按行政途徑

  • 口服
  • 腸外

第10章按地區

  • 北美
    • 美國
    • 加拿大
    • 墨西哥
  • 歐洲
    • 德國
    • 英國
    • 法國
    • 意大利
    • 西班牙
    • 歐洲其他地區
  • 南美洲
    • 巴西
    • 阿根廷
    • 其他南美地區
  • 亞太地區
    • 中國
    • 印度
    • 日本
    • 澳大利亞
    • 亞太其他地區
  • 中東/非洲

第 11 章 競爭格局

  • 主要發展和戰略
  • 公司股份分析
  • 產品基準

第十二章公司簡介

  • BioMarin
    • 公司簡介
    • 產品組合和描述
    • 主要亮點
    • 財務摘要
  • SOM Innovation Biotech SL
  • Homology Medicines, Inc.
  • Par Pharmaceutical
  • PTC Therapeutics
  • Synlogic, Inc.

第 13 章 數據管理

簡介目錄
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