시장보고서
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1140769

세계의 페닐케톤뇨증 치료 시장(2022-2029년)

Global Phenylketonuria Treatment Market - 2022-2029

발행일: | 리서치사: DataM Intelligence | 페이지 정보: 영문 200 Pages | 배송안내 : 1-2일 (영업일 기준)

    
    
    


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시장 개요

페닐케톤뇨증 치료 시장은 예측 기간(2022-2029년)에 CAGR 9.46%에 달할 것으로 추정됩니다.

목차

  • 조사 방법
  • 조사 목적 및 조사 범위

제2장 시장의 정의와 개요

제3장 주요 요약

제4장 시장 역학

  • 시장 영향요인
    • 촉진요인
      • 페닐케톤뇨증을 치료하기 위해 시장 관계자가 시행하는 임상시험은 시장의 성장을 촉진할 것으로 예상된다.
    • 억제요인
      • 희귀질환에 관한 낮은 인지도가 시장 성장을 저해할 전망
      • 높은 치료비
    • 기회
    • 영향 분석

제5장 산업 분석

  • Porter's Five Forces 분석
  • 공급망 분석
  • 규제 분석

제6장 COVID-19의 분석

  • COVID-19의 시장 분석
    • COVID-19 이전 시장 시나리오
    • COVID-19의 현재 시장 시나리오
    • COVID-19 이후 또는 미래 시나리오
  • COVID-19 중 가격 역학
  • 수요-공급 스펙트럼
  • 팬데믹시 시장에 관련된 정부 구상
  • 제조업체의 전략적 구상
  • 결론

제7장 유형별

  • 고페닐알라닌혈증
  • 경증 PKU
  • 중등도 또는 변이형
  • 고전적 PKU
  • 기타

제8장 치료 유형별

  • 약물 요법
    • Kuvan
    • Pegvaliase
    • Palynziq
    • CNSA-001
    • SYNB1618
    • 기타
  • 식이보충제
  • 기타

제9장 투여 경로별

  • 경구
  • 비경구

제10장 지역별

  • 북미
    • 미국
    • 캐나다
    • 멕시코
  • 유럽
    • 독일
    • 영국
    • 프랑스
    • 이탈리아
    • 스페인
    • 기타 유럽
  • 남미
    • 브라질
    • 아르헨티나
    • 기타 남미 지역
  • 아시아태평양
    • 중국
    • 인도
    • 일본
    • 호주
    • 기타 아시아태평양
  • 중동·아프리카 지역

제11장 경쟁 구도

  • 주요 개발과 전략
  • 기업 점유율 분석
  • 제품 벤치마킹

제12장 기업 개요

  • BioMarin
    • 기업 개요
    • 제품 포트폴리오와 설명
    • 주요 하이라이트
    • 재무 개요
  • SOM Innovation Biotech SL
  • Homology Medicines, Inc.
  • Par Pharmaceutical
  • PTC Therapeutics
  • Synlogic, Inc.

제13장 DataM

KSA 22.10.31

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
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