表紙:プロピオン酸血症治療薬の世界市場-2023年~2030年
市場調査レポート
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1374823

プロピオン酸血症治療薬の世界市場-2023年~2030年

Global Propionic Acidemia Drugs Market -2023-2030

出版日: | 発行: DataM Intelligence | ページ情報: 英文 180 Pages | 納期: 約2営業日

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プロピオン酸血症治療薬の世界市場-2023年~2030年
出版日: 2023年11月01日
発行: DataM Intelligence
ページ情報: 英文 180 Pages
納期: 約2営業日
ご注意事項 :
本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
  • 全表示
  • 概要
  • 目次
概要

概要

プロピオン酸血症は、体内でタンパク質や脂質(脂肪)の特定の部分を適切に処理できない遺伝性の疾患です。有機酸障害に分類され、有機酸として知られる特定の酸が異常に蓄積する疾患です。

プロピオン酸血症は、プロピオニル-CoAからメチルマロニル-CoAへの変換を触媒する酵素であるプロピオニル-CoAカルボキシラーゼ(PCC)の欠損によって生じます。新生児スクリーニング検査でPAを発症した新生児は、C3(プロピオニルカルニチン)が上昇しています。症状がある人や新生児スクリーニングで検出された人の尿中有機酸を検査すると、3-ヒドロキシプロピオン酸の上昇、メチルシトレート、チグリグリシン、プロピオニルグリシン、乳酸の存在が明らかになります。

市場力学:市場促進要因と市場抑制要因

プロピオン酸血症の有病率の上昇

プロピオン酸血症の有病率の上昇は、予測期間中に市場が成長するのに役立つ重要な要因の一つです。例えば、NCBIに掲載された論文によると、プロピオン酸血症は米国で約10万人に1人が罹患しています。

この症状は、グリーンランドのイヌイット集団、一部のアーミッシュコミュニティ、サウジアラビア人など、世界中のいくつかの集団でより一般的であるようです。さらに、治療オプションの進歩、ケトーシス性高グリシン血症の有病率の上昇なども、予測期間中の市場成長を促進すると思われます。

市場力学:抑制要因

プロピオン酸血症市場の抑制要因の1つは、プロピオン酸血症に対する新薬治療の臨床試験の登録と実施が非常に複雑であることです。これは、これらの先天性代謝異常に関連する希少性、不均一性、死亡率、予測不能な転帰のためです。これらの臨床試験のための臨床エンドポイントの選択は特に困難です。したがって、これらの希少疾患に対する新しい治療法の研究を支援するために、どのプロピオン酸血症のバイオマーカーが代替エンドポイントになり得るかを同定する必要があります。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

  • 影響要因
    • 促進要因
      • プロピオン酸血症の有病率の上昇
      • 治療オプションの進歩
    • 抑制要因
      • 薬剤に関する課題
    • 機会
    • 影響分析

第5章 産業分析

  • ポーターのファイブフォース分析
  • サプライチェーン分析
  • 価格分析
  • 規制分析
  • ロシア・ウクライナ戦争の影響分析
  • DMIの見解

第6章 COVID-19分析

第7章 薬剤クラス別

  • L-カルニチン
  • 抗生物質
  • その他の医薬品サプリメント

第8章 投与経路別

  • 経口
  • 静脈内
  • 筋肉内

第9章 流通チャネル別

  • 病院薬局
  • 小売薬局
  • オンライン薬局

第10章 地域別

  • 北米
    • 米国
    • カナダ
    • メキシコ
  • 欧州
    • ドイツ
    • 英国
    • フランス
    • イタリア
    • スペイン
    • その他欧州
  • 南米
    • ブラジル
    • アルゼンチン
    • その他南米
  • アジア太平洋
    • 中国
    • インド
    • 日本
    • オーストラリア
    • その他アジア太平洋
  • 中東・アフリカ

第11章 競合情勢

  • 競合シナリオ
  • 市況/シェア分析
  • M&A分析

第12章 企業プロファイル

  • Leadiant
    • 会社概要
    • 製品ポートフォリオと説明
    • 財務概要
    • 主な動向
  • Pfizer
  • ScotMedcare
  • Cambridge Healthcare
  • Teva Pharmaceuticals
  • Wellona Pharma
  • Merck
  • AdvaCare Pharma
  • SERVE PHARMACEUTICALS
  • Vitane Pharmaceuticals, Inc.

第13章 付録

目次
Product Code: PH7250

Overview

Propionic acidemia is an inherited disorder in which the body is unable to process certain parts of proteins and lipids (fats) properly. It is classified as an organic acid disorder, which is a condition that leads to an abnormal buildup of particular acids known as organic acids.

Propionic acidemia is caused by a deficiency of propionyl-CoA carboxylase (PCC), the enzyme that catalyzes the conversion of propionyl-CoA to methyl malonyl-CoA. Newborns with PA tested by expanded newborn screening have elevated C3 (propionylcarnitine). Testing of urine organic acids in persons who are symptomatic or those detected by newborn screening reveals elevated 3-hydroxypropionate and the presence of methylcitrate, tiglylglycine, propionylglycine, and lactic acid.

Market Dynamics: Drivers and Restraints

Rise in the prevalence of propionic acidemia

The rise in the prevalence of propionic acidemia is one of the significant factors that helps the market to grow during the forecast period. For instance, according to the article published in NCBI Propionic acidemia affects about 1 in 100,000 people in the United States.

The condition appears to be more common in several populations worldwide, including the inuit population of Greenland, some amish communities, and saudi arabians. Furthermore, the rise in the advancements in treatment options, the rise in the prevalence of ketotic hyperglycinemia and others will also drive the market growth during the forecast period.

Market Dynamics: Restraint

One of the restraint factors of the propionic acidemia market is it is highly complex to enroll and conduct a clinical trial of a new drug treatment for propionic acidemia, due to the rarity, heterogeneity, mortality, and unpredictable outcomes associated with these inborn errors of metabolism. Selecting clinical endpoints for these trials is particularly challenging. Thus, there is a clear need to identify which biomarkers for propionic acidemia could be surrogate endpoints to support the investigation of new therapies for these rare diseases.

Segment Analysis

The global propionic acidemia drugs is segmented based on drug class, route of administration distribution channel and region.

The L-carnitine from the drug class segment accounted for approximately 40.2 % of the market share

The L-carnitine from the drug class segment accounted for 40.2% and it is expected to be dominated during the forecast period. L-carnitine is a chemical that is made in the human brain, liver, and kidneys. It helps the body turn fat into energy. L-carnitine is important for heart and brain function, muscle movement, and many other body processes. The body can convert L-carnitine to other chemicals called acetyl-L-carnitine and propionyl-L-carnitine

Treatment with L-carnitine greatly enhanced the formation and excretion of short-chain acylcarnitines in three patients with propionic acidemia and in three normal controls. The use of fast atom bombardment mass spectrometry and linked scanning at constant magnetic (B) to electric ? field ratio identified acylcarnitine as propionylcarnitine in patients with propionic acidemia. The normal children excreted mostly acetylcarnitine.

Geographical Analysis

North America accounted for approximately 38.4% of the market share in 2022

North America is estimated to hold about 38.4% of the total market share throughout the forecast period, owing, to the improved healthcare infrastructure, FDA approvals, rise in the prevalence of propionic acidemia, collaborations and acquisitions with the key players and others expected to dominate by this region during the forecast period.

For instance, in May 2022, the U.S. Food and Drug Administration (FDA) granted the National Institutes of Health (NIH)'s National Center for Advancing Translational Sciences (NCATS) an orphan drug designation for AAV9-hPCCA (NCATS-BL0746), investigational gene therapy for the treatment of propionic acidemia resulting from a deficiency of Propionyl-CoA Carboxylase, alpha subunit (PCCA).

COVID-19 Impact Analysis

COVID-19 had a substantial impact on the propionic acidemia market where evidence suggests that patients with propionic acidemia who contract COVID-19 may be at risk of metabolic decompensation, but the clinical course of the infection is generally mild and recovery is possible without severe clinical consequences.

Another case report published in NCBI it describes those two patients with underlying metabolic disorders, including propionic acidemia, who contracted COVID-19. The report suggests that individuals with an underlying inborn error of metabolism (IEM) are at risk of metabolic decompensation in the setting of a COVID-19 infection. However, the virus does not appear to cause a more severe metabolic deterioration than is typical.

Competitive Landscape

The major global players in the propionic acidemia drugs market include: Leadiant, Pfizer, ScotMedcare, Cambridge Healthcare, Teva Pharmaceuticals, Wellona Pharma, Merck, AdvaCare Pharma, serve pharmaceuticals, Vitane Pharmaceuticals, Inc and among others.

Key Developments

  • In May 2023, Moderna, Inc, a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, stated that an abstract reporting on interim data from the Phase I/II trial of mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA) has been accepted for presentation at the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting being held May 16-20, 2023 in Los Angeles, CA.
  • In August 2022, BridgeBio Pharma stated the Dosing of the First Patient in the Phase I Trial of BBP-671, a Potential best-in-class treatment for propionic acidemia (PA) and methylmalonic acidemia (MMA)BBP-671 is an investigational oral therapy intended to increase CoA levels by allosterically modulating pantothenate kinases, key enzymes in the CoA biosynthesis pathway.
  • In January 2021, Food and Drug Administration (FDA) approved a new indication for Carbaglu for use as adjunctive therapy in the treatment of acute hyperammonemia due to propionic acidemia (PA).

Why Purchase the Report?

  • To visualize the global propionic acidemia drugs market segmentation-based drug class, route of administration, distribution channel and region as well as understand key commercial assets and players.
  • Identify commercial opportunities by analyzing trends and co-development
  • Excel data sheet with numerous data points of propionic acidemia drugs market-level with all segments.
  • PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
  • Product mapping available as excel consisting of key products of all the major players.

The global propionic acidemia drugs market report would provide approximately 69 tables, 70 figures, and 185 Pages.

Target Audience 2023

  • Manufacturers/ Buyers
  • Industry Investors/Investment Bankers
  • Research Professionals
  • Emerging Companies

Table of Contents

1. Methodology and Scope

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

2. Definition and Overview

3. Executive Summary

  • 3.1. Snippet by Drug Class
  • 3.2. Snippet by Route of Administration
  • 3.3. Snippet by End User
  • 3.4. Snippet by Region

4. Dynamics

  • 4.1. Impacting Factors
    • 4.1.1. Driver
      • 4.1.1.1. Rise in the prevalence of propionic acidemia
      • 4.1.1.2. Advancements in treatment options
    • 4.1.2. Restraints
      • 4.1.2.1. Challenges associated with the drugs
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Industry Analysis

  • 5.1. Porter's Five Force Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Pricing Analysis
  • 5.4. Regulatory Analysis
  • 5.5. Russia-Ukraine War Impact Analysis
  • 5.6. DMI Opinion

6. COVID-19 Analysis

  • 6.1. Analysis of COVID-19
    • 6.1.1. Scenario Before COVID
    • 6.1.2. Scenario During COVID
    • 6.1.3. Scenario Post COVID
  • 6.2. Pricing Dynamics Amid COVID-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During Pandemic
  • 6.5. Manufacturers Strategic Initiatives
  • 6.6. Conclusion

7. By Drug Class

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
    • 7.1.2. Market Attractiveness Index, By Drug Class
  • 7.2. L-Carnitine *
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. Antibiotics
  • 7.4. Other Drug Supplements

8. By Route of Administration

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 8.1.2. Market Attractiveness Index, By Route of Administration
  • 8.2. Oral *
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3. Intravenous
  • 8.4. Intramuscular

9. By Distribution Channel

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 9.1.2. Market Attractiveness Index, By Distribution Channel
  • 9.2. Hospital Pharmacies *
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 9.3. Retail Pharmacies
  • 9.4. Online Pharmacies

10. By Region

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), 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 Drug class
    • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of administration
    • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 10.2.6.1. 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 Drug class
    • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of administration
    • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 10.3.6.1. Germany
      • 10.3.6.2. UK
      • 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 Drug class
    • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of administration
    • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 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 Drug class
    • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of administration
    • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 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 Drug class
    • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of administration
    • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel

11. Competitive Landscape

  • 11.1. Competitive Scenario
  • 11.2. Market Positioning/Share Analysis
  • 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

  • 12.1. Leadiant
    • 12.1.1. Company Overview
    • 12.1.2. Product Portfolio and Description
    • 12.1.3. Financial Overview
    • 12.1.4. Key Developments
  • 12.2. Pfizer
  • 12.3. ScotMedcare
  • 12.4. Cambridge Healthcare
  • 12.5. Teva Pharmaceuticals
  • 12.6. Wellona Pharma
  • 12.7. Merck
  • 12.8. AdvaCare Pharma
  • 12.9. SERVE PHARMACEUTICALS
  • 12.10. Vitane Pharmaceuticals, Inc.

LIST NOT EXHAUSTIVE

13. Appendix

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