フリードライヒ運動失調症治療の世界市場 - 2023年～2030年

概要

世界のフリードライヒ運動失調症治療市場は、そのダイナミクスに影響を与える様々な要因によって、長年にわたって大きな成長と変貌を遂げてきました。フリードライヒ運動失調症と呼ばれる稀な遺伝的変性疾患は、脳の小脳領域、末梢神経、脊髄に害を及ぼします。この病気は一般的に子供や10代で発症し、時間の経過とともに悪化します。症状が悪化すると、不安定で不快な動きや、神経損傷による感覚の欠如が起こる。

特に新興経済諸国における政府投資と研究は、オマベロキソロンやプロプラノロールのような新薬の利用を引き続き促進し、世界のフライライヒ失調症治療市場を押し上げると思われます。それぞれの革新的な医薬品は複数の研究者によって開発され、現在試験中です。

市場力学：市場促進要因と市場抑制要因

共同研究や買収の増加

新興経済諸国は、高額の投資、所得水準、インフラ整備を原動力として、ヘルスケア分野の改善において急速な成長を遂げています。いくつかの国では、研究の高まりにより、フリードライヒ運動失調症治療に対する大きな需要が発生しています。様々な国間での共同研究、買収、技術的進歩を伴う製品の発売は、市場の成長を促進する重要な要因です。

2023年1月9日、Voyager Therapeutics, Inc.とNeurocrine Biosciences, Inc.は、神経疾患治療のための遺伝子治療を前進させるため、最近新たな戦略的提携を結びました。ボイジャーの前臨床GBA1遺伝子治療プログラムは、GBA1遺伝子置換ペイロードとボイジャーのTRACERTM（Tropism Redirection of AAV via Cell-type-specific Expression of RNA）プラットフォームの新しいカプシドを統合したもので、この提携の一環です。

さらに最近、ミケーレ・ルフィーノ博士がAtaxia UK、オックスフォード大学、ファイザー社、UCL、インペリアル・カレッジ・ロンドンとの間でフリードライヒ失調症（FA）の新しい共同創薬研究を主導することが明らかになっています。この提携を推進したのは、全国的な運動失調症慈善団体であるAtaxia UKです。英国の遺伝医学研究所と米国マサチューセッツ州ケンブリッジ（ファイザーの本拠地）にいるファイザーの専門家と協力する大学の研究者たちを結集します。

さらに、いくつかの研究目的のために、新しい技術の利用や製品の発売が増加しています。フリードライヒ運動失調症患者の増加と革新的な治療製品開発のための研究の増加は、フリードライヒ運動失調症治療市場の成長を促進する主な要因です。

フリードライヒ運動失調症治療薬に関連する副作用

胃痛、下痢、吐き気などの可能性があります。この薬の使用者の多くは、重大な副作用はないと報告しています。重篤な副作用は、足/足首/手のむくみ、突然の/原因不明の体重増加、ドキドキする心拍、息切れなどです。

肝障害の徴候には、止まらない吐き気/嘔吐、食欲不振、胃痛/腹痛、黄ばんだ目/皮膚、濃い尿などがあります。発疹、かゆみ/腫れ（特に顔、舌、首）、極度の意識障害、呼吸困難など、この薬が非常に重大なアレルギー反応を引き起こすことはまれです。

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
    • 共同研究や買収の増加
    • 技術的進歩と開発の高まり
  • 抑制要因
    • フリードライヒ運動失調症治療治療薬に伴う副作用
  • 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
• SWOT分析
• 特許分析
• ロシア・ウクライナ戦争の影響分析
• DMI意見

第6章 COVID-19分析

第7章 薬剤別

• オマベロキソロン
• プロプラノロール
• 5-ヒドロキシトリプトファン
• コエンザイムQ
• イデベノン
• その他

第8章 投与経路別

• 経口
• 非経口

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
  • その他の欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他の南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • オーストラリア
  • その他のアジア太平洋
• 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

• Reata Pharmaceuticals, Inc.
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Retrotope Inc.
• Minoryx Therapeutics
• Design Therapeutics, Inc.
• PTC Therapeutics
• Lexeo Therapeutics, Inc.
• Larimar Therapeutics, Inc.
• Metro International Biotech, LLC
• Astellas Gene Therapies, Inc.
• Voyager Therapeutics, Inc.

第13章 付録

Overview

The global friedreich's ataxia treatment market has witnessed significant growth and transformations over the years, with various factors influencing its dynamics. A rare, genetic, degenerative condition called friedreich's ataxia harms the cerebellar region of the brain, peripheral nerves, and the spinal cord. This illness typically appears in kids and teenagers and gets worse over time. As the condition worsens, unsteady, uncomfortable movements and a lack of sensation from nerve damage occur.

Government investments and research, particularly in developing economies, will continue to drive utilization of novel medicines like omaveloxolone or propranolol and boost the global friedreich's ataxia treatment market. The respective innovative medications are being developed by several researchers and currently are under trials.

Market Dynamics: Drivers & Restraints

Growing collaborations and acquisitions

Developed economies have been witnessing rapid growth in betterment of their healthcare sectors, driven by high investments, income levels, and infrastructure development. Several countries have experienced substantial demand for friedreich's ataxia treatment owing to the rising research. Various collaborations, acquisitions and product launches with technological advanecments among different countries will be a crucial factor driving the growth of the market.

On January 9, 2023, In order to advance several gene treatments for the treatment of neurological illnesses, Voyager Therapeutics, Inc. and Neurocrine Biosciences, Inc. recently forged a new strategic partnership. The preclinical GBA1 gene therapy program from Voyager, which integrates a GBA1 gene replacement payload with new capsids from Voyager's TRACERTM (Tropism Redirection of AAV via Cell-type-specific Expression of RNA) platform, is a part of the collaboration.

Additionally, recently, it was revealed that Dr. Michele Lufino would be leading a new cooperative drug discovery study in Friedreich's ataxia (FA) between Ataxia UK, the University of Oxford, Pfizer Inc., UCL, and Imperial College London. The national ataxia charity, Ataxia UK, was the driving force for the partnership. It brings together university researchers who will collaborate with Pfizer experts situated in the UK's Genetic Medicine Institute as well as in Cambridge, Massachusetts, in the US (Pfizer's home base).

Furthermore, the rising utilization of novel technology and product launches for the several research purposes. Rising friedreich's ataxia cases and increasing research for innovative treatment product development will be a major factor driving the growth of the friedreich's ataxia treatment market.

Side effects associated with the friedreich's ataxia medications

There could be stomach pain, diarrhea, or nausea. Many users of this medicine report no significant negative effects. Serious side effects are swelling feet/ankles/ hands, sudden/unexplained gain in weight, pounding heartbeat, and shortness of breath.

Signs of liver problems include nausea/vomiting that doesn't stop, appetite loss, stomach/abdominal pain, yellowing eyes/skin, and dark urine. It is uncommon for this medication to cause a very significant allergic reaction, which includes rash, itching/swelling (particularly of the face, tongue, and neck), extreme disorientation, and difficulty breathing.

Segment Analysis

The global friedreich's ataxia treatment market is segmented based on medications, route of administration, distribution channel and region.

Omaveloxolone segment accounted for approximately 61% of market share

Reata Pharmaceuticals, Inc. is developing omaveloxolone (SKYCLARYS), a small molecule, orally active, semi-synthetic triterpenoid medication that boosts antioxidant activity, to treat Friedreich's ataxia. Specifically, omaveloxolone is employed to treat a condition that affects the muscles and nerves.

Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway suppression in Friedreich's ataxia patients is linked to oxidative stress, mitochondrial malfunction, and cell damage, especially to central and peripheral neurons. Omaveloxolone may cause the Nrf2 pathway to be active because it prevents Nrf2 from being ubiquitinated and degraded. Omaveloxolone was authorized for use in the USA in February 2023 to treat Friedreich's ataxia.

On February 28, 2023, the U.S. Food and Drug Administration ("FDA") has approved Reata Pharmaceuticals, Inc.'s SKYCLARYS (omaveloxolone) for the management of Friedreich's ataxia in adults as well as adolescents aged 16 and older. Reata Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and marketing new treatments for patients with severe diseases. The FDA gave a priority review voucher for a rare pediatric condition with this approval.

Geographical Penetration

North America accounted for around 38% of market share in 2022

Due to the rising need for friedreich's ataxia treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for friedreich's ataxia treatment.

Increasing expenditure on healthcare and rising research studies, advancement of technologies and different types of medicines, and increase in biopharmaceutical or biotechnology business establishment across the region are also contributing to the growth of friedreich's ataxia treatment market share of this region. The market in this area is growing as people become more aware of various novel approved medicines. The aforementioned elements further attest to North America's hegemonic position in the world.

North America continues to be a key player in the global friedreich's ataxia treatment market, with United States leading the way. Government initiatives promoting infrastructure development and investment, and a focus on rising advancements have fueled the demand for friedreich's ataxia treatment in the U.S. United States have been proactive in executing several initiatives or researches, stimulating friedreich's ataxia treatment demand.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global friedreich's ataxia treatment market. As countries grappled with lockdowns, supply chain disruptions and reduced economic activity, the pharmaceutical sector, with a significant consumer of various treatments, was significantly impacted. Several efforts all throughout the world were impacted by the pandemic's broad lockdowns and limitations that started in early 2020.

Major medical and biotechnology industries came to a standstill and shifted their attention towards the management of COVID-19, leading to a slump in demand for friedreich's ataxia treatment. Now several research studies have been initiated and companies have again started trial for their products efficiency. Overall, the impact of the pandemic on the global friedreich's ataxia treatment market is expected to be relatively moderate, with the market continuing to grow steadily due to the ongoing need and research for inovative medications.

Key Developments

• On August 14, 2023, initial findings from Design Therapeutics, Inc.'s phase one multiple-ascending dose (MAD) clinical study of DT-216 in patients having Friedrich ataxia (FA) were revealed. Design Therapeutics, Inc. is a clinical-stage biotechnology business exploring treatments for severe degenerative genetic illnesses. The findings demonstrated that DT-216 was usually well tolerated and increased the levels of frataxin (FXN) mRNA in skeletal muscle biopsies in a dose-dependent and statistically significant manner.
• On May 31, 2023, Topline data from the MOVE-FA study of vatiquinone in individuals with Friedreich ataxia were released by PTC Therapeutics. The primary goal of the trial, which was a statistically significant shift in mFARS score in the main analysis population at 72 weeks, was not achieved. However, the use of vatiquinone did show a significant improvement in important illness endpoints and subscales.
• On November 29, 2022, a Phase 2 double-blind, randomized, placebo-controlled investigation into the impact of leriglitazone on patients with Friedreich's ataxia (FRAMES study) was published in Neurology Genetics by Minoryx Therapeutics, a late-stage biotech company specializing in the development of therapies for orphan central nervous system (CNS) disorders.

Competitive Landscape

The major global players in the market include: Reata Pharmaceuticals, Inc., Retrotope Inc., Minoryx Therapeutics, Design Therapeutics, Inc., PTC Therapeutics, Lexeo Therapeutics, Inc., Larimar Therapeutics, Inc., Metro International Biotech, LLC, Astellas Gene Therapies, Inc. and Voyager Therapeutics, Inc. among others.

Why Purchase the Report?

• To visualize the global friedreich's ataxia treatment market segmentation based on medications, 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 friedreich's ataxia treatment 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 friedreich's ataxia treatment market report would provide approximately 61 tables, 59 figures and 186 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 Medications
• 3.2. Snippet by Route of Administration
• 3.3. Snippet by Distribution Channel
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Growing collaborations and acquisitions
    • 4.1.1.2. Rising technological advancements and developments
  • 4.1.2. Restraints
    • 4.1.2.1. Side effects associated with the friedreich's ataxia medications
  • 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. SWOT Analysis
• 5.6. Patent Analysis
• 5.7. Russia-Ukraine War Impact Analysis
• 5.8. 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 Medications

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Medications
  • 7.1.2. Market Attractiveness Index, By Medications
• 7.2. Omaveloxolone*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Propranolol
• 7.4. 5-Hydroxytryptophan
• 7.5. Coenzyme Q
• 7.6. Idebenone
• 7.7. Others

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. Parenteral

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 Medications
  • 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 Medications
  • 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 Medications
  • 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 Medications
  • 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 Medications
  • 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. Reata Pharmaceuticals, Inc.*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Retrotope Inc.
• 12.3. Minoryx Therapeutics
• 12.4. Design Therapeutics, Inc.
• 12.5. PTC Therapeutics
• 12.6. Lexeo Therapeutics, Inc.
• 12.7. Larimar Therapeutics, Inc.
• 12.8. Metro International Biotech, LLC
• 12.9. Astellas Gene Therapies, Inc.
• 12.10. Voyager Therapeutics, Inc.

LIST NOT EXHAUSTIVE

13. Appendix

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