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1909226

urefnersen 的銷售預測與市場規模分析（2034 年）

Ulefnersen Sales Forecast, and Market Size Analysis - 2034

出版日期: 2026年01月01日 | 出版商:

DelveInsight | 英文 30 Pages | 商品交期: 2-10個工作天內

價格

簡介目錄圖表

urefnersen 的關鍵成長促進因素

1. 擴大市場佔有率並增加新患者數量

隨著人們對基因標靶治療的興趣日益濃厚，ulefnersen 在 ALS 研究和開發領域中獲得了早期發展勢頭。
參與臨床試驗的患者人數不斷增加，這反映出 ALS 專業中心的積極參與以及 ALS 患者群體中基因篩檢的擴大。
贊助商主導的教育活動以及與關鍵意見領袖的合作提高了人們的認知，並將 ulefnersen 確立為一種有前途的緩解疾病藥物候選物，而不是一種症狀治療藥物。

2. 拓展至主要適應症領域

肌萎縮側索硬化症（ALS）：主要針對基因定義的 ALS 亞群，並針對上游疾病機制，而非症狀管理。
神經退化性疾病的遺傳分層：這種機制支持其適用於涉及相似分子路徑的神經退化性疾病。
精準醫療策略：不斷擴大的產品線預計將繼續採用基於生物標記和基因型的方法，以符合個人化神經醫學的發展趨勢。
正在進行的轉化研究可能會在概念驗證後，為未來擴大適應症提供可能。

3. 地理擴張

臨床研發主要集中在北美和歐洲，這兩個地區擁有先進的ALS檢測基礎設施和基因檢測能力。
隨著全球 ALS 試驗網路的加強和與監管機構的討論取得進展，預計將擴展到更多地區。
與學術機構和全球 ALS 聯盟的合作正在促進臨床試驗機會的增加和數據的產生。

4. 新的適應症和監管進展

Ulefnersen 目前處於臨床開發階段，與監管機構的討論重點是研究設計、生物標記檢驗和加速核准途徑。
監管機構對 ALS 的創新終點和替代生物標記越來越持開放態度，這可能有助於未來的應用。
如果成功，這將使 ALS 的治療選擇多樣化，目前緩解疾病藥物選擇有限。

5. ALS研發勢頭強勁

肌萎縮側索硬化症（ALS）仍然是一個需求未被充分滿足的市場，治療選擇有限，對基於機制的干涉措施有強烈的需求。
越來越重視早期診斷、基因分層以及在神經神經退化晚期之前進行干預，這為urefnersen 的開發提供了理論基礎。
患者權益倡導團體的參與和真實世界自然史資料集正在推動試驗進展並幫助解釋終點。

6. 競爭差異化與市場趨勢

其基於反義寡核苷酸 (ASO) 的作用機制能夠選擇性地調節與疾病相關的靶點，使烏列夫納森區別於小分子療法和症狀治療。
由於其作用於中樞神經系統標靶的潛力，烏列夫納森有望成為下一代神經退化治療藥物。
精準神經學、生物標記主導的開發和適應性試驗設計等更廣泛的趨勢支持其長期定位。
預計對真實世界數據和長期 ALS 登記數據的依賴將補充臨床試驗結果。

urefnersen 的最新進展

2024年11月，大塚製藥與Ionis Pharmaceuticals公司達成一項全球獨家許可協議，授權其生產和銷售ulefnersen（通用名，研發代號ION363），該藥是一種用於治療肌萎縮側索硬化症（ALS）的候選藥物。此藥適用於FUS（融合肉瘤）基因突變引起的ALS患者。

《Ulefnersen銷售預測及市場規模分析（2034）》報告全面深入分析了Ulefnersen在七個主要市場（美國、四個歐盟國家（德國、法國、義大利和西班牙）、英國、加拿大和日本）的潛在適應症，例如肌萎縮側索硬化症（ALS）。本報告詳細展望了Ulefnersen在七個主要市場（美國、四個歐盟國家（德國、法國、義大利和西班牙）、英國和日本）的現有使用情況、預期市場准入以及在潛在適應症中的表現，研究週期為2020年至2034年。報告中還詳細描述了Ulefnersen的潛在適應症。此外，本Ulefnersen市場報告還深入分析了Ulefnersen的銷售預測、作用機制、劑量和給藥途徑，以及研發和其他開發活動，包括監管里程碑。此外，該報告還包括ulefnersen的歷史和當前表現、未來市場評估（包括對七個主要國家潛在適應症的ulefnersen市場預測分析）、SWOT分析、分析師觀點、市場競爭對手的全面概述，以及各適應症中其他新興治療方法的概述。報告還提供了ulefnersen銷售額的預測分析以及市場促進因素。

Urefnersen藥物概述

Ulefnersen（ION363，曾使用名jasifusen）是由Ionis Pharmaceuticals公司開發的第二代正向作用反義寡核苷酸（ASO）。它針對FUS基因突變引起的肌萎縮側索硬化症（ALS）（FUS-ALS），約佔所有ALS病例的0.3-0.9%，且往往發病早、進展迅速。此藥物透過鞘內注射標靶突變型FUS RNA轉錄本，透過RNA分解和翻譯抑制來抑制毒性FUS蛋白的表達。臨床前小鼠模型和擴大用藥病例均證實，可減少功能獲得性毒性、核定位異常和運動神經元退化。目前正在進行I/III期FUSION試驗（NCT04768972），以評估其安全性、功能衰退（ALSFRS-R評分）、神經絲輕鍊和腦脊髓液FUS水平等生物標記以及存活率。該藥物已獲得歐盟和美國的孤兒藥資格認定。儘管目前仍面臨一些挑戰，但早期仿單標示外用藥已顯示其可分佈至中樞神經系統，降低部分患者的功能衰退，並延緩功能衰退。本報告提供有關ulefnersen的銷售業績、成長障礙和促進因素、上市後狀況以及多種適應症的核准情況的資訊。

脲呋喃市場報告範圍

本報告深入分析了以下內容：

對 ulefnersen 進行全面的產品概述，包括其作用機制 (MoA)、產品說明、劑量和給藥方法，以及在肌萎縮側索硬化症 (ALS) 等潛在適應症方面的研究和開發活動。
本市場報告提供了有關 ulefnersen 監管里程碑和其他開發活動的詳細資訊。
該報告還重點介紹了 ulefnersen 在美國、歐洲和日本的成本估算和區域差異、已報告和估計的銷售額以及潛在適應症的研發活動。
ulefnersen 市場報告還涵蓋專利資訊、學名藥上市及其對降低成本的影響。
ulefnersen 市場報告包括 ulefnersen 在潛在適應症方面的當前和預測銷售額，直至 2034 年。
此外，也全面介紹了每種適應症中新興的後期治療方法。
ulefnersen 市場報告還包含 SWOT 分析，分析師對 ulefnersen 在各種潛在適應症方面的看法。

調查方法：

這份尿道市場報告主要基於從內部資料庫、一手和二手研究以及DelveInsight行業專家團隊的內部分析中獲得的數據和資訊。二級資訊來源資訊和資料來自各種印刷和非印刷管道，例如搜尋引擎、新聞網站、全球監管機構網站、行業期刊、白皮書、雜誌、書籍、行業協會、貿易入口網站以及對現有資料庫的存取。

DelveInsight 的 Ulefnellsen 分析觀點

對尿道奈菲森市場的詳細評估

這份關於烏勒夫納森（ulefnersen）銷售市場預測的報告，詳細評估了烏勒夫納森在七個主要市場（美國、四個歐盟國家（德國、法國、義大利、西班牙）、英國和日本）的市場狀況，重點關注其在肌萎縮側索硬化症（ALS）等潛在適應症方面的應用。報告的這一部分提供了烏勒夫納森截至2034年的當前銷售數據和預測銷售數據。

烏列夫納森的臨床評價

ulefnersen 市場報告提供了 ulefnersen 針對潛在適應症的臨床試驗資訊，包括研究干預措施、研究條件、實驗狀況、開始和完成日期。

Refnelsen的競爭格局

本報告深入分析了該領域的競爭對手和市售產品，並概述了新興產品及其預計上市日期，這些產品將推動市場出現激烈的競爭。

ulefnersen 的市場潛力和收入預測

烏列夫納森及其主要適應症的預期市場規模
ulefnersen 的預計銷售潛力（銷售尖峰時段預測）
urefnersen 的定價策略和報銷狀況

Urefnelsen 的競爭訊息

在研競爭藥物數量（研發管線分析）
與現有治療方法相比，ulefnersen 的市場定位
相對於競爭對手，urefnersen 的優勢和劣勢

ulefnersen 的監管和商業里程碑

ulefnersen 的關鍵監管核准和預計上市時間
商業合作、授權協議和併購活動

尿素的臨床鑑別診斷

與現有藥物相比，ulefnersen 具有更優的療效和安全性。
Urefnersen 的獨特優勢

Urefnellsen市場報告要點

未來幾年，由於 ulefnersen 的廣泛應用、處方箋的增加以及在多種免疫學適應症中的更廣泛採用，預計其市場格局將發生變化，這將推動市場規模的成長。
ulefnersen相關企業致力於開發以治療和改善疾病狀態的新方法為重點的治療方法，評估可能影響 ulefnersen 優勢的挑戰和機會。
預計其他用於治療肌萎縮側索硬化症 (ALS) 的新興產品將對 ulefnersen 構成激烈的市場競爭，而近期推出的後期治療方法可能會對市場產生重大影響。
對監管里程碑和開發活動的詳細描述，展現了 ulefnersen 在潛在適應症方面的當前開發狀況。
分析 ulefnersen 的成本、定價趨勢和市場定位，以支持免疫學領域的策略決策。
我們對 ulefnersen 銷售預測數據（截至 2034 年）的詳細分析將透過確定 ulefnersen 在潛在適應症方面的整體情況，幫助您制定治療方案決策流程。

烏列夫納森在潛在適應症的市場前景
對七個主要國家的分析
- 烏列夫納森在七個主要國家的潛在適應症市場規模
各國市場
- 烏列夫納森在美國潛在適應症的市場規模
- 烏列夫納森在德國潛在適應症的市場規模
- 在英國，ulefnersen 的潛在適應症市場規模

第6章尤雷夫納森的SWOT分析

第7章分析師觀點

第8章附錄

第9章 DelveInsight 服務

第10章免責聲明

第11章關於 DelveInsight

第12章報告購買選項

簡介目錄圖表

Product Code: DIDM1536

Key Factors Driving Ulefnersen Growth

1. Market Share Gains and New Patient Starts

Ulefnersen is building early momentum within the ALS research and clinical development landscape, driven by rising interest in genetically targeted therapies.
Patient enrollment in clinical studies is increasing, reflecting strong engagement from specialized ALS centers and growing genetic screening in ALS populations.
Sponsor-driven education and collaboration with key opinion leaders (KOLs) are supporting awareness and positioning Ulefnersen as a promising disease-modifying candidate rather than symptomatic therapy.

2. Expansion Across Key Indications

Amyotrophic Lateral Sclerosis (ALS): Primary focus on genetically defined ALS subpopulations, targeting upstream disease mechanisms rather than downstream symptom control.
Genetically Stratified Neurodegenerative Disease: The mechanism supports potential applicability in broader neurodegenerative disorders where similar molecular pathways are implicated.
Precision Medicine Strategy: Pipeline expansion is expected to remain biomarker- and genotype-driven, aligning with trends toward personalized neurology care.
Ongoing translational research may enable future indication expansion pending proof-of-concept efficacy.

3. Geographic Expansion

Clinical development is concentrated in North America and Europe, where advanced ALS trial infrastructure and genetic testing capabilities are well established.
Expansion into additional geographies is anticipated as global ALS trial networks strengthen and regulatory engagement progresses.
Collaboration with academic institutions and global ALS consortia is supporting broader trial accessibility and data generation.

4. New Indication and Regulatory Progress

Ulefnersen remains in clinical development, with regulatory interactions focused on trial design, biomarker validation, and accelerated approval pathways.
Regulatory agencies have shown increasing openness to innovative endpoints and surrogate biomarkers in ALS, which may support future filings.
Successful progression could diversify the ALS treatment landscape, which currently has limited disease-modifying options.

5. Strong ALS Research and Development Momentum

ALS remains a high-unmet-need market, with limited therapeutic choices and strong demand for mechanism-based interventions.
Growing emphasis on early diagnosis, genetic stratification, and intervention prior to advanced neurodegeneration supports Ulefnersen's development rationale.
Trial momentum is reinforced by patient advocacy involvement and real-world natural history datasets, aiding endpoint interpretation.

6. Competitive Differentiation and Market Trends

Antisense oligonucleotide (ASO)-based mechanism enables selective modulation of disease-relevant targets, differentiating Ulefnersen from small-molecule and symptomatic therapies.
Potential for central nervous system target engagement positions Ulefnersen among next-generation neurodegenerative therapeutics.
Broader trends such as precision neurology, biomarker-driven development, and adaptive trial designs favor its long-term positioning.
Increasing reliance on real-world evidence and longitudinal ALS registries is expected to complement clinical trial findings.

Ulefnersen Recent Developments

In November 2024, Otsuka Pharmaceutical announced an exclusive worldwide licensing agreement with Ionis Pharmaceuticals for rights to manufacture and market Ionis' Ulefnersen (generic name; development code is ION363), a drug candidate under development for the treatment of patients with amyotrophic lateral sclerosis (ALS) caused by mutation of the fused in sarcoma (FUS) gene.

"Ulefnersen Sales Forecast, and Market Size Analysis - 2034" report provides comprehensive insights of Ulefnersen for potential indication like Amyotrophic lateral sclerosis in the 7MM. A detailed picture of Ulefnersen's existing usage in anticipated entry and performance in potential indications in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2020 -2034 is provided in this report along with a detailed description of the Ulefnersen for potential indications. The Ulefnersen market report provides insights about Ulefnersen's sales forecast, mechanism of action (MoA), dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of historical and current Ulefnersen performance, future market assessments inclusive of the Ulefnersen market forecast analysis for potential indications in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in respective indications. It also provides analysis of Ulefnersen sales forecasts, along with factors driving its market.

Ulefnersen Drug Summary

Ulefnersen (also known as ION363 or formerly Jacifusen) is an investigational generation 2+ antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene (FUS-ALS), which accounts for about 0.3-0.9% of cases and often presents with early-onset, aggressive progression. It targets the mutant FUS RNA transcript via intrathecal administration, reducing toxic FUS protein expression through RNA degradation and translation inhibition, thereby mitigating gain-of-function toxicity, nuclear mislocalization, and motor neuron degeneration as shown in preclinical mouse models and expanded access cases. Currently in Phase I/III FUSION trial (NCT04768972) evaluating safety, functional decline (ALSFRS-R), biomarkers like neurofilament light and CSF FUS levels, and survival, it has orphan drug status in the EU and US, with early compassionate use demonstrating CNS distribution, FUS reduction, and slowed decline in select patients despite ongoing challenges. The report provides Ulefnersen's sales, growth barriers and drivers, post usage and approvals in multiple indications.

Scope of the Ulefnersen Market Report

The report provides insights into:

A comprehensive product overview including the Ulefnersen MoA, description, dosage and administration, research and development activities in potential indication like Amyotrophic lateral sclerosis.
Elaborated details on Ulefnersen regulatory milestones and other development activities have been provided in Ulefnersen market report.
The report also highlights Ulefnersen's cost estimates and regional variations, reported and estimated sales performance, research and development activities in potential indications across the United States, Europe, and Japan.
The Ulefnersen market report also covers the patents information, generic entry and impact on cost cut.
The Ulefnersen market report contains current and forecasted Ulefnersen sales for potential indications till 2034.
Comprehensive coverage of the late-stage emerging therapies for respective indications.
The Ulefnersen market report also features the SWOT analysis with analyst views for Ulefnersen in potential indications.

Methodology:

The Ulefnersen market report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight's team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.

Ulefnersen Analytical Perspective by DelveInsight

In-depth Ulefnersen Market Assessment

This Ulefnersen sales market forecast report provides a detailed market assessment of Ulefnersen for potential indication like Amyotrophic lateral sclerosis in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides current and forecasted Ulefnersen sales data uptil 2034.

Ulefnersen Clinical Assessment

The Ulefnersen market report provides the clinical trials information of Ulefnersen for potential indications covering trial interventions, trial conditions, trial status, start and completion dates.

Ulefnersen Competitive Landscape

The report provides Insights on competitors and marketed products within the domain, along with a summary of emerging products and their respective launch dates, posing significant competition in the market.

Ulefnersen Market Potential & Revenue Forecast

Projected market size for the Ulefnersen and its key indications
Estimated Ulefnersen sales potential (Ulefnersen peak sales forecasts)
Ulefnersen Pricing strategies and reimbursement landscape

Ulefnersen Competitive Intelligence

Number of competing drugs in development (pipeline analysis)
Ulefnersen Market positioning compared to existing treatments
Ulefnersen Strengths & weaknesses relative to competitors

Ulefnersen Regulatory & Commercial Milestones

Ulefnersen Key regulatory approvals & expected launch timelines
Commercial partnerships, licensing deals, and M&A activity

Ulefnersen Clinical Differentiation

Ulefnersen Efficacy & safety advantages over existing drugs
Ulefnersen Unique selling points

Ulefnersen Market Report Highlights

In the coming years, the Ulefnersen market scenario is set to change due to strong adoption, increased prescriptions and broader uptake in multiple immunological indications; which would expand the size of the market.
The Ulefnersen companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence Ulefnersen's dominance.
Other emerging products for Amyotrophic lateral sclerosis are expected to give tough market competition to Ulefnersen and launch of late-stage emerging therapies in the near future will significantly impact the market.
A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of Ulefnersen in potential indications.
Analyse Ulefnersen cost, pricing trends and market positioning to support strategic decision-making in the immunology landscape.
Our in-depth analysis of the forecasted Ulefnersen sales data uptil 2034 will support the clients in decision-making process regarding their therapeutic portfolio by identifying the overall scenario of Ulefnersen in potential indications.

Key Questions:

What is the class of therapy, route of administration and mechanism of action of Ulefnersen? How strong is Ulefnersen's clinical and commercial performance?
What is Ulefnersen's clinical trial status in each individual indications such as Amyotrophic lateral sclerosis and study completion date?
What are the key collaborations, mergers and acquisitions, licensing and other activities related to the Ulefnersen Manufacturers?
What are the key designations that have been granted to Ulefnersen for potential indications? How are they going to impact Ulefnersen's penetration in various geographies?
What is the current and forecasted Ulefnersen market scenario for potential indications? What are the key assumptions behind the forecast?
What are the current and forecasted sales of Ulefnersen in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain) and the United Kingdom, and Japan?
What are the other emerging products available and how are these giving competition to Ulefnersen for potential indications?
Which are the late-stage emerging therapies under development for the treatment of potential indications?
How cost-effective is Ulefnersen? What is the duration of therapy and what are the geographical variations in cost per patient?

1. Report Introduction

2. Ulefnersen Overview in potential indication like Amyotrophic lateral sclerosis

2.1. Product Detail
2.2. Ulefnersen Clinical Development
- 2.2.1. Ulefnersen Clinical studies
- 2.2.2. Ulefnersen Clinical trials information
- 2.2.3. Safety and efficacy
2.3. Other Developmental Activities
2.4. Product Profile

3. Ulefnersen Competitive Landscape (Marketed Therapies)

4. Competitive Landscape (Late-stage Emerging Ulefnersen Therapies)

5. Ulefnersen Market Assessment

5.1. Ulefnersen Market Outlook in potential indications
5.2. 7MM Analysis
- 5.2.1. Ulefnersen Market Size in the 7MM for potential indications
5.3. Country-wise Market Analysis
- 5.3.1. Ulefnersen Market Size in the United States for potential indications
- 5.3.2. Ulefnersen Market Size in Germany for potential indications
- 5.3.3. Ulefnersen Market Size in France for potential indications
- 5.3.4. Ulefnersen Market Size in Italy for potential indications
- 5.3.5. Ulefnersen Market Size in Spain for potential indications
- 5.3.6. Ulefnersen Market Size in the United Kingdom for potential indications
- 5.3.7. Ulefnersen Market Size in Japan for potential indications