Retinoic Acid Receptor-Y (RAR-Y) Targeted Therapies - Target Population, Competitive Landscape, and Market Forecast - 2034

Key Highlights:

• RAR-Y targeted therapies selectively activate retinoic acid receptor gamma (RAR-Y), a nuclear receptor primarily expressed in skin, cartilage, and bone. This activation regulates cell differentiation and tissue remodeling, offering precise therapeutic effects with minimal systemic side effects.
• These therapies are showing broad therapeutic promise across multiple indications, including rare skeletal disorders such as Fibrodysplasia Ossificans Progressiva (FOP), dermatological conditions like acne vulgaris, and a growing range of solid tumors, underscoring their relevance in underserved, high-need medical areas.
• To date, only two RAR-Y targeted therapies have been approved: SOHONOS (Ipsen) for FOP, AKLIEF (Galderma) for acne vulgaris. These approvals mark an important step toward validating the clinical relevance of RAR-Y modulation in rare bone and skin disorders.
• Several companies are actively developing RAR-Y therapies. Ipsen and Galderma lead in approved indications, while Io Therapeutics is advancing pipeline candidates that target RAR-Y for use in oncology and other areas.
• IRX5010 by Io Therapeutics is the only emerging RAR-Y targeted therapy in oncology. It is currently in Phase I clinical trials for multiple solid tumors, including triple-negative breast cancer (TNBC), non-small cell lung cancer (NSCLC), colon cancer, and prostate cancer.
• In December 2024, Io Therapeutics presented promising preclinical data for IRX5010 at the San Antonio Breast Cancer Symposium, demonstrating its potential to treat triple-negative breast cancer, a subtype with limited therapeutic options.
• Preclinical models suggest that IRX5010 may modulate the tumor microenvironment and enhance anti-tumor immunity, offering a novel mechanism of action distinct from conventional chemotherapies.
• As the RAR-Y therapeutic class matures, ongoing clinical and translational research is expected to expand its applications, particularly in oncology, where RAR-Y modulation could provide a targeted approach to treatment-resistant cancers.

DelveInsight's "RAR-Y targeted therapies - Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the RAR-Y targeted therapies, historical and competitive landscape as well as its market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The RAR-Y targeted therapies' market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM RAR-Y targeted therapies' market size from 2020 to 2034. The report also covers current RAR-Y targeted therapies' treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

RAR-Y targeted therapies Understanding and Treatment Algorithm

RAR-Y targeted therapies Overview

RAR-Y targeted therapies are selective compounds that activate the gamma subtype of retinoic acid receptors, which are nuclear receptors involved in regulating gene expression. RAR-Y is predominantly found in tissues like skin, cartilage, and bone, where it plays a crucial role in controlling cell differentiation, proliferation, and tissue remodeling. By binding to RAR-Y, these targeted therapies influence specific genetic pathways that can suppress abnormal cell growth, reduce inflammation, or promote healthy tissue regeneration. This targeted action helps in treating diseases where these processes are disrupted, such as excessive bone formation, cartilage degeneration, or abnormal skin turnover. Their selectivity allows for focused therapeutic effects in affected tissues while limiting broader systemic impact.

RAR-Y Targeted Therapies Market Overview

RAR-Y targeted therapies are emerging as promising therapies across diverse indications, including FOP, acne vulgaris, and multiple solid tumors. Their unique mechanism of selectively modulating retinoid signaling pathways offers targeted therapeutic benefits with potentially fewer side effects. The growing clinical validation in rare skeletal disorders, dermatology, and oncology signals a positive shift in the treatment landscape, expanding therapeutic possibilities and driving momentum across high-need, underserved markets.

RAR-Y targeted therapies Epidemiology

The epidemiology chapter of RAR-Y targeted therapies in the report provides historical as well as forecasted epidemiology segmented as total cases in selected indications for RAR-Y targeted therapies, total eligible patient pool in selected indications for RAR-Y targeted therapies, and total treated cases in selected indications for RAR-Y targeted therapies in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034.

• In the US, there were about 70mn prevalent cases of FOP in 2024.
• In 2024, HER2-negative represented the highest number of cases among all TNBC types across the 7MM.
• In Japan, colorectal cancer affects more males than females, with approximately 87,000 cases in males and 68,000 in females.

RAR-Y targeted Therapies Drug Chapters

The drug chapter segment of the RAR-Y targeted therapies report encloses a detailed analysis of approved RAR-Y targeted therapies, late-stage (Phase III and Phase II) RAR-Y targeted therapies. It also helps understand the clinical trial details of RAR-Y targeted therapies, expressive pharmacological action, agreements and collaborations, approval, and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Marketed RAR-Y Targeted Therapies

SOHONOS (palovarotene): Ipsen Pharmaceuticals

SOHONOS is an oral medicine with particular selectivity for the gamma subtype of retinoic-acid receptors, which are an important regulator of skeletal development and ectopic bone in the retinoid signaling pathway. The medicine is designed to mediate the interactions between the receptors, growth factors, and proteins within the retinoid signaling pathway to reduce new abnormal bone formation in FOP.

SOHONOS received an Orphan Drug and Breakthrough Therapy Designation from the US FDA for the treatment of FOP and was granted a Priority Review of the NDA. SOHONOS is also under review with several other regulatory authorities. In July 2023, the European Commission did not grant marketing authorization for SOHONOS. It was approved in August 2023 for the reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with FOP. With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher. The voucher can be used for subsequent drug applications that would not qualify for a priority review.

AKLIEF (trifarotene): Galderma

AKLIEF Cream is a retinoid indicated for the topical treatment of acne vulgaris in patients 9 years of age and older. In October 2019, Galderma announced that the US FDA approved AKLIEF Cream, 0.005% for the topical treatment of acne. Trifarotene is the first new retinoid molecule to receive US FDA approval for the treatment of acne in more than 20 years.

In December 2019, Galderma announced that AKLIEF (trifarotene 50 mcg/g cream) has concluded its European decentralized procedure resulting in the approval of its summary of product characteristics, package leaflet, and labeling, by all involved European member states. Each of the 16 concerned member states will now issue individual national marketing authorization for the cutaneous treatment of acne vulgaris of the face and/or the trunk in patients from 12 years of age and older when many comedones, papules, and pustules are present.

Emerging RAR-Y Targeted Therapies

IRX5010: Io Therapeutics

IRX5010 is an investigational oral anti-cancer agent developed by Io Therapeutics, acting as a selective RAR-Y targeted therapies. Unlike traditional chemotherapies, IRX5010 works by modulating the tumor microenvironment to enhance anti-tumor immunity. It has shown preclinical efficacy in multiple cancer models, including triple-negative breast cancer, NSCLC, colon cancer, and prostate cancer.

Data presented at the SITC 2024 meeting demonstrated that IRX5010 significantly suppressed tumor growth in mice by increasing effector memory T-cell infiltration and reducing immunosuppressive myeloid-derived suppressor cells (MDSCs). Furthermore, at the 2024 San Antonio Breast Cancer Symposium, combining IRX5010 with an anti-PD-L1 checkpoint inhibitor led to an 84% tumor reduction in a triple-negative breast cancer model, showing synergistic immune activation. These promising results support IRX5010's advancement into clinical trials as a monotherapy and in combination with immunotherapies for various cancers.

Currently, it is in Phase I of the development process for the treatment of triple-negative breast cancer, NSCLC, prostate cancer, and colon cancer.

RAR-Y Targeted Therapies Market Outlook

The RAR-Y targeted therapies space is gaining momentum with approvals in both rare and common conditions. SOHONOS, approved in 2023 for FOP, and AKLIEF (trifarotene), approved in 2019 for acne, have validated the therapeutic and regulatory potential of this class. Now, oncology is emerging as the next frontier, with IRX5010 in Phase I trials for multiple cancers showing promising immune-mediated tumor suppression. As the class expands beyond dermatology and skeletal disorders into immuno-oncology, RAR-Y targeted therapies are positioned for broader clinical adoption and commercial growth.

RAR-Y targeted therapies Drugs Uptake

This section focuses on the uptake rate of potential approved and emerging RAR-Y targeted therapies expected to be launched in the market during 2025-2034.

RAR-Y targeted therapies Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key players involved in developing targeted therapeutics. The presence of numerous drugs under different stages is expected to generate immense opportunity for RAR-Y targeted therapies market growth over the forecast period.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for RAR-Y targeted therapies.

KOL Views

To keep up with current and future market trends, we take Industry Experts' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on RAR-Y targeted therapies' evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.

DelveInsight's analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as Washington University and others.

Their opinion helps understand and validate current and emerging therapy treatment patterns or RAR-Y targeted therapies market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health Technology Assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug.

In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs including Medicare, Continuing Medical Education (CME) program, the Children's Health Insurance Program (CHIP), and the state and federal health insurance marketplaces are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs), and third-party organizations that provide services, and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Key Updates on RAR-Y Targeted Therapies

• In December 2024, Io Therapeutics unveiled promising preclinical results for its novel compound IRX5010 during a presentation at the San Antonio Breast Cancer Symposium, highlighting its potential as a treatment for triple-negative breast cancer.
• In August 2023, the US FDA approved Ipsen's SOHONOS capsules, the first and only treatment for people with FOP. It also has been granted ODD and BTD by the US FDA.
• In October 2019, Galderma announced that the US FDA approved AKLIEF Cream, 0.005% for the topical treatment of acne. Later in the same year, Galderma announced that AKLIEF (trifarotene 50 mcg/g cream) had concluded its European decentralized procedure resulting in the approval of its summary of product characteristics, package leaflet, and labeling, by all involved European member states.

The abstract list is not exhaustive and will be provided in the final report.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of the RAR-Y targeted therapies, explaining its mechanism, and therapies (current and emerging).
• Comprehensive insight into the competitive landscape, and forecasts, the future growth potential of treatment rate, drug uptake, and drug information have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current landscape.
• A detailed review of the RAR-Y targeted therapies market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM RAR-Y targeted therapies market.

RAR-Y Targeted Therapies Report Insights

• RAR-Y targeted therapies Targeted Patient Pool
• Therapeutic Approaches
• RAR-Y targeted therapies Pipeline Analysis
• RAR-Y targeted therapies Market Size and Trends
• Existing and future Market Opportunities

RAR-Y Targeted Therapies Report Key Strengths

• 10 years Forecast
• The 7MM Coverage
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

RAR-Y Targeted Therapies Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT)

Key Questions:

• What was the total market size of RAR-Y targeted therapies, the market size by therapies, market share (%) distribution, and what would it look like in 2034? What are the contributing factors for this growth?
• Which drug is going to be the largest contributor in 2034?
• Which is the most lucrative market for RAR-Y targeted therapies?
• What are the pricing variations among different geographies for approved therapies?
• How the reimbursement landscape has for RAR-Y targeted therapies evolved since the first one was approved? Do patients have any access issues that are driven by reimbursement decisions?
• What are the risks, burdens, and unmet needs of treatment with RAR-Y targeted therapies? What will be the growth opportunities across the 7MM for the patient population of RAR-Y targeted therapies?
• What are the key factors hampering the growth of the market for RAR-Y targeted therapies?
• What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
• What key designations have been granted to the therapies for RAR-Y targeted therapies?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the RAR-Y targeted therapies market.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of indication-wise current and emerging therapies under the conjoint analysis section to provide visibility around leading indications.
• Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.