With porphyria increasingly affecting populations worldwide and contributing to comorbidities such as diabetes, cardiovascular disease, and certain cancers, the demand for safer and more effective treatments is growing. According to DelveInsight, the porphyria pipeline includes over 10 pharmaceutical and biotech companies actively developing more than 10 therapeutic candidates. These therapies are at various stages of clinical and preclinical development, highlighting significant innovation and dedication to tackling this critical public health challenge.
DelveInsight’s “Porphyria Pipeline Insight 2025” report offers a comprehensive and strategic review of the current R&D landscape. It examines clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report is an essential resource for stakeholders—including researchers, healthcare investors, and decision-makers—seeking insights into the evolving porphyria therapeutics market and the innovations shaping its future.
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Key Takeaways from the Porphyria Pipeline Report
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DelveInsight’s porphyria pipeline report illustrates a vibrant landscape, featuring more than 10 active companies developing over 10 therapeutic candidates for porphyria.
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In September 2021, Disc Medicine raised $90 million in Series B funding to advance its pipeline, including bitopertin, an oral GlyT1 inhibitor in Phase II trials for erythropoietic porphyrias, with the potential to become the first disease-modifying therapy.
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Key players driving innovation in porphyria include Clinuvel Pharmaceuticals, Mitsubishi Tanabe Pharma, Gilead Sciences, Disc Medicine, Atlas Molecular Pharma, and others, all working to expand and improve treatment options.
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Prominent pipeline candidates under development include Dersimelagon, DISC-0974, and several additional therapies at various stages of clinical investigation.
Porphyria Overview:
Erythropoietic Protoporphyria (EPP) is a hereditary disorder that affects the heme production pathway, most frequently caused by mutations in the ferrochelatase (FECH) gene. A less common form, X-Linked Protoporphyria (XLP), results from mutations in the aminolevulinic acid synthase-2 (ALAS2) gene. Both conditions cause protoporphyrin to build up in the blood, red blood cells, and tissues, leading to increased skin sensitivity to light. Symptoms typically appear in early childhood and include severe, painful reactions to sunlight, often preceded by tingling, stinging, or burning sensations on exposed skin. These reactions can occur within 10 minutes of sun exposure.
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Porphyria Pipeline Analysis
The Porphyria pipeline insights report 2025, provides insights into:
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Provides comprehensive insights into key companies developing therapies in the Porphyria Market.
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Categorizes Porphyria therapeutic companies by development stage: early, mid, and late-stage.
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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.
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Reviews emerging Porphyria drugs under development based on:
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Stage of development
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Porphyria Route of administration
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Target receptor
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Monotherapy vs. combination therapy
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Porphyria Mechanism of action
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Molecular type
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Offers detailed analysis of:
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Company-to-company and company-academia collaborations
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Porphyria Licensing agreements
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Funding and investment activities supporting future Porphyria market advancement.
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Unlock key insights into emerging Porphyria therapies and market strategies here: https://www.delveinsight.com/report-store/porphyria-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr
Porphyria Emerging Drugs
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Dersimelagon: Mitsubishi Tanabe Pharma
Dersimelagon is an innovative, orally administered, non-peptide small molecule that selectively activates the melanocortin-1 receptor (MC1R). It has demonstrated potential in preventing phototoxic reactions in patients with erythropoietic protoporphyria (EPP). Mitsubishi Tanabe Pharma Corporation (MTPC) is developing dersimelagon for EPP treatment. In the Phase II ENDEAVOR trial, MT-7117 significantly prolonged the time to onset of prodromal sunlight-related symptoms at week 16, meeting its primary endpoint. The therapy was generally well tolerated and showed a favorable safety profile. In June 2018, the FDA granted Fast Track Designation to dersimelagon.
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DISC-0974: Disc Medicine
DISC-0974 is a novel monoclonal antibody that targets the hemojuvelin (HJV) co-receptor, a crucial regulator of the hepcidin pathway. Hepcidin is the primary hormone controlling iron balance by limiting both iron absorption and release from storage. DISC-0974 is being developed to treat anemia of inflammation by lowering hepcidin levels, thereby improving iron availability for red blood cell production. Preclinical studies and human genetic data indicate that inhibiting HJV effectively decreases hepcidin and raises serum iron levels.
Porphyria Pipeline Therapeutic Assessment
Porphyria Assessment by Product Type
• Mono
• Combination
• Mono/Combination
Porphyria By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
Porphyria Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
Porphyria Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
Download sample pages to get an in-depth assessment of the emerging Porphyria therapies and key Porphyria companies
Table of Contents
1. Report Introduction
2. Executive Summary
3. Porphyria Current Treatment Patterns
4. Porphyria - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Porphyria Late-Stage Products (Phase-III)
7. Porphyria Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Porphyria Discontinued Products
13. Porphyria Product Profiles
14. Porphyria Key Companies
15. Porphyria Key Products
16. Dormant and Discontinued Products
17. Porphyria Unmet Needs
18. Porphyria Future Perspectives
19. Porphyria Analyst Review
20. Appendix
21. Report Methodology
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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