Erythropoietic protoporphyria (EPP) is a rare genetic disorder causing excessive accumulation of protoporphyrin, leading to severe photosensitivity and skin damage. According to Yinan Wang et al., 2024, the global prevalence of EPP ranges from 1 in 75,000 to 1 in 200,000 individuals. Current therapies focus on symptom management and photoprotection, with afamelanotide being a leading approved treatment, alongside supportive options like oral beta-carotene. The growing focus on novel therapies and gene-targeted treatments is driving research and development in the EPP drug pipeline. According to the erythropoietic protoporphyria pipeline analysis by Expert Market Research, the therapeutic landscape is anticipated to expand steadily in the coming years.
Major companies involved in the erythropoietic protoporphyria pipeline analysis include Disc Medicine, Inc., Mitsubishi Tanabe Pharma America Inc., and others.
Leading drugs currently in the pipeline include DISC-1459, MT-7117, PORT-77, and others.
The pipeline shows robust growth driven by innovative gene therapies, targeted photodynamic treatments, and increasing clinical trial activities. Advancements in delivery methods and regulatory support are expected to accelerate development.
The Erythropoietic Protoporphyria Pipeline Analysis Report by Expert Market Research gives comprehensive insights into erythropoietic protoporphyria therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for erythropoietic protoporphyria. The erythropoietic protoporphyria report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The erythropoietic protoporphyria pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with erythropoietic protoporphyria treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to erythropoietic protoporphyria.
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Erythropoietic protoporphyria (EPP) is a rare genetic metabolic disorder caused by mutations affecting heme synthesis. This defect results in excess protoporphyrin in red blood cells, causing severe phototoxic reactions even after brief light exposure. Patients often experience burning, swelling, and long-lasting skin damage, leading to social withdrawal to avoid sunlight and artificial light.
Erythropoietic protoporphyria treatments aim to prevent phototoxicity and improve quality of life. Scenesse® (afamelanotide), a subcutaneous controlled-release implant, provides systemic photoprotection for adult patients and is administered every two months under medical supervision. In October 2020, Clinuvel Pharmaceuticals announced that the Australian Therapeutic Goods Administration (TGA) approved Scenesse® for preventing phototoxicity in adult EPP patients. This landmark approval introduces the first systemic therapy in Australia, following a nine-month priority review, with healthcare professional training and long-term patient safety monitoring in place.
As per Yinan Wang et al., 2024, erythropoietic protoporphyria (EPP) has a global prevalence ranging from 1 in 75,000 to 1 in 200,000, with Europe reporting 9.2 cases per 1,000,000. According to Herbert L. Bonkovsky et al., 2025, underdiagnosis may lead to higher observed rates, as in the UK Biobank study, indicating 1 in 17,000. About 10% of patients develop chronic liver disease, while pigment gallstones are common. These insights highlight the critical need for continued drug development and targeted therapies for EPP.
This section of the report covers the analysis of erythropoietic protoporphyria drug candidates based on several segmentations, including:
By Phase
By Drug Class
By Route of Administration
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase III with 57%, covers a major share of the total erythropoietic protoporphyria clinical trials, highlighting the advanced development of key therapies. Phase II contributes 28%, while phase I holds 14%. The strong presence in Phase III and II reflects significant clinical progress, indicating promising market growth opportunities and potential future product approvals.
The drug molecule categories covered under the erythropoietic protoporphyria pipeline analysis include small molecules, peptides, biologics, gene therapies, and glycine transport inhibitors. The erythropoietic protoporphyria report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for erythropoietic protoporphyria. Small molecule therapies are gaining attention for their potential to manage phototoxicity symptoms. For instance, in March 2024, Dersimelagon (MT-7117), an orally administered selective melanocortin 1 receptor agonist, was under evaluation in a Phase 3 study. It was designed to increase sunlight tolerance in patients with EPP or X-linked protoporphyria, addressing a critical unmet need beyond sun protection.
The EMR report for the erythropoietic protoporphyria pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed erythropoietic protoporphyria therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in erythropoietic protoporphyria clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for erythropoietic protoporphyria. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of erythropoietic protoporphyria drug candidates.
DISC-1459 (Bitopertin) is an investigational, orally administered GlyT1 inhibitor, currently being developed by Disc Medicine, Inc and is working to modulate heme biosynthesis in participants with erythropoietic protoporphyria (EPP). The study is examining the safety, tolerability, and efficacy of Bitopertin, which works by regulating glycine availability in developing red blood cells. This Phase 2/3 open-label, long-term extension study is enrolling 200 participants and is continuing through 2028.
Dersimelagon (MT-7117) is a novel, orally administered, non-peptide small molecule acting as a selective melanocortin 1 receptor (MC1R) agonist. Developed by Mitsubishi Tanabe Pharma America Inc., this Phase 3 extension study is evaluating the long-term safety and tolerability of dersimelagon in patients with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP). The study is examining the drug’s ability to increase pain-free light exposure in individuals with a history of phototoxicity, aiming to provide a potential therapeutic option for these rare conditions.
PORT-77, an orally bioavailable inhibitor of the ABCG2 (BCRP) transport protein, is specifically being developed for the treatment of adults with erythropoietic protoporphyria (EPP). Sponsored by Portal Therapeutics, Inc., this Phase 2a study is examining the safety, tolerability, and efficacy of PORT-77 compared to placebo. The study is actively assessing how PORT-77 reduces protoporphyrin accumulation and phototoxic reactions, aiming to improve patient outcomes in EPP. The trial is ongoing and enrolling 14 participants, with completion expected in January 2026.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Erythropoietic Protoporphyria Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for erythropoietic protoporphyria. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into erythropoietic protoporphyria collaborations, regulatory environments, and potential growth opportunities.
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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