Porphyria Market

DelveInsight launched a new report on Porphyria Market Insights, Epidemiology, and Market Forecast-2032.

DelveInsight’s “Porphyria Market Report 2032” report delivers an in-depth understanding of the Porphyria, historical and forecasted epidemiology as well as the Porphyria market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

Porphyria: Overview

Erythropoietic Protoporphyria (EPP) is an inherited disorder of the heme biosynthetic pathway that results from mutations of the ferrochelatase (FECH) gene or, less commonly X-Linked Protoporphyria (XLP) that results from mutations in the aminolevulinic acid synthase-2 (ALAS2) gene. Both EPP and XLP are characterized by accumulation of protoporphyrin in blood, erythrocytes and tissues and cutaneous photosensitivity. EPP and XLP usually present early in childhood with extremely painful phototoxic reactions which are preceded by a “prodrome” of tingling, stinging, and/or burning of sun-exposed skin. The onset of prodromal symptoms after direct sun exposure varies but may occur in less than 10 minutes.

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Porphyria Key Companies:

• Clinuvel Pharmaceuticals

• Mitsubishi Tanabe Pharma

• Gilead Sciences

• Disc Medicine

• Atlas Molecular Pharma

Porphyria Emerging Drugs

Dersimelagon: Mitsubishi Tanabe Pharma

Dersimelagon is a novel synthetic, orally-administered, non-peptide small molecule, which acts as a selective agonist of melanocortin-1 receptor (MC1R) with a potential for being effective in the prevention of phototoxicity in erythropoietic protoporphyria (EPP) patients. Mitsubishi Tanabe Pharma Corporation (MTPC) is developing dersimelagon for the treatment of EPP. ENDEAVOR is a Phase II  proof of concept study for MT-7117 as a treatment option for EPP. ENDEAVOR met its primary endpoint of change from baseline in average daily time (minutes) to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at week 16. MT-7117 was generally well tolerated with an acceptable safety profile. Mitsubishi Tanabe Pharma Corporation received Fast Track Designation for dersimelagon by the U.S. Food and Drug Administration in June 2018

DISC-0974: Disc Medicine

DISC-0974 is an investigational, first-in-class monoclonal antibody designed to suppress hepcidin production by inhibiting the hemojuvelin (HJV) co-receptor, a highly selective and critical target of the hepcidin pathway. Hepcidin is the primary regulatory hormone of iron homeostasis and plays a central role by restricting iron absorption and preventing deployment from internal iron stores. DISC-0974 is being developed as a potential treatment for anemia of inflammation by suppressing hepcidin and enhancing iron availability for erythropoiesis. Preclinical studies of DISC-0974 and human genetic evidence validate that inhibition of HJV results in potent suppression of hepcidin and increased serum iron.

Porphyria Market Outlook

The Porphyria market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Porphyria market trends by analyzing the impact of current Porphyria therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

This segment gives a thorough detail of Porphyria market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Porphyria market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Porphyria market in 7MM is expected to witness a major change in the study period 2019-2032.

Table of contents

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