#USA #Jersey City #EPP #Dersimelagon #XLP

Tanabe Pharma America Announces Positive Results for Dersimelagon

Tanabe Pharma America, Inc., a fully owned subsidiary of Tanabe Pharma Corporation, has recently unveiled encouraging topline results from its pivotal Phase 3 clinical trial, known as the INSPIRE study. This trial evaluated Dersimelagon (MT-7117), a selective agonist of the melanocortin-1 receptor (MC1R), designed for the treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP) in adult and adolescent patients.

Understanding EPP and XLP

EPP and XLP are rare genetic disorders that lead to significant challenges for patients due to their debilitating symptoms. Patients with these conditions suffer from severe skin pain upon exposure to sunlight and certain artificial lights, resulting in a profound impact on their daily lives. EPP affects around 1 in 75,000 to 1 in 200,000 individuals in Europe, although the prevalence in the United States remains unclear.

Bijan Nejadnik, Head of Global Development and Regulatory Affairs, highlighted the critical lack of treatment options for adults with EPP and XLP, particularly emphasizing the distress faced by adolescents due to the absence of any approved therapies. "The limited treatment options... have created a care deficit, forcing many to endure severe life disruptions," he noted.

Key Insights from the INSPIRE Study

The INSPIRE study was a comprehensive, randomized, double-blind, placebo-controlled trial involving 165 participants aged 12 to 75 years. Participants were assigned to either receive Dersimelagon (200 mg once daily) or a placebo over 16 weeks. The study's primary goal was to measure the time until the first appearance of prodromal symptoms—such as burning, tingling, itching, or stinging—after sunlight exposure.

The results confirmed that Dersimelagon not only met its primary endpoint but also exhibited a favorable safety and tolerability profile. The majority of adverse events reported were categorized as mild or moderate, a promising indication for future use in this vulnerable population.

Following the double-blind period, a 36-week open-label extension study for continuous treatment is currently underway, further solidifying the commitment of Tanabe Pharma to support the porphyria community.

Fast Track and Orphan Drug Designation

Dersimelagon, still in its investigational phase, gained the Fast Track Designation from the U.S. FDA in June 2018, highlighting its potential to address serious conditions with unmet medical needs. Additionally, it received Orphan Drug Designation in June 2020 due to its focus on treating rare diseases.

The Future of EPP and XLP Treatment

Dersimelagon is a unique synthetic, orally administered medication that acts on the melanocortin-1 receptor, facilitating increased melanin production, thereby offering hope for more pain-free light exposure for patients affected by these conditions. Tanabe Pharma Corporation is dedicated to evaluating and developing therapies for EPP and XLP with the aim of improving lives and addressing patient concerns effectively.

Broader Implications for Patients

The results from the INSPIRE study not only provide valuable data to guide further research but also represent hope for individuals facing the severe challenges that EPP and XLP impose. Addressing such complex and painful conditions requires innovative solutions like Dersimelagon, which aligns with Tanabe Pharma's mission—to create hope for all facing illness.

For continuous updates on developments regarding Dersimelagon and its implications for EPP and XLP, please follow Tanabe Pharma America and their ongoing commitment to patient care and medical advancement.

About Tanabe Pharma

Founded in 1678, Tanabe Pharma Corporation is one of the oldest pharmaceutical companies globally, dedicated to advancing healthcare through innovative medical solutions. The company focuses on various therapeutic areas, emphasizing precision medicine and patient-centric approaches.