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Dersimelagon Phosphate

Recent clinical trials are investigating the use of Dersimelagon Phosphate, also known as MT-7117, for the treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). These rare genetic disorders cause extreme sensitivity to sunlight, leading to painful skin reactions. The studies aim to evaluate the drug’s efficacy, safety, and tolerability in improving patients’ ability to tolerate sunlight exposure and manage symptoms associated with these conditions.

Table of Contents

What is Dersimelagon Phosphate?

Dersimelagon Phosphate, also known as MT-7117, is an investigational drug being developed by Mitsubishi Tanabe Pharma America, Inc. It is currently being studied as a potential treatment for rare genetic disorders that cause extreme sensitivity to sunlight[1]. Dersimelagon is formulated as an oral tablet, making it a potentially convenient option for patients if approved[1].

What Conditions Does Dersimelagon Treat?

Dersimelagon is being investigated primarily for the treatment of two closely related conditions:

  • Erythropoietic Protoporphyria (EPP): A rare genetic disorder that causes severe pain and burning sensations when the skin is exposed to sunlight or other sources of visible light[1].
  • X-Linked Protoporphyria (XLP): A similar condition to EPP, but with a different genetic cause[1].

Both EPP and XLP are characterized by extreme photosensitivity, which can significantly impact a person’s quality of life by limiting their ability to spend time outdoors or in brightly lit environments[1].

How Does Dersimelagon Work?

While the exact mechanism of action is not fully detailed in the provided information, Dersimelagon is being studied for its potential to increase the amount of time patients can spend in sunlight before experiencing symptoms[1]. It may work by addressing the underlying causes of photosensitivity in EPP and XLP, potentially allowing patients to tolerate more sun exposure without pain or other adverse reactions.

Clinical Trials and Research

Dersimelagon is currently being evaluated in Phase 3 clinical trials, which are advanced stages of research designed to confirm the drug’s effectiveness and safety[1][2]. Two significant studies are underway:

  1. INSPIRE Study (MT-7117-A-302): This is a randomized, double-blind, placebo-controlled study aimed at evaluating the efficacy, safety, and tolerability of Dersimelagon in adults and adolescents with EPP or XLP[1].
  2. Long-term Extension Study: An open-label study to assess the long-term safety and tolerability of oral Dersimelagon in subjects with EPP or XLP[2].

These studies are looking at various aspects of the drug’s performance, including:

  • Time to onset of first symptoms (burning, tingling, itching, or stinging) when exposed to sunlight[1].
  • Patient-reported improvements in their condition[1].
  • Number of sunlight-induced pain events[1].
  • Long-term safety and tolerability[2].

How is Dersimelagon Administered?

Dersimelagon is being developed as an oral tablet[1][2]. This form of administration could offer a convenient option for patients, as it can be taken at home without the need for injections or medical procedures.

Safety and Side Effects

As Dersimelagon is still in clinical trials, comprehensive information about its safety profile and potential side effects is not yet available. The ongoing studies are closely monitoring for any adverse events, including:

  • Changes in liver function[1][2].
  • Effects on skin health, including monitoring for any changes in moles or development of skin cancers[1][2].
  • Any other unexpected side effects or safety concerns[2].

It’s important to note that all medications can have side effects, and the full safety profile of Dersimelagon will be better understood as more data from the clinical trials becomes available.

Who is Eligible for Dersimelagon Treatment?

Currently, Dersimelagon is only available through clinical trials. Eligibility criteria for these trials include:

  • Confirmed diagnosis of EPP or XLP[1].
  • Age between 12 and 75 years[1].
  • No history of other photodermatoses (light-sensitive skin conditions)[1][2].
  • No significant liver disease or abnormal liver function tests[1][2].
  • No history of melanoma or other skin cancers[1][2].

There are additional specific criteria for each trial, and potential participants would need to discuss their eligibility with the research team.

Future Prospects and Ongoing Research

Dersimelagon has been granted orphan drug designation by the European Medicines Agency, which recognizes its potential to address an unmet medical need in a rare disease[1][2]. If the current clinical trials demonstrate favorable results, Dersimelagon could become an important treatment option for people living with EPP and XLP.

Ongoing research aims to further understand the long-term effects and optimal use of Dersimelagon. Patients and healthcare providers should stay informed about the progress of these studies and any future developments in the treatment of EPP and XLP.

Aspect Details
Drug Name Dersimelagon Phosphate (MT-7117)
Conditions Studied Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)
Study Phase Phase 3
Main Objectives Evaluate efficacy, safety, and tolerability; Increase sunlight exposure time; Reduce sunlight-induced pain events
Study Duration 16 weeks (initial study); Up to 130 weeks (long-term extension)
Age Range 12 to 75 years
Administration Oral tablet
Key Measurements Time to onset of symptoms, Patient Global Impression of Change, Number of sunlight-induced pain events
Safety Monitoring Adverse events, Physical examinations, Vital signs, Laboratory tests, ECG, Nevi appearance

FAQ

  • What is Dersimelagon Phosphate? Dersimelagon Phosphate, also known as MT-7117, is an investigational drug being studied for the treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). It is taken orally in tablet form and is designed to help patients with these conditions better tolerate sunlight exposure.
  • What are the main goals of the clinical trials for Dersimelagon Phosphate? The main goals of the clinical trials are to evaluate the efficacy, safety, and tolerability of Dersimelagon Phosphate in patients with EPP or XLP. Specifically, they aim to investigate its effects on increasing sunlight exposure time before symptoms occur, reducing the number of sunlight-induced pain events, and assessing overall patient improvement.
  • Who can participate in these clinical trials? The trials are open to male and female patients aged 12 to 75 years with a confirmed diagnosis of EPP or XLP. Participants must be willing to go outside in sunlight during daylight hours and comply with the study requirements. However, there are specific inclusion and exclusion criteria that determine eligibility.
  • How long do the clinical trials for Dersimelagon Phosphate last? The initial Phase 3 study lasts for 16 weeks, with a primary assessment at Week 16. There is also a long-term extension study that can last up to 130 weeks (about 2.5 years) to evaluate the long-term safety and tolerability of the drug.
  • What are the potential benefits of participating in these trials? Participants may potentially experience increased tolerance to sunlight exposure, reduced frequency and severity of sunlight-induced pain events, and overall improvement in their condition. Additionally, they contribute to the advancement of medical knowledge and potential future treatments for EPP and XLP.

Ongoing Clinical Trials on Dersimelagon Phosphate

  • Study on the Safety and Tolerability of Oral Dersimelagon for Patients with Erythropoietic Protoporphyria or X-Linked Protoporphyria

    Not recruiting

    3 1 1
    Investigated drugs:
    Bulgaria Czechia France Germany Italy The Netherlands +4

  • Study on the Effects of Dersimelagon Phosphate for Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria

    Not recruiting

    3 1
    Investigated drugs:
    Bulgaria Czechia France Italy The Netherlands Poland +1

Glossary

  • Erythropoietic Protoporphyria (EPP): A rare genetic disorder that causes extreme sensitivity to sunlight, leading to painful skin reactions when exposed to sun or bright light.
  • X-Linked Protoporphyria (XLP): A rare genetic disorder similar to EPP, also causing extreme sensitivity to sunlight, but with a different genetic cause linked to the X chromosome.
  • Dersimelagon Phosphate: An investigational drug (also known as MT-7117) being studied for the treatment of EPP and XLP, taken orally to potentially increase sunlight tolerance.
  • Prodromal symptoms: Early warning signs or sensations (such as burning, tingling, itching, or stinging) that occur before the full onset of a sunlight-induced reaction in EPP or XLP patients.
  • Phototoxic reactions: Severe skin reactions caused by exposure to sunlight in patients with EPP or XLP, often resulting in pain, swelling, and burning sensations.
  • Patient Global Impression of Change (PGIC): A scale used to measure a patient's overall assessment of their improvement or worsening condition during a clinical trial.
  • Photodermatoses: Skin conditions triggered or worsened by exposure to light, including sunlight.
  • Cytochrome P450 (CYP) 3A4: An important enzyme in the body involved in the metabolism of many medications.
  • P-glycoprotein (P-gp): A protein that helps pump foreign substances out of cells, affecting how some medications are absorbed and distributed in the body.
  • Glomerular Filtration Rate (eGFR): A measure of how well the kidneys are filtering waste from the blood, used to assess kidney function.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effects-of-dersimelagon-phosphate-for-adults-and-adolescents-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/
  2. http://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-oral-dersimelagon-for-patients-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/