MedPath

Afamelanotide

Generic Name
Afamelanotide
Brand Names
Scenesse
Drug Type
Small Molecule
Chemical Formula
C78H111N21O19
CAS Number
75921-69-6
Unique Ingredient Identifier
QW68W3J66U
Background

Afamelanotide is a first-in-class, synthetic, 13-amino acid peptide analogue of the endogenous alpha melanocyte-stimulating hormone (α-MSH). It differs structurally from its endogenous counterpart by only two amino acids - these structural differences improve biological efficacy by imparting a greater affinity for its target and a longer biological half-life. Afamelanotide is currently the only approved drug therapy used in the management of erythropoietic protoporphyria, having received approval in the EU in December 2014 and subsequent FDA approval in October 2019. Despite its relatively recent approval, afamelanotide has been available for use as an orphan drug in both the US and EU since 2008.

Indication

Afamelanotide is indicated for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP).

Associated Conditions
Phototoxicity reaction

Clinuvel Advances Phase III Trial for Vitiligo Treatment with SCENESSE®, Recruiting Over 200 Patients

• Clinuvel Pharmaceuticals has successfully completed recruitment of more than 200 patients across North America, Africa, and Europe for its Phase III trial of SCENESSE® in vitiligo treatment. • The 20-week randomized trial (CUV105) evaluates SCENESSE® as a systemic repigmentation therapy in combination with narrowband ultraviolet B phototherapy, with first results expected in late 2026. • Early clinical observations show promising repigmentation results, particularly in patients with darker skin types, addressing a significant unmet need in the estimated 820,000 vitiligo patients across North America.

Clinuvel Seeks EMA Approval for Increased SCENESSE Dosage in Europe

• Clinuvel is in discussions with the EMA to increase the maximum annual dosage of SCENESSE for erythropoietic protoporphyria (EPP) patients from four to six doses. • The proposed change aims to align European guidelines with those in the US and other regions, allowing for year-round treatment and improved patient outcomes. • Clinical and real-world evidence supports the submission, indicating no change in SCENESSE's safety profile with the increased dosage. • The EMA's decision, expected in Q1 2025, could significantly enhance treatment options for EPP patients across Europe.

Disc Medicine's Bitopertin Poised for Potential Accelerated FDA Approval in Erythropoietic Protoporphyria

• Disc Medicine's bitopertin may receive accelerated FDA approval for erythropoietic protoporphyria (EPP) treatment based on existing clinical data. • The FDA has agreed to the design of Disc's Phase III APOLLO trial, which will serve as a confirmatory study for bitopertin. • Bitopertin aims to reduce protoporphyrin IX (PPIX) levels, potentially alleviating pain and burns from sun exposure in EPP and XLP patients. • The Phase III APOLLO trial is set to begin in mid-2025 and will assess bitopertin's safety and efficacy in patients aged 12 and older.

Disc Medicine Announces End-of-Phase 2 Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria

• Disc Medicine will host a conference call on November 4 to discuss feedback from the FDA regarding the end-of-Phase 2 meeting for bitopertin. • Bitopertin is an investigational, orally administered inhibitor of GlyT1, designed to modulate heme biosynthesis for hematologic diseases. • The company is developing bitopertin as a potential disease-modifying therapy for erythropoietic porphyrias, including EPP and XLP. • Current treatments for EPP and XLP are limited to managing symptoms, highlighting the need for new therapies like bitopertin.

Patient-Researcher's Journey Leads to Breakthrough Treatment for Rare Light Sensitivity Disorder

• Dr. Jasmin Barman-Aksözen's personal experience with erythropoietic protoporphyria (EPP) led her to contribute to the development and approval of Scenesse, the first treatment for this rare genetic disorder. • Clinical trials demonstrated Scenesse allows EPP patients to spend up to 20 additional minutes in sunlight daily without pain, with some achieving three hours of sun exposure while using the treatment. • The drug's approval marked a historic milestone as the first treatment approved through EMA's patient experience pilot program, though limitations remain and new therapeutic candidates are under development.

7th Annual OCT DACH Conference to Address Clinical Trial Innovation, Investment, and Diversity Challenges

• The 7th Annual Outsourcing in Clinical Trials DACH 2024 conference will convene in Zurich this October, featuring key discussions on European ICH GCP V3 regulations and emerging technologies in clinical trials. • Industry leaders from Bristol Myers Squibb, Novartis, and other major companies will address critical challenges in clinical trial conduct, including AI integration and regional trial revitalization strategies. • The conference will highlight WHO's new guidance on clinical trial diversity and inclusion, with special focus on improving representation of underserved populations in medical research.
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