Afamelanotide

Generic Name: Afamelanotide

Brand Names: Scenesse

Drug Type: Small Molecule

Chemical Formula: C_₇₈H_₁₁₁N_₂₁O_₁₉

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

Associated Therapies: -

Clinical Trials

Injecting hope: Clinuvel eyes higher SCENESSE usage in Europe

Clinuvel Pharmaceuticals discusses with EMA to raise SCENESSE dosage for EPP patients from four to six doses, aligning with US guidelines. This aims to provide year-round treatment, supported by global safety data from 16,000 doses administered.

clinicaltrialsarena.com

2 months ago

Putting the DACH region on the map for clinical trials

The OCT DACH 2024 conference in Zurich discussed EU regulations, digitalisation, AI, and ethics in clinical trials, emphasizing the DACH region's challenges and opportunities.

clinicaltrialsarena.com

2 months ago

Disc teases potential NDA for rare skin disorder drug

Disc Medicine has secured FDA agreement for its investigational drug bitopertin to treat erythropoietic protoporphyria (EPP), with an accelerated approval application based on existing clinical data. The Phase III APOLLO trial, planned for mid-2025, will serve as a confirmatory study, focusing on pain-free sunlight exposure for EPP and X-linked protoporphyria (XLP) patients. The trial will measure average monthly time in sunlight without pain and other secondary outcomes.

Related Clinical Trials:

Phase III Confirmatory Study in Erythropoietic Protoporphyria

globenewswire.com

2 months ago

Disc Medicine to Host Conference Call on End of Phase 2 FDA

Disc Medicine to host conference call on Nov 4 to discuss FDA feedback on bitopertin, an investigational GlyT1 inhibitor for hematologic diseases, including erythropoietic porphyrias. Bitopertin, licensed from Roche, aims to be the first disease-modifying therapy for EPP and XLP, rare, life-threatening conditions affecting heme biosynthesis.

pharmaceutical-technology.com

2 months ago

How a patient helped shape a treatment for rare skin disorder

Dr Jasmin Barman-Aksӧzen, diagnosed with erythropoietic protoporphyria (EPP), joined a University of Zurich research group working on Scenesse, a treatment increasing skin pigmentation to protect against light-induced skin damage. Scenesse, approved in Europe in 2014 and the US in 2019, allows EPP patients to spend more time in sunlight without pain. Barman-Aksӧzen co-founded the International Porphyria Patient Network (IPPN) and advocates for head-to-head studies comparing new treatments to existing therapies.

Afamelanotide

Injecting hope: Clinuvel eyes higher SCENESSE usage in Europe

Putting the DACH region on the map for clinical trials

Disc teases potential NDA for rare skin disorder drug

Related Clinical Trials:

Phase III Confirmatory Study in Erythropoietic Protoporphyria

Disc Medicine to Host Conference Call on End of Phase 2 FDA

How a patient helped shape a treatment for rare skin disorder

Related Clinical Trials:

Phase III Confirmatory Study in Erythropoietic Protoporphyria

Drug Development Updates