RARE Daily

NICE Recommends Amryt’s EB Therapy in Draft Guidance

August 21, 2023

Rare Daily Staff

The National Institute for Health and Care Excellence, the United Kingdom’s watchdog on drug prices, has recommended Amyrt pharmaceuticals’ Filsuvez for treating skin wounds associated with dystrophic and junctional epidermolysis bullosa in final draft guidance.

This is the first time NICE has recommended a treatment for epidermolysis bullosa (EB).

EB is a rare group of hereditary disorders of the skin, mucous membranes, and internal epithelial linings characterized by extreme skin fragility and blister development. Patients with severe forms of EB suffer from severe chronic blistering, ulceration, and scarring of the skin, mutilating scarring of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, a high risk of developing aggressive squamous cell carcinomas, infections, and risk of premature death.

Filsuvez is a birch bark extract that in a clinical trial led to quicker wound healing than a control gel in people with dystrophic and junctional EB. The results also suggest it may reduce the amount of skin affected.

The committee heard from the patient experts that, with no disease modifying treatments available, there are substantial treatment requirements to manage symptoms. These include daily cleaning of wounds and dressing changes that can often total 37 hours a week and which themselves can cause substantial pain,” Helen Knight, director of health technology assessment at NICE. “Birch bark extract, by reducing the time taken for wounds to heal and therefore the time needed to clean and redress them and the pain associated with that, has the potential to improve the quality of life for people with EB and their carers and help free up time for other activities.”

Photo: Helen Knight, director of health technology assessment at NICE

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