the first therapeutic option approved for patients living with recessive dystrophic epidermolysis bullosa (RDEB) is now also available in Italy. It is a drug in the form of a topical gel for the treatment of partial thickness wounds, starting from 6 months of age, based on a mixture of triterpenes extracted from birch bark.
It is now also available in Italy the first treatment option approved for patients living with recessive dystrophic epidermolysis bullosa (RDEB). It is a drug in the form of topical gel for the treatment of partial thickness wounds, starting from 6 months of age, based on a mixture of triterpenes extracted from birch bark.
The drug, fully reimbursable, was recently approved by the FDA after the green light obtained from the EMA in 2022. The centralized marketing authorization is valid throughout the EU.
The drug became part of the portfolio of Chiesi Group with the recent acquisition of Amryt Pharma, a biopharmaceutical company dedicated to the development of innovative treatments for rare diseases. Once prescribed by the doctor in dedicated specialist centers, the drug can be administered at home and can be integrated with conventional treatments.
L’epidermolysis bullosa (EB) it is a rare, hereditary and debilitating pathology, which affects both sexes, characterized by extreme epidermal fragility with blisters and ulcerations generated even with slight friction and minor trauma, painful and widespread on the body, which highlights a reduction in life expectancy. Receiving an intense hug can cause epidermal blisters which can ulcerate and become infected, or become chronic, with the risk of developing tumors.
“For Chiesi, but above all for the families of people living with this disease, the green light from the EMA and FDA to the topical gel is an important milestone – he comments Nicola Gianfelice, director of the Rare Disease business unit of Chiesi Italia -. The drug, which has just been approved by AIFA, can now be prescribed in our country and is reimbursed for forms of recessive dystrophic epidermolysis bullosa (RDEB); thus bringing immediate concrete help to all people, in most cases children, to alleviate the effects of a devastating pathology on both a physical and psychological level.”
