RARE DERMATOLOGICAL DISEASES

A Growing Need for Rare Skin Disease Treatment

Rare diseases affect approximately 30 million people in the European Union and 25-30 million in USA, representing a major health issue still to be addressed.

Among them, over 1000 rare skin diseases are known, many of which are of genetic origin and manifest in childhood. Children who experience these skin conditions have high rates of complications resulting in long and costly hospital stays and strong psychological impact. There exists a significant unmet need for more efficacious and safer treatment options that treat the underlying cause of disease as opposed to managing the symptoms.

Some orphan and rare dermatological diseases do not have any effective treatments on the market, including Epidermolysis Bullosa and Hailey Hailey disease.

The orphan and rare dermatology premium products market is projected to grow rapidly from $1.64bn in 2017 to $6.07bn in 2024, at a compound annual growth rate (CAGR) of 20.54%.

The premium products market size in the US is expected to increase from $794m in 2017 to $2.73bn in 2024, at a CAGR of 19.3%, accounting for just under half of the global orphan and rare dermatology market.

Epidermolysis Bullosa

Epidermolysis Bullosa (EB) is a group of rare, genetic, life-threatening connective tissue disorders characterized by skin blistering throughout the body as well as severe impact to internal organs.

"Butterfly Children" is a term often used to describe younger patients because the skin is said to be as fragile as butterfly's wings. It is estimated to impact 1:20,000 births with limited life expectancy and approximately half a million patients globally.

Epidermolysis Bullosa Treatment

For Epidermolysis Bullosa, no approved treatments or cure are available. The standard of care is currently palliative treatments for blisters and wounds all over the body for the entire life. Itching, pain, and scarring strongly reduce the quality of life while persistent skin inflammation is correlated to tumor incidence and death.

EB imposes a major burden for global health care, and the cost of the treatment of a severely affected patient in the United States can approach $300,000 per year, whereas wound care supplies for those with EB are estimated more than $10,000 per month.

We are currently developing a new treatment of blisters and wounds in EB patients. Find out more

Hailey Hailey Disease

Hailey Hailey Disease (HHD) is a bullous genetic rare disorder with high impact on the quality of life of the patients, their family and on society: social exclusion, disability, short life expectancy. It usually begins in the third or fourth decade of life.

Classic clinical features consist of vesicles, erosions, weeping plaques, fissures, and scale crust symmetrically distributed in intertriginous areas, with the most common areas being the axillae, groin, neck, inframammary folds and perineum.

It is estimated to impact 1:50,000 births. No approved treatments or cure are available. There are only palliative treatments for vesicles and erosions for the entire life.

Management of this condition is difficult and existing treatments do not provide a long lasting positive therapeutic benefit.

Recent studies suggest that skin lesions are associated with complex multipath molecular alterations and oxidative stress. On the other side, superinfection with bacteria, fungi, and viruses play an important role in exacerbations and persistence of lesions.

There is a big unmet medical need due to loss of efficacy treatment for chronic or relapses forms with only few innovative treatments under development.

Our treatment, currently under development, could support the healing process and thus possibly lead to a protective antioxidant response. Find out more