The Orpha-Biotech project focuses on the research and development of molecules directed against rare diseases (Rare Diseases). In Europe, a disease is defined as rare if it has a prevalence equal to or less than 5 cases per 10,000 people. The number of rare diseases is high, the estimates currently number between 5,000 and 8,000, which affect between 6% and 8% of the European population (source: 2015 report, Rare Diseases in the Lazio region, Lazio Region a link to national or institutional level pls.). Rare diseases are often fatal or debilitating diseases and represent 10% of the diseases that afflict humanity.
A rare disease is defined as a disease that affects less than one person for every 2,000 inhabitants in Europe. In the field of rare diseases, there are those ultra rare or very rare that affect less than one person per million. Furthermore, the scope of orphan diseases is being defined. Orphan diseases are those diseases that do not represent a common object of research, those for which specific therapies are not available, and those which, due to lack of funds and basic research, are of limited interest to researchers and doctors. As a result, they have been referred to as “orphans” due to the state of abandonment left to patients in the world of medical care.
The common denominator is always that of the rarity of the event characterized by a low frequency in the population and the absence of an effective therapy to combat it.
Orpha-Biotech carries out research, development, production and marketing of innovative products in the field of bio-sciences and bio-technologies in particular through molecules that act for therapeutic purposes on rare / orphan diseases; our research concerns products or drugs aimed at treating autoimmune diseases such as Systemic Sclerosis.
HOW MUCH DO THEY AFFECT?
In Italy, based on the prevalence data available at European level, those affected by orphan diseases could range from 3.5 to 5 million.
Although each individual orphan disease affects only a small number of people, the total number of patients involved is very large, with around 350 million individuals worldwide.
Many of the rare diseases until recently were ignored by doctors and adequate medical and scientific knowledge did not exist. For most of them there is still no cure but appropriate treatments can improve the quality and life span.
About 25 thousand people suffer from systemic sclerosis in Italy. Nine out of ten patients are women and, often, the onset of the disorder occurs in the years before or after menopause. It is a systemic autoimmune disease: it causes fibrosis of the skin and organs (a hardening due to tissue damage and the accumulation of connective tissue) and circulatory disorders, linked to alterations of the immune system. The symptoms are progressive and have a strong impact on the quality of life of patients.
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WHAT DO YOU NEED TO KNOW ABOUT ORPHAN DISEASES?
- The term “orphan” includes pathologies of very different origins and types: rare and congenital, infectious, tumor, genetic, degenerative; however the mode of transmission by genetic route is predominant which is estimated to affect 80% of all orphan diseases.
- Almost all orphan diseases are also chronic and debilitating, and the affected patient must live with the symptoms throughout life, often from birth. In many cases these are pathologies that are dangerous for survival, which reduce average life expectancy and which always lead to a reduction in the quality of life.
- During the course of the disease, the patient can often find himself suffering from the isolation and misunderstanding of the community in which he lives due to the lack of information and knowledge about the disease and its manifestations.
- It is now believed that over 70% of those affected by rare diseases are children.
- Rare diseases can turn into common pathologies, as it happened to AIDS and the Ebola virus.
Orpha Biotech product line
- Orpha biotech proposes two molecules for the treatment of progressive cutaneous systemic sclerosis.
- Orpha biotech has developed a peptide for tissue regeneration that finds applications in the field of dermatology and anti-aging.
The target market
The company, through its technologies targets the following markets:
- The rare-diseases market: this market shows a TAM of 131b USD and projects a CAGR of 10.8% on average until 2023