Innovating for patients across the globe

Our mission at Orpha Biotech is to develop innovative solutions for the treatment of diseases that occur at rare frequency and for which effective therapies currently do not exist.

Such diseases are often referred to as orphan diseases, since their low incidence has meant their market size has been too small for mainstream drug developers to place resources behind them, leaving the patient population in effect neglected and orphaned.

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A rare disease is defined in Europe as any life-threatening or chronically debilitating disease that affects fewer than 5 in 10,000 people. Thousands of rare diseases have been identified and the cumulative population prevalence of rare diseases is estimated to be 3.5-5.9% globally at any given time. In other words, while the incidence of each rare disease is by definition quite low, the overall number of people across the globe affected by rare diseases is substantial.

At Orpha Biotech, we focus on where we see a need. We strive for a world where no patient is left abandoned in the world of medical care. Through creativity and innovation, and in solidarity with the wider rare disease scientific community, we aim to alleviate orphan disease burden to serve patients across the globe.

“The vision of the Sustainable Development Goals is a world in which no one is left behind, including people who suffer from rare diseases. Just because a disease affects a small number of people does not make it irrelevant or less important than diseases that affect millions.”

– Dr Tedros Adhanom Ghebreyesus, WHO Director-General


We target central molecular determinants and biological mechanisms of disease pathogenesis. Together with our CRO partner Molecular Research Pharma CT, we employ structure-based computational ‘virtual screening’ and allied docking simulations, and we have generated a platform of small molecules that can be developed as potential agonist or antagonist NCEs for a broad spectrum of pathologies.

Our initial focus is on orphan autoimmune indications, particularly on developing small molecules for the treatment of systemic sclerosis. Our virtual screens have yielded novel antifibrotic agents that go beyond inhibiting disease progression to promoting tissue regeneration and repair in preclinical studies; their feasibility for other indications is being explored.

Our endeavor to accelerate the delivery of treatments to patients across the globe is supported by the well-established commercial market for orphan products that have a streamlined, expedited and cheaper regulatory approval process compared to regular medicines. For other indications, there is the added advantage that approval and market entry for a non-orphan indication are substantially facilitated following approval for an orphan indication on the same metabolic pathway.



Therapeutic Area




Phase I

Phase II

Phase III


Rare diseases

Systemic sclerosis


Provides an inventory of rare diseases and related information for over 6000 of these diseases, as well as a directory of expert resources in the countries of the consortium.
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Provides services for patients and their families, rare disease patient organizations, medical professionals, and those seeking to develop new diagnostics and treatments.
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Seeks to improve the quality of life of people living with rare diseases in Europe through advocacy at the European level, support for research and medicines development, facilitating networking amongst patient groups, raising awareness, and many other actions designed to reduce the impact of rare diseases on the lives of patients and family.
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The mission is to connect, empower and inspire the rare disease community.
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Unites governmental and non-profit funding bodies, companies, patient advocacy organizations, and scientific researchers to promote international collaboration and advance rare diseases research worldwide.
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