Orphan Europe (UK) Ltd. Company Profile

20:08 EST 18th December 2018 | BioPortfolio

Orphan Europe was started with the objective of providing orphan drugs to patients afflicted by rare disorders.Over the years an information network has been established to increase the awareness about rare disorders among healthcare professionals. The company has committed itself to provide support necessary to those working in this field. This is achieved through an extensive collaboration with experts in respective disease areas. Information and educational services can be provided upon request.
In another approach to strengthen the Orphan Drug status in Europe the company has provided input to the recently enacted European Orphan Drug Legislation.
We have highly experienced staff in our medical, regulatory and marketing departments with previous experience from major pharma companies. Being a small company allows us to be flexible and act rapidly. We are taking advantage of this rapid effective approach in our objective of providing healthcare professionals, and ultimately patients, with treatments for rare disorders.


Isis House 43 Station Road
United Kingdom


Phone: 44 (0) 1491 414 333
Fax: 44 (0) 1491 414 443

News Articles [465 Associated News Articles listed on BioPortfolio]

15% Reader Discount on Registration at the World Orphan Drug Congress Europe

The 9th annual World Orphan Drug Congress is the marketplace for orphan drug professionals looking at the complete value chain of orphan drug development, from clinical development and R&D to corp...

Orphan drug status for new compound 'great news' says Amryt Pharma PLC's COO

Amryt Pharma PLC (LON:AMYT) has been granted orphan drug status in the US for  a compound called AP102 to treat patients with the excess growth condition acromegaly. Chief operating officer Ror...

Cytheris: Encouraging responses and orphan drug designation in Europe for CYT107 for the treatment of PML

In this episode of PharmaTelevision News Review, filmed at #BIO2012 Convention in Boston, Fintan Walton talks to Damian Marron, CEO of Cytheris about encouraging responses and orphan drug designatio...

Are Orphan Drugs Getting Too Much Attention By Payers And Policymakers?

Success of the Orphan Drug Act has brought with it pushback from payers, policymakers, and legislators. Pushback may be warranted in some instances. But, a case-by-case approach examining the value of...

EMA panel backs orphan drug status for cystic fibrosis drug

The European Medicines Agency's Committee for Orphan Medicinal Products adopted a positive opinion on orphan drug status for  -More- 

Martin Pharmaceuticals’ Linvantra Receives FDA Orphan Drug Designation in Second Indication

NewsThe company has been granted Orphan Drug Designation by the FDA Livantra in the treatment of Pulmonary Arterial Hypertension.

Prescribe Robust Distribution to Commercialize Your Orphan Drug

Finding a home for your biopharmaceutical company’s new orphan drug doesn’t just happen after it is FDA approved. The successful commercialization of your promising new drug requires a focused and...

Defining orphan conditions in the context of the European orphan regulation: challenges and evolution

Drugs and Medications [8 Associated Drugs and Medications listed on BioPortfolio]

Nasal decongestant [SUPERVALU INC.]

Equaline 44-112

Carbaglu [Orphan Europe, SARL]

These highlights do not include all the information needed to use CARBAGLU safely and effectively.  See full prescribing information for CARBAGLU.   CARBAGLU (carglumic acid) TabletsInitial U.S. App...

Nizatidine [Dispensing Solutions Inc.]

Nizatidine Capsules USP Rx only


Akne-mycin (Erythromycin)

Kepivance [Swedish Orphan Biovitrum AB (publ)]

These highlights do not include all the information needed to use Kepivance safely and effectively. See full prescribing information for Kepivance.Kepivance® (palifermin) For injection, for intraveno...

PubMed Articles [392 Associated PubMed Articles listed on BioPortfolio]

Insights into the Pharmaceuticals and Mechanisms of Neurological Orphan Diseases: Current Status and Future Expectations.

Several rare or orphan diseases have been characterized that singly affect low numbers of people, but cumulatively reach ∼6% - 10% of the population in Europe and in the United States. Human genetic...

An autoregulatory loop controlling orphan nuclear receptor DAX-1 gene expression by orphan nuclear receptor ERRγ.

Dose-Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective Analysis.

In the development process for new drugs, dose-finding studies are of major importance. Absence of these studies may lead to failed phase 3 trials and delayed marketing authorization. In our study we ...

Towards the risk of Yellow Fever transmission in Europe.

Yellow fever (YF) is an emerging expanding fatal arboviral disease that has been imported at least six times in Europe during the first half of 2018, while only seven travel-associated YF cases have b...

Speed breeding orphan crops.

This review explores how speed breeding protocols that hasten plant growth and development could be applied to shorten breeding cycles and accelerate research activities in orphan crops. There is a gr...

Clinical Trials [665 Associated Clinical Trials listed on BioPortfolio]

Safety and Feasibility of Normothermic Machine Perfusion to Rescue Orphan Livers

Orphan livers are organs that have been declined for clinical use by all centers due to their marginality. The current standard of care of liver preservation before transplant is cold stor...

Orphan Europe Carbaglu® Surveillance Protocol

The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United ...

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying th...

In-market Utilisation of Liraglutide Used for Weight Management in Europe: a Retrospective Medical Record Review Study

This study is conducted in Europe. The aim of this study is to investigate usage of liraglutide for weight management in clinical practice.

Development of In Vitro Functional Assays From Primary Cells of Patients With Monogenic Diseases

The objective of this project is to build a collection of biological samples consisting of fibroblasts and primary keratinocytes from patients with orphan monogenic diseases and to use the...

Companies [972 Associated Companies listed on BioPortfolio]

AOP Orphan Pharmaceuticals AG

AOP Orphan is a multinational company with headquarters in Vienna, Austria, focusing on clinical research, development and distribution of medicines for rare and complex diseases....

Orphan Europe (UK) Ltd.

Orphan Europe was started with the objective of providing orphan drugs to patients afflicted by rare disorders.Over the years an information network has been established to increase the awareness abou...

Recordati Rare Diseases Canada Inc.

Recordati Rare Diseases Canada Inc. is a biopharmaceutical company committed to providing often overlooked orphan therapies to underserved rare disease communities in Canada. Reco...

Orphan Medical Incorporated

Orphan Medical Incorporated is a development stage company, was formed to acquire, develop and market products of high medical value intended to address inadequately treated or uncommon diseases of we...

Santhera Pharmaceuticals Holding Ltd

Santhera Pharmaceuticals is a Swiss biopharmaceutical company focusing on the discovery, development and marketing of small molecule pharmaceutical products for the treatment of neuromuscular diseases...

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Health care (or healthcare) is the diagnosis, treatment, and prevention of disease, illness, injury, and other physical and mental impairments in humans. Health care is delivered by practitioners in medicine, chiropractic, dentistry, nursing, pharmacy, a...

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