Orphan Europe has been developing, co-developing and distributing orphan drugs since 1990, well before the orphan drug legislation was introduced in Europe. Thanks to a complete dedication to and understanding of this market, Orphan Europe is experiencing a 20-30% annual growth in sales.

Today Orphan Europe maintains a good balance between in-house developed drugs, co-development and distribution agreements. It is a highly qualified organisation with expertise in medical, regulatory, marketing and distribution issues for orphan drugs, providing 10+ medicinal products to patients all over the world.

 

Synergies

Orphan Europe cooperates with a number of well-established pharmaceutical and biotechnology companies as well as research institutes. These partnerships have involved co-development, clinical studies, regulatory applications, production, marketing and worldwide distribution.

For example, Orphan Europe and InfaCare are collaborating in the development of a new therapy for the treatment of severe hyperbilirubinaemia in neonates. The common goal is to bring Stanate (stannsoporfin) to market, both in Europe and in the United States, as a unique and innovative drug to efficiently treat this potentially life-threatening clinical condition, which affects premature babies and full-term newborns alike.

Thanks to an innovative mechanism of action that blocks transiently the transformation of haemoglobin – the pigment in red blood cells – into bilirubin, this drug discovered by American scientists from Rockefeller University will potentially eliminate the need for intensive phototherapy. The Rockefeller University and InfaCare have carried out numerous studies and clinical trials worldwide.

While InfaCare will continue the drug development of Stanate for the U.S. market, Orphan Europe will complete the clinical and pre-clinical development in Europe, aiming at European marketing authorization in the shortest possible timeframe, for the treatment of newborns that are at higher risk of developing severe hyperbilirubinaemia and kernicterus.

Another example of a fruitful cooperation is the licensing of a drug for the treatment of acute attacks of porphyria from the Finnish company Leiras (today belonging to Bayer GmbH). The drug was only approved in Finland but Orphan Europe submitted the regulatory dossier. In 1995 Orphan Europe obtained the Marketing Authorization in France and in successive waves of Mutual Recognition Procedure Orphan Europe extended the approval to the rest of Europe.

Looking for a partner?

Are you a company working with orphan drugs? Do you need a co-development partner? Are you looking for expertise in developing your European regulatory dossier? Do you need access to the European or Middle East market?

Please contact our business development department at: