Rare disease Archives

News and Trends 1 Jul 2015

Two of Alexion’s rare-disease drugs get the thumbs up from the EU

The European Medicines Agency (EMA) is about to approve a couple of rare diseases drugs developed by Alexion. The two enzymes, Strensiq and Kanuma, received positive feedback from the EMA’s Committee for Medicinal Products for Human Use, which normally leads to the approval of the drug. The Committee for Medicinal Products for Human Use has […]

July 1, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 12 May 2015

Alexion will invest €450 million to expand to Dublin

Alexion Pharmaceuticals will travel across the ocean to build its first ever biologics manufacturing facility outside the United States. This €450 million, four-year project, will be constructed at the Alexion’s College Park site in Blanchardstown (Ireland) and should create approximately 200 additional full-time jobs. This will bring Alexion’s total workforce in Ireland to almost 500 employees. […]

May 12, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 16 Apr 2015

Wilson Therapeutics on its way to cure Wilson disease

Wilson Therapeutics has closed a €37.4M Series B financing to advance its late-stage development program for Wilson’s disease. The company will present its ongoing Phase II trial of WTX101-201, the company’s treatment for this disease, at the upcoming 50th International Liver Congress 2015. Approximately 1 in 15000 people worldwide have Wilson Disease, a rare genetic disorder that prevents the […]

April 16, 2015 - 2 minutesmins - By Lourdes Riquelme

AOP Orphan Pharmaceuticals Confluence Pharmaceuticals Fragile X

News and Trends 23 Mar 2015

AOP Orphan and Confluence Pharmaceuticals, together for Fragile X Syndrome

AOP Orphan Pharmaceuticals and Confluence Pharmaceuticals, signed today a definitive agreement to co-develop and market Confluence’s lead asset, a product for the treatment of Fragile X Syndrome in Europe and the Middle East. Confluence is an American biopharmaceutical company focused on developing therapeutic treatments for Fragile X Syndrome and Autism Spectrum Disorders. The company has […]

March 23, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 5 Mar 2015

Baxter’s Bioscience Business Acquires SuppreMol

Baxter has acquired SuppreMol for €200M before working capital and other adjustments. The acquisition includes SuppreMol’s early-stage development portfolio of novel biologic immunoregulatory therapeutics for the treatment of autoimmune diseases. SuppreMol is a privately held biopharmaceutical company based in Martinsried, Germany developing treatment options for autoimmune and allergic diseases, focusing on the modulation of Fc […]

March 5, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 18 Feb 2015

Highway to Brain

Researchers in Barcelona have developed a shuttle to transport drugs into the brain, running through the blood-brain barrier. If there is an irreplaceable organ, for the moment, that’s the brain. The gray matter is not only protected by the skull, but also by a barrier of cells that tightly regulates the transport of substances into this […]

February 18, 2015 - 2 minutesmins - By Lourdes Riquelme

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