Can Orphan Drugs Soften the Blow of the Patent Cliff?

With an estimated $67 billion worth of biosimilar patents due to expire before 2020, as reported by GaBI in a guest blog for TotalBiopharma, increasing research and development costs, and slowing rates of approval for mass market drugs currently under development, how are some of the world’s leading pharmaceutical companies planning to avoid falling off the patent cliff? Well, a …

#AMT’s #HemophiliaB #GeneTherapy gains #OrphanDrug Designation

AMT's  gene therapy program for the treatment of hemophilia B has today gained orphan drug designation in the US from the FDA. AMT’s hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College …