Shire's new drug has been approved as Europe's first vascular haemophilia recombinant VWF drug

Recently, the pharmaceutical company Shire announced that the European Commission (EC) has approved Veyvondi (vonicog alfa) for use in adult patients aged 18 and over with vasovascular haemophilia (VWD) for the treatment of haemorrhage in the single use of deamination (DDAVP) when it is ineffective or not applicable, as well as for the treatment/prevention of surgical bleeding. The drug, a recombinant vascular haemophilia factor (rVWF), was approved to make it the first and only RVWF drug in Europe to treat VWD, which specifically addresss the primary defects or dysfunctions of VWF while allowing the body to restore or maintain sufficient factor VIII (FVIII) plasma levels.
in the U.S., vonicog alfa, a brand named Vonvendi, was approved in December 2015 for on-demand treatment and haemorrhage control in adult patients with VWD, and in April this year, the drug was approved by the FDA for peri-surgery management in adult patients with VWD. To date, Vonvendi remains the first and only RVWF drug approved by the FDA in the U.S. market to treat adult patients with VWD.
Andreas Busch, head of research and development and chief scientific officer at Shire, said the EU approval of Vonvendi marked a key regulatory milestone in our commitment to addressing the significant unseconded medical needs of VWD patients. Next, we will ensure that Vonvendi is widely available throughout Europe.
approval is based on data from three clinical studies. In the study, a total of 80 VWD patients received Veyvondi treatment, including: 1) a multi-center, controlled, randomized, single-blind, dose increment phase I study that assessed the safety, toerability and pharmacodynamics of rVWF in patients aged 18-60 years; The PK, safety and effectiveness of rFIII and rVWF for the treatment of bleeding events in adult patients with severe VMD were evaluated, and 3) a forward-looking, open-label, non-controlled, non-randomized Phase III study assessed the effectiveness and safety of rVWF (joint or unused rFIII) for severe VWD patients undergoing large, small, oral option surgery.
VWD is the most common hereditary hemorrhagic disease, affecting about 1% of the world's population and about 100,000 people in the European Union. The disease is caused by a lack or dysfunction of vascular haemophilia factors ( one of several proteins in the blood that promote blood clotting ) that can cause the patient's blood to fail to coagulate effectively, leading to severe bleeding in the nose, gums, intestines, and muscles and joints. Female patients may have severe abnormally persistent menstruation and may experience excessive bleeding after childbirth.
, developed by Baxalta, became a global leader in the development of rare disease drugs in January 2016 when Shire acquired it for $32 billion. Currently, Shire is also developing Vonvendi as a preventive drug and paediatric adaptation. (Bio Valley)