Business/Regulatory

Starting, running, or even working in a pharmaceutical company focused on orphan drugs requires people to think differently.
Shire plc and NPS Pharma have entered into a merger agreement in which Shire will acquire all the outstanding shares of NPS Pharma for approximately $5.2 billion ($46.00 per share in cash). The transaction has been approved unanimously by the Boards of Directors of both Shire and NPS Pharma.
The NPS in NPS Pharma stands for Nexus of Patients and Science.
In this exclusive interview with RDR, Dr. Francois Nader discusses the history of NPS Pharmaceuticals.
“The Committee accepted that eculizumab is a step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need.”
BioMarin Pharmaceuticals announced it plans to buy Prosensa Holding NV for approximately $840 million ($17.75 for all shares).
As they say, “we are now open for business”
The open-label study will evaluate the efficacy and safety of eteplirsen in Duchenne muscular dystrophy patients over 48 weeks (30 mg/kg once a week)and in approximately 39 sites across the United States.
I never imagined I would be the CEO of a small rare disease company.
Evaluate Pharma has released their report on orphan drugs and what the landscape will look like in 6 years.
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