For patients with deadly and debilitating conditions, FDA drug approvals can be cause for celebration. Many of these patients and their families have been waiting years for better therapies – or any therapy at all. They consume every public announcement about drug approvals for their particular disease like it’s water in the desert. But there is also a sense of trepidation once the drug launches. How much will this new drug cost? What will insurance cover? What will be our share?

BioMarin Pharmaceutical announced that the U.S. FDA approved Brineura (cerliponase alfa) for the rare disease, late infantile neuronal ceroid lipofuscinosis type 2 (CLN2).

Shares of Raptor Pharmaceutical Corp. were up after Horizon Pharma announced it had struck a deal to acquire the rare disease company for $800 million.

Karmiel, Israel-based Protalix Biotherapeutics, Inc. (PLX) announced today that it was selling its share of a collaboration deal back to Pfizer Inc. (PFE)   Under the original deal, Pfizer took 60 percent and Protalix took 40 percent of revenues and expenses for the development and commercialization of ELELYSO, except in Israel and Brazil. Under the […]