For the first time, the US Food and Drug Administration (FDA) has expanded a drug's indication without additional clinical data. FDA announced it was expanding the use of Vertex Pharmaceuticals' cystic fibrosis drug Kalydeco (ivacaftor) based solely on new in vitro data to include an additional 23 genetic mutations, more than tripling the number of mutations the drug is approved to treat. With the expanded indication, Kalydeco is approved to treat mutations affecting ...

The US Food and Drug Administration (FDA) on Thursday unveiled 38 new specific recommendations on the types of bioequivalence trials generic drug companies should run in order to win approval for their abbreviated new drug applications (ANDAs). The list targets what trials should be run by those developing a number of new generic oncology treatments, though many have yet to lose patent protection and some were recently approved, including Pfizer’s breast cancer drug Ibra...

Hell hath no fury like a Wall Street analyst scorned. Or at least that's what Cambridge-based Vertex Pharmaceuticals might be thinking to itself today after admitting it had inadvertently overstated the effectiveness of its new combo therapy for cystic fibrosis (CF), causing shares of Vertex ( $VRTX ) to plummet. Vertex has been testing the new therapy, which consists of its already-approved drug Kalydeco (ivacaftor) and a new experimental compound known as VX 809. Unli...

The European Medicines Agency (EMA) has recommended Vertex Pharmaceuticals' cystic fibrosis (CF) drug Kalydeco (ivacaftor), moving the drug one step closer to becoming the drug the first in Europe for the treatment of CF in patients with a specific gene mutation. The drug received the Committee for Medicinal Products for Human Use's (CHMP) recommendation on 25 May after being reviewed under a 150-day accelerated approval program. EMA notes Kalydeco is the eighth pro...