Small patient populations have suddenly become big business for biotechnology and pharmaceutical companies alike. The pipeline is rapidly filling with candidate medicines targeting rare and ultra-rare diseases. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), last year biopharmaceutical research companies had approximately 460 medicines targeting rare diseases either in clinical trials or awaiting FDA review. Read More
Optimer Pharmaceuticals Inc.'s Dificid (fidaxomicin), the first new product approved for Clostridium difficile-associated diarrhea (CDAD) in more than 25 years, has a superior rate of sustained clinical response through 25 days after the end of treatment compared to oral vancomycin. (See BioWorld Today, May 31, 2011.) Read More
Put a bunch of seasoned biotech investor veterans on stage and you are sure to attract an audience. That was certainly the case for "Forecasting 2013: Trick or Treat?" the closing plenary session of the BIO Investor Forum in San Francisco last week, which had a standing room-only crowd.
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