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Late-Stage Orphan Disease Therapies

February 16, 2013

As discussed in a previous Orphan Druganaut Blog, one of the last catalysts before a final FDA decision, comes from a drug’s late-stage Phase III clinical trial data. Positive trial results can result in shares popping. There is also the reverse situation of failed clinical trial results causing shares to sink. Thus, late-stage Phase III clinical trials is one of the most critical steps of the orphan drug development life cycle. This is the critical time where demands for data on the drug’s safety and efficacy, comparisons to competitors, and other factors are important.

Medical Marketing and Media publishes an article by Marc Iskowitz, titled, “The Pipeline Report 2013: Late-Stage Standouts”. The Report highlights 196 late-stage (mostly Phase III) drugs generating buzz in the last few months. Twelve drugs are identified for orphan diseases. Here are several :

  1. BioMarin’s BMN-110/GALNS for Mucopolysaccharidosis
  2. Novartis’s Pasireotide for Acromegaly
  3. Pfizer’s Tafamidis Meglumine for Amyloid Polyneuropathy.

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