- Orphan drug developer PTC Therapeutics (PTCT +16.1%) is exploring selling itself amid rumors of interest from Shire plc (SHPG +1.9%) and BioMarin Pharmaceuticals (BMRN +1.8%).
- PTC's lead product candidate is Translarna (ataluren) for the treatment of patients with genetic disorders due to a nonsense mutation. It is in clinical development for Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD) and cystic fibrosis caused by a nonsense mutation (mnCF). The FDA designated it an Orphan Drug for both indications.
- The company's market cap is $2.2B while DMD competitor Sarepta Therapeutics is valued at $637M. BioMarin gobbled up the other DMD player, Prosensa Holding N.V. in November for $680M plus milestones.
- Previously: BioMarin buys Prosensa (Nov. 24, 2014)