Raptor Pharmaceutical (RPTP +1.1%) says its Procysbi delayed-release capsules for the treatment...

Raptor Pharmaceutical (RPTP +1.1%) says its Procysbi delayed-release capsules for the treatment of nephropathic cystinosis are now available in the U.S. for shipment. Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.

Comments (2)
  • Bentio Gonzalez
    , contributor
    Comments (26) | Send Message
    At 25 times the cost of the already approved drug on the market, a formulation that merely modifies, but does not significantly alter drug delivery, it is doubtful this company is going anywhere. Where are the insurance companies ready to pay $250,000/yr/pt? RPTP has a drug without a market willing to buy its product.
    20 Jun 2013, 02:19 PM Reply Like
  • Jason Boyce
    , contributor
    Comments (29) | Send Message
    RPTP is all about their second indication for Huntington's disease, where cystine also plays a role. Huntington's is a horrible genetic neurodegenerative disease resulting in death within 15-20 years of symptom onset. There are no therapies available to slow the disease. The company estimates the market potential for this indication at over $2 billion. This is not reflected in their valuation.....Even if they don't get over $200k for Procysbi it will fund their ph3 for Huntingtons...
    21 Jun 2013, 09:40 AM Reply Like
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