Again we have the media troubled by new, improved drugs that cost more than the generic they are based on rather than trumpeting the improvements. Raptor Pharmaceutical received approval of its Procysbi (cysteamine bitartrate) for the treatment of nephropathic cystinosis, an orphan disease affecting an estimated 500 patients in the U.S. FDA approved Procysbi under 505(b)(2) on April 30, 2013 based on the listed drug, Cystagon. Cystagon has been used off-label as the only treatment for this fatal disease. It is an immediate release product taken every 6 hours, while Procysbi is a delayed-release capsule taken twice a day. Moreover, the IR tablet causes nausea, vomiting, bad breath and body odor. Procycbi is a capsule containing enteric-coated beads that delay the release past the stomach, reducing the adverse events.
Nephropathic cystinosis is the build-up of cystine in the body that often leads to kidney failure. As noted above, cysteamine is the standard of care but with a q.i.d. regimen it is difficult to maintain adequate trough levels. Procycbi’s b.i.d. regimen was studied in an active-controlled Phase 3 trial of 43 patients (adults and pediatrics over 6 years of age) with nephropathic cystinosis. According the FDA press release “Patients were randomly assigned to receive Cystagon or Procysbi for three weeks before being switched to the other product for an additional three weeks. Blood testing showed Procysbi was as effective as Cystagon in controlling cystine levels.”
So the question becomes, if the generic works as well as the Procycbi, but compliance and its sequelae means kidney transplant, or premature death, what is the cost difference between the two regimens? According to the NYTimes, the generic costs $8,000 per year while Procycbi will cost $250,000 (no citations).