DR Cysteamine is Raptor's proprietary, enteric-coated micro-bead formulation of immediate release cysteamine bitartrate capsules, the current standard of care for treating cystinosis.
Bruce Barshop, M.D., Ph.D., Professor of Clinical Pediatrics at UCSD and principal investigator for the cystinosis clinical trial of DR Cysteamine, stated, "The excellent tolerability of DR Cysteamine, as demonstrated in this study, represents a major step forward in potentially improving the treatment of cystinosis. Raptor's formulation has the potential to improve dosing compliance and long-term outcomes for cystinosis patients."
Raptor's phase 2b clinical trial followed earlier clinical trials with an enteric-coated cysteamine prototype conducted by Ranjan Dohil, M.D., Associate Professor of Pediatrics at UCSD and funded by the Cystinosis Research Foundation ("CRF"). The CRF also supported Raptor's phase 2b clinical trial.
Patrice P. Rioux, M.D., Ph.D., chief medical officer of Raptor, said, "The results from our phase 2b trial are consistent with previous clinical findings from Dr. Dohil and support our plans to provide a twice-daily cysteamine therapy for cystinosis patients. We look forward to continuing our development program of DR Cysteamine in cystinosis with a phase 3 clinical trial."
Cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to treat cystinosis can cause serious health consequences, including renal failure and resultant kidney transplant, growth failure, rickets, photophobia and blindness. Symptom onset typically occurs within the first year of life, when cystine crystals accumulate in various tissues and
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