Information for Patients and Caregivers |
Here, patients and caregivers will find information including standards of care, clinical progress, as well as links to other resources for the following medical conditions:
Raptor's goal is to empower patients so they can make informed decisions about their treatment options and their future.
Phase 3 Clinical Trial of RP103 (DR Cysteamine) for Nephropathic Cystinosis
Nephropathic cystinosis is a rare lysomal storage disease that may affect as many as 2,000 people worldwide. The disease is usually diagnosed in early childhood.
RP103 is an enteric coated microbead formulation of cysteamine bitartrate, being developed to efficiently deliver cysteamine in a well-tolerated twice-daily formulation. (Dohil 2006). Enteric coating allows the medication to transit through the stomach to the small intestine before it is released.
Challenges of Cysteamine Bitartrate Therapy
Cysteamine bitartrate therapy may be effective at preventing or delaying kidney transplants in nephropathic cystinosis patients (Gahl 2007, Levtchenko 2006, Markello 1993, Kleta, Bernadini 2004). However, patient adherence is challenging due to frequent dosing and gastrointestinal side effects associated with cysteamine bitartrate therapy (Levtchenko 2006, Kleta, Bernardini 2004, Kleta, Gahl 2004, Cystagon Package Insert).
In its Phase 3 clinical trial, Raptor has demonstrated that RP103 therapy met the primary endpoint of non-inferiority compared to Cystagon®, the currently marketed immediate-release cysteamine bitartrate. Addtionally, RP103 met the endpoint at a lower average daily dose and with a lower dosing frequency than Cystagon®. (RP103 was administered once every 12 hours compared to once every six hourse for Cystagon®)
Raptor’s Phase 3 Clinical Trial Meets Primary Endpoint
Raptor’s Phase 3 clinical trial of RP103 (Delayed-Release or DR Cysteamine) for the treatment of nephropathic cystinosis met the primary endpoint of non-inferiority compared to the currently marketed, immediate-release cysteamine bitartrate. Patients received RP103 at a lower average daily dose and with a lower dosing frequency than the currently marketed compound.
Raptor plans to file for marketing approval of RP103 in the US and EU in 2012.
Raptor Collaborates with NIDDK
Raptor signs Collaborative Research and Development Agreement With NIDDK on Phase 2b Clinical Trial of RP104 (DR Cysteamine Tablets) in Non-Alcoholic Steatohepatitis (NASH)
Phase 2 NASH Clinical Trial Completed
Raptor presented at the Digestive Disease Week 2010 Conference. Primary endpoints met.