In addition, Raptor is developing RP103, known commercially as PROCYSBI, in multiple therapeutic areas such as nephropathic
cystinosis and Huntington's and mitochondrial diseases including Leigh syndrome.
cystinosis is a rare, inherited condition caused by a build up of cystine that affects every cell in the body.
The FDA approval of PROCYSBI was based on a New Drug Application (NDA) comprising data from six clinical trials, including a multi-center randomized, active-controlled Phase 3 trial of 43 patients with nephropathic
cystinosis and extension data from that trial.
cystinosis, an orphan disease, is estimated to effect a population of 2,000 patients worldwide, including 500 patients in the U.
There are three types of cystinosis, which can appear at any age: infantile nephropathic
(usually from 6-8 months after birth), late-onset nephropathic
(usually between ages 12 and 13), and benign non-nephropathic
(usually in adulthood).
com/research/sgngb4/raptors_rp_103) has announced the addition of the "Raptor's RP 103 for nephropathic
cystinosis - an orphan genetic disease" report to their offering.
The company offers PROCYSBI, a delayed-release capsule, which is used for the management of nephropathic
cystinosis in adults, as well as in six years and older children in the United States, in the 28 member states of the European Union, Norway, Liechtenstein, and Iceland.
Orphan Europe works with the following diseases: acute porphyria, congenital sucrase-isomaltase deficiency, homocystinuria, hyperammonaemia due to NAGS deficiency, nephropathic
cystinosis, patent ductus arteriosus, severe combined immunodeficiency - adenosine deaminase deficiency, vitamin E deficiency in chronic cholestasis and Wilson's disease.
Nasdaq:RPTP) has announced that it has enrolled the first patient in a pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic
The company is currently running clinical trials in patients with aldehyde dehydrogenase deficiency, nephropathic
cystinosis and non-alcoholic steatohepatitis.
Controls included healthy volunteers and nephropathic
patients with various degrees of renal insufficiency.
received $164,483 for a one-year study on the "Characterization of Novel Lysosomal Genes for Immune Regulation and Spermatogenesis in Nephropathic
Raptor Pharmaceuticals) announced that it has collaborated with DaVita Clinical Research (DCR) to screen blood samples from patients with end-stage renal disease (ESRD) in an effort to identify patients with unrecognized late-onset nephropathic
The data supports the effectiveness of pre-dose plasma cysteamine concentration in determining the correct cysteamine dose for nephropathic
cystinosis ("cystinosis") patients.
Raptor Pharmaceutical) announced that it has received a positive opinion from the European Committee for Medicinal Products for Human Use (CHMP), recommending marketing authorization for PROCYSBI 25mg and 75mg gastro-resistant hard capsules, cysteamine (as mercaptamine bitartrate) for the treatment of proven nephropathic