for the management of nephropathic cystinosis
in adults and children ages two years and older.
This clarifies our near-term priorities, which are to maximize the reach of PROCYSBI in nephropathic cystinosis
, further the development of RP103 in Huntington's and mitochondrial diseases, prepare for QUINSAIR'S launch and initiate at least one trial in nontuberculous mycobacteria or bronchiectasis.
The program will provide access to Raptor Pharmaceuticals Corp's PROCYSBI[R] (cysteamine bitartrate) for individual patients with nephropathic cystinosis
Food and Drug Administration (FDA) has approved PROCYSBI (TM)(cysteamine bitartrate) delayed release capsules for the treatment of nephropathic cystinosis
in adults and children 6 years and older.
The company said the FDA's decision on its New Drug Application for RP103 (PROCYSBI) for the potential treatment of nephropathic cystinosis
would be delayed by three months.
In its application, Raptor has requested Priority Review of the NDA, which, if granted, could lead to a decision for marketing approval from the FDA of RP103 for the potential treatment of nephropathic cystinosis
in the fourth calendar quarter of 2012.
The company is currently running clinical trials in patients with aldehyde dehydrogenase deficiency, nephropathic cystinosis
and non-alcoholic steatohepatitis.
Kaiser-Kupfer experienced success in reducing visual loss associated with the rare metabolic disorders gyrate atrophy and nephropathic cystinosis
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.
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 cystinosis
The data supports the effectiveness of pre-dose plasma cysteamine concentration in determining the correct cysteamine dose for nephropathic cystinosis