Horizon Pharma plc announced the launch of Cystinosis United, an initiative created for people living with nephropathic cystinosis
, a rare genetic metabolic disease that causes the amino acid cystine to accumulate in all organs of the body.
The patient was diagnosed with nephropathic cystinosis
based on these findings and the presence of ocular crystalline deposits.
Clinical and molecular aspects of nephropathic cystinosis
The program will provide access to Raptor Pharmaceuticals Corp's PROCYSBI[R] (cysteamine bitartrate) for individual patients with nephropathic cystinosis
Steady-state pharmacokinetics and pharmacodynamics of cysteamine bitartrate in paediatric nephropathic cystinosis
Biopharmaceutical company Raptor Pharmaceutical Corp (Nasdaq:RPTP) announced on Thursday the approval by the European Commission (EC) of PROCYSBI gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate), as an orphan medicinal product for the treatment of proven nephropathic cystinosis
for marketing in the European Union (EU).
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
Academic achievement in individuals with infantile nephropathic cystinosis
Long-term treatment with growth hormone in short children with nephropathic cystinosis