The Phase 2 clinical trial is a single arm, open label study designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) ELX-02 in 6 patients with nephropathic cystinosis
with at least 1 nonsense mutation in the cystinosis gene.
Study 003 is a single arm, open label study designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of subcutaneous ELX-02 in patients with nephropathic cystinosis
with at least 1 nonsense mutation in the cystinosin gene.
The capsules are FDA-approved for children one year of age and older and adults living with nephropathic cystinosis
Procysbi, for the treatment of infantile nephropathic cystinosis
, a genetic disease affecting the kidneys;
Long-term treatment with growth hormone in short children with nephropathic cystinosis
. J Pediatr 2001;138:880-7.
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
. J Mol Med (Berl) 1998;76:295-302.
The program will provide access to Raptor Pharmaceuticals Corp's PROCYSBI[R] (cysteamine bitartrate) for individual patients with nephropathic cystinosis
. Through the program PROCYSBI[R] will be available worldwide, except for the USA where the drug is already commercially available, LATAM and a small number of European territories.
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.
Food and Drug Administration ("FDA") seeking approval to market its investigational drug candidate, Cysteamine Bitartrate Delayed- release Capsules (RP103), for the potential treatment of nephropathic cystinosis
. 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.