Date: 2013-06-28
Type of information: Granting of the orphan status in the EU
Product name: Soliris®
Compound: eculizumab
Therapeutic area: Rare diseases
Action mechanism: monoclonal antibody. Eculizumab is a recombinant humanized monoclonal IgG2/4 antibody that specifically binds to the complement protein C5, inhibiting its cleavage by the C5 convertase which prevents the generation of the terminal complement complex C5b-9.
Company: Alexion Pharmaceuticals (USA - CT)
Disease: neuromyelitis optica
Latest news:
- • On July 16, 2013, Alexion Pharmaceuticals, has announced that Soliris® (eculizumab) has received a positive opinion for orphan medicinal product designation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for the treatment of neuromyelitis optica (NMO). The positive opinion of the COMP has now been forwarded to the European Commission for final approval and publication in the community register.
• On June 27, 2013, Alexion Pharmaceuticals has announced that Soliris® (eculizumab) has been granted an orphan drug designation by the FDA for the treatment of neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder. In a Phase 2 study presented at the 2012 annual meeting of the American Neurological Association (ANA), Soliris® treatment was associated with a significant reduction in the frequency of relapses (recurring attacks) in patients with severe, relapsing NMO. The Phase 2 study reported at the ANA 2012 annual meeting and subsequently published in The Lancet Neurology13 was a single-arm, open-label, investigator-initiated trial in 14 women with severe, relapsing NMO who were treated for one year. The study met its primary efficacy endpoint, reduction in annualized relapse rate, with high degrees of clinical and statistical significance: a decline in the median annualized attack rate from 3.0 attacks per year pre-Soliris treatment to 0 attacks per year during 12 months of chronic Soliris treatment (p<0.0001). After 12 months of treatment, 86% (12 of 14) of these severely affected patients were completely attack-free.
- Soliris® was generally well-tolerated, with the three most common adverse events being headache, nausea, and dizziness. One case of meningococcal sepsis occurred. The patient made an uneventful recovery and restarted treatment with eculizumab to complete the study.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2013-06-24
Orphan status UE: 2013-08-05
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
