Date: 2017-03-08

Type of information: Acceptation for review of a sNDA

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 Europe (Switzerland)

Disease: myasthenia gravis

Latest news:

  • On March 8, 2017, Alexion Pharmaceuticals announced that the FDA has accepted for review the Company's supplemental Biologics License Application (sBLA) to extend the indication for Soliris® (eculizumab) as a potential treatment for patients with refractory generalized myasthenia gravis who are anti-acetylcholine receptor (AChR) antibody-positive. The sBLA submission is supported by comprehensive data from the Phase 3 REGAIN study. The FDA set a Prescription Drug User Fee Act (PDUFA) date of October 23, 2017 .
  • If approved, Soliris® would be the first and only complement inhibitor for patients with refractory AChR-positive generalized myasthenia gravis. Soliris has received orphan drug designation  for the treatment of patients with myasthenia gravis in the U.S. and EU.
  •   • On June 13, 2014, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending eculizumab for designation as orphan medicinal product for the treatment of myasthenia gravis.


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: 2014-06-13

Orphan status UE: 2014-07-29

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes