close

Products

Date: 2017-08-21

Type of information: Granting of a Market Authorisation 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 Europe (Switzerland)

Disease: myasthenia gravis, refractory generalized myasthenia gravis  in patients who are anti-acetylcholine receptor (AChR) antibody-positive

Latest news:

  • • On August 21, 2017, the European Commission approved the extension of the indication for Soliris® (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive. Soliris® is the first and only complement-based therapy approved in the European Union (EU) for this ultra-rare subset of patients.
  • Soliris® will be launched for this new indication initially in Germany, and Alexion is evaluating launches in additional EU countries.
  • The EC based its approval of the extended indication for Soliris on comprehensive clinical data from the Phase 3 REGAIN study (MG-301) and its long-term open-label extension study (MG-302).
  • A supplemental Biologics License Application (sBLA) in the U.S. and a supplemental new drug application in Japan for Soliris as a treatment for patients with anti-AChR antibody-positive refractory gMG have been accepted for review by the FDA and the Japanese Ministry of Health, Labour and Welfare (MHLW), respectively.
  • • On June 22, 2017, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for Soliris®. The new indication is as follows: "Refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive." The full indications for Soliris® will be as follows: • paroxysmal nocturnal haemoglobinuria (PNH). •atypical haemolytic uremic syndrome (aHUS) • refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive.
  • • 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.

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: 2017-08-21

Favourable opinion UE: 2017-06-22

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