Type of information: Granting of a Market Authorisation in the EU
Product name: Soliris®
Therapeutic area: Rare diseases
- 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
- • 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.
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
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: