Type of information: Granting of a Market Authorisation in Japan
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 December 26, 2017, Alexion Pharmaceuticals announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has approved Soliris® (eculizumab) as a treatment for patients with generalized myasthenia gravis who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy or plasmapheresis. Soliris is the first and only complement inhibitor approved in Japan as a treatment for these patients.
- In the Phase 3 REGAIN study and its ongoing open-label extension study, Soliris® demonstrated treatment benefits for patients with anti-AChR antibody-positive generalized myasthenia gravis who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms, which can include difficulties seeing, walking, talking, swallowing and breathing. These patients are at an increased risk of disease exacerbations and crises that may require hospitalization and intensive care and may be life-threatening.
- Japan's MHLW based its approval of this new indication of Soliris® on comprehensive clinical data from the Phase 3, randomized, double-blind, placebo-controlled, multicenter REGAIN study (ECU-MG-301).
- • 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: