Eculizumab
ArticlesRefrigerated StorageLactation Safety InformationNew Medicines · Published , updated
426801002
Articles
23 September 2020
Safety in Lactation: Drugs used in hypoplastic, haemolytic, and renal anaemias
Biosynthetic erythropoietins are almost clinically indistinguishable from the natural form of erythropoietin, which is a normal component of breast-milk. They will not be absorbed from… Darbepoetin alfa Deferiprone Eculizumab HydroxycarbamideRefrigerated Storage
SolirisAlexion Pharma UK
Alexion Pharma UK
Soliris
300 mg concentrate for solution for infusion (10 mg/ml)
In the event of an inadvertent temperature excursion the following data may be used:
Soliris vials in the original package may be removed from refrigerated storage for only one single period of up to 3 days. At the end of this period the product can be put back in the refrigerator.
Please also refer to the manufacturer’s product literature for recommended storage information at https://www.medicines.org.uk
Contact Alexion for additional information on cases where a deviation from the recommended storage conditions has occurred. Refer to the current BNF for company contact details.
No
Yes if removed from the refrigerator for only one single period of up to 3 days.
17 April 2019
London MI Service
Lactation Safety Information
Limited published evidence of safety
No eculizumab detected in breast milk and no long-term effects noted in breastfed infants
Although large protein molecules may appear in colostrum, risk to preterm infants and neonates is considered to be small and unproven
17 September 2018
New Medicines
SolirisRelapsing neuromyelitis optica (NMO) - second-line or add-on therapy
Information
Soliris
Licence extension / variation
Alexion
Alexion
Development and Regulatory status
Launched
Launched
Launched
Yes
Yes
Aug 19Approved in EU [11].
Jul 19Recommended for EU approval by CHMP - the additional indication is "treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease [10].
Jun 19Approved in US for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive [8,9].
May 19Filed in the US and EU. Granted priority review status in the US with PDUFA date set for Jun 19 [7].
Apr 14Eculizumab was awarded orphan drug status in the EU (EU/3/13/1185) in Aug 13 and in the US in Jun 13 [3]
Category
First in class, humanised monoclonal antibody C5 complement inhibitor
Neuromyelitis Optica is a rare disorder affecting eyes and spinal cord leading loss of vision, mobility, and sensation. 90% of patients have relapsing NMO. It affects about 0.4 in 10,000 people in the EU equivalent to a total of around 20,000 people.
Relapsing neuromyelitis optica (NMO) - second-line or add-on therapy
Intravenous infusion
Further information
Yes
Suspended
Trial or other data
Sep 18PIII PREVENT study meets primary endpoint demonstrating that treatment with eculizumab reduces risk of NMOSD relapse by 94.2% vs placebo (p=<0.0001). At 48 weeks, 97.9% percent of patients receiving eculizumab were free of relapse vs 63.2% of patients receiving placebo [6].
Dec 17NCT01892345 (PREVENT) has completed enrolment [5].
Apr 16NCT01892345 (PREVENT) continues to recruit and is now due for completion Dec 16 [4]
Apr 14NCT01892345 (PREVENT) is a PIII randomized, double-blind, placebo-controlled, multi-centre trial of eculizumab in 132 patients with relapsing neuromyelitis optica (NMO). Recruitment is open to adults with a diagnosis of NMO or NMO spectrum disorder with relapsing disease. The primary outcome is time to first relapse. This trial is based on results from an investigator-initiated study which was recently published in Lancet Neurology. The study starts Jan 14 and is due to complete Jan 16. NCT02003144 is an open-label safety extension of this study which will continue until Dec 18 [1,2].
Evidence based evaluations
SolirisNeuromyelitis optica spectrum disorder (NMOSD) in children and adolescents (aged 2 to <18 years)
Information
Soliris
Licence extension / variation
Alexion
Alexion
Development and Regulatory status
None
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes
Category
First in class, humanised monoclonal antibody C5 complement inhibitor
Neuromyelitis Optica is a rare disorder affecting eyes and spinal cord leading loss of vision, mobility, and sensation. 90% of patients have relapsing NMO. It affects about 0.4 in 10,000 people in the EU equivalent to a total of around 20,000 people.
Neuromyelitis optica spectrum disorder (NMOSD) in children and adolescents (aged 2 to <18 years)
Intravenous infusion
SolirisGeneralised myasthenia gravis in children and adolescents
Information
Soliris
Licence extension / variation
Alexion
Alexion
Development and Regulatory status
None
None
Phase III Clinical Trials
Yes
Jun 20Alexion Pharma UK has confirmed that it does not intend to make a submission to NICE for the appraisal of eculizumab for treating refractory myasthenia gravis because the technology will not be launched in the UK for this indication [5].
Category
First in class, humanised monoclonal antibody C5 complement inhibitor
The incidence ranges from 0.3 to 2.8 per 100,000. It is estimated to affect more than 700,000 people worldwide. The prevalence of MG in the UK is estimated at about 15 per 100,000 population. Peak incidence is in the third decade for women and the sixth or seventh decade in men [1]. Precise data on incidence and prevalence in children are not known; paediatric presentation of MG is more common in Oriental than in Caucasian populations [2].
Generalised myasthenia gravis in children and adolescents
Intravenous infusion
eculizumab biosimilar (ABP 959) Paroxysmal nocturnal haemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)
Information
eculizumab biosimilar (ABP 959)
Biosimilar
Amgen
Amgen
Development and Regulatory status
Phase III Clinical Trials
Phase III Clinical Trials
None
Category
Monoclonal antibody that binds to C5 complement and blocks progression of the complement cascade preventing generation of the potent anaphylatoxin C5a and the cytolytic C5b-9 complex.
Prevalence of PNH is estimated at approximately 1 per 500,000. Prevalence of aHUS is 1-9 per 1,000,000 [1].
Paroxysmal nocturnal haemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)
Intravenous infusion
Paroxysmal nocturnal haemoglobinuria (PNH)
Information
Biosimilar
Samsung Bioepsis
Samsung Bioepsis
Development and Regulatory status
Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Category
Monoclonal antibody that binds to C5 complement and blocks progression of the complement cascade preventing generation of the potent anaphylatoxin C5a and the cytolytic C5b-9 complex.
Prevalence of PNH is estimated at approximately 1 per 500,000.
Paroxysmal nocturnal haemoglobinuria (PNH)
Intravenous infusion