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Articles

Comparison of products used in Hereditary Angioedema in the absence of Berinert

15 June 2020This table provides a summary of clinical factors to consider when using alternative complement regulatory proteins to Berinert (C1-esterase inhibitor).  Supplies of Berinert are expected…

Safety in Lactation: Drugs used for allergic conditions

22 May 2017Additional information relating to breastfeeding To be used in conjunction with individual drug entries for specific information and guidance. Antihistamines Non-sedating antihistamines (NSA) are preferred…
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Lactation Safety Information

Yes
-
No published evidence of safety
Low levels anticipated in milk due to the drug’s properties and likely to be degraded in infant’s gastrointestinal tract
Used in full-term neonates from birth
6 September 2021

New Medicines

Berinert (EU), Haegarda (US)Hereditary angioedema -prophylaxis, subcutaneous formulation

Information

Berinert (EU), Haegarda (US)
New formulation
CSL Behring
CSL Behring

Development and Regulatory status

Licensed but not launched
Phase III Clinical Trials
Launched
Yes
Dec 21Availability in UK uncertain [13].
Dec 18A launch date has not yet been finalised in the UK [12].
Jan 18MA approved by the MHRA for a Berinert 2000 unit and 3000 unit preparation (powder and solvent) for solution for injection. Assume it is the SC product previously launched in the US [11].
Jul 17In the US, Haegarda is available as as a white lyophilized powder supplied in single-use vials containing 2000 or 3000 International Units of C1-INH [10].
Jul 17Launched in US. Priced at around $140,000 a year - 30% less than Cinryze [9].
Jun 17Haegarda subcutaneous formulation won an Orphan Drug designation in the US [7].
Jun 17Approved in US with launch expected Q3 2017. Pricing details haven´t yet been released [6,7].
Aug 16CSL Behring announce that the FDA has accepted the Biologics Licence Application for subcutaneous low volume c1 esterase inhibitor as prophylaxis of HAE attacks [5].
Feb 15PIII COMPACT study now due to complete Q3 2015 [4].
Mar 14P3 COMPACT study due to complete Q2 2015 [3].

Category

C1 esterase inhibitor
HAE is a genetic disorder with an estimated incidence of 1 in 50,000. There may be many undiagnosed cases.
Hereditary angioedema -prophylaxis, subcutaneous formulation
Subcutaneous

Trial or other data

Jun 17Approval in US was based on efficacy results for Haegarda demonstrated in a multicentre controlled clinical trial of 90 subjects aged 12 to 72 years with symptomatic HAE. Subjects were randomised to receive twice per week subcutaneous doses of either 40 IU/kg or 60 IU/kg, and the treatment effect was compared to a placebo treatment period. During the 16 week treatment period, patients in both treatment groups experienced a significantly reduced number of HAE attacks vs placebo. The most common side effects included injection site reactions, hypersensitivity (allergic) reactions, nasopharyngitis (swelling of the nasal passages and throat) and dizziness [8].
Feb 14The first patient enrolled in the PIII COMPACT study (NCT01912456; Clinical Studies for Optimal Management in Preventing Angioedema with Low-Volume Subcutaneous C1-inhibitor Replacement Therapy) [2].
Aug 13NCT01912456 is a double-blind, randomized, placebo-controlled, cross-over study to evaluate the clinical efficacy and safety of subcutaneous human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema. 80 patients will be randomised to one of 4 groups as follows: high or low dose C1-esterase inhibitor for 16 weeks followed by placebo, or placebo for 16 weeks followed by high or low dose inhibitor. The primary outcome is the time-normalized number of hereditary angioedema attacks up to 32 weeks. The study starts Oct 13 and is due to complete Jun 15 [1].

Berinert (EU), Haegarda (US)Kidney transplant rejection - antibody-mediated rejection in adults who have had an inadequate response to standard-of-care treatment

Information

Berinert (EU), Haegarda (US)
Licence extension / variation
CSL Behring
CSL Behring

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Jan 21Development discontinued due to commercial uncertainties arising from COVID-19 pandemic [5].

Category

C1 esterase inhibitor
Most kidney transplants develop chronic rejection within a decade, and there are currently no effective treatment options [2].
Kidney transplant rejection - antibody-mediated rejection in adults who have had an inadequate response to standard-of-care treatment
Intravenous and
Subcutaneous injection