Contract: Nhs framework - icatibant and c1 esterase
products for the treatment of hereditary angioedema
It was reported yesterday that the US Food and Drug Administration (USFDA) has approved the technology transfer of Ireland-based Shire's Cinryze - a C1 esterase
inhibitor - drug product manufacturing process to its Vienna, Austria manufacturing site.
Objective: Hereditary angioedema (HAE) is a rare autosomal-dominant disease characterized by recurring attacks of nonpruritic, nonpitting edema caused by an inherited deficiency or dysfunction in the C1 esterase
inhibitor (C1 INH).
Angioedema can be classified into three forms: a) an idiopathic form with unknown pathogenetic mechanisms, b) an extrinsic factor-induced form (due to histamine [IgE- and non IgE- triggered] or kinin-mediated pathophysiology), and c) a hereditary/acquired form associated with C1 esterase
inhibitor deficiency or dysfunction (5).
00) and a slightly elevated C1 esterase
inhibitor (C1-INH) level of 0.
Patients have low levels or activity of C1 esterase
inhibitor (C1INH), C4, and C1q.
HAE is caused by a deficiency of the C1 esterase
inhibitor protein, which is present in blood and helps control inflammation (swelling) and parts of the immune system.
1,2) Hereditary and acquired cases usually result from a deficiency of C1 esterase
inhibitor deficiency, which causes an accumulation of bradykinin, leading to soft tissue oedema.
Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase
inhibitor deficiency workshop and beyond.
Ruconest (conestat alfa) is a recombinant human C1 esterase
inhibitor approved for the treatment of angioedema attacks in patients with HAE in the USA, Israel, all 27 EU countries plus Norway, Iceland and Liechtenstein, concluded the companies.
This C1 esterase
inhibitor complements Shire's FIRAZYR icatibant injection for the treatment of acute HAE attacks.
Santarus' product development pipeline includes the investigational drug RUCONEST([R])(recombinant human C1 esterase
inhibitor) for treatment of acute attacks of hereditary angioedema.