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Hereditary Angioedema Therapeutics Pipeline Analysis

Hereditary Angioedema Therapeutics Pipeline Analysis, 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments

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The pipeline of hereditary angioedema is increasing on account of rising instances of angioedema, globally. Currently, an estimated prevalence of hereditary angioedema in the world is 1 in 10,000 to 1 in 150,000. Major factors driving the growth of pipeline for hereditary angioedema include increasing prevalence of attacks of angioedema and rising cases of angioedema in association with other conditions like significant hypotension resulting from abdominal attacks, low market penetration as very less number of companies are making an analog and increasing awareness of health issues. The impairment in quality of life and rising medical cost also act as the driving factor for the hereditary angioedema pipeline.

Angioedema can be defined as swelling of mucosal or skin tissues, which leads to extravasation of fluid into the interstitium and loss of vascular integrity. There are two basic types of angioedema, histaminergic angioedema and non-histaminergic angioedema. Histaminergic is basically associated with urticaria. Non-histaminergic angioedema is usually not associated with urticaria, and is only mediated by bradykinin. The non-histaminergic angioedema is further divided in four categories namely, angiotensin-converting enzyme (ACE) inhibitor-associated angioedema, hereditary angioedema (HAE), idiopathic angioedema and acquired angioedema. Hereditary angioedema is symptomatized as episodic swelling disorder and arises from autosomal dominant trait. Clinical features include nonpitting edema of external body surfaces, abdominal pain, swelling of the extremities and swelling of the mouth and throat, which can lead to asphyxiation.

Many trials are being conducted for the treatment of Hereditary angioedema. Pharming Technologies B.V. is developing drug plasma derived rhC1INH or pdC1INH, also known as Ruconest which is C1 inhibitor. The deficiency of C1 inhibitor can be inherited or acquired or both as it leads to recurrent life threatening angioedema, when the larynx is involved. The C1 inhibitor deficiency is an autosomal dominant trait. The company is also assessing its safety and efficacy study in Hereditary angioedema. BioCryst Pharmaceuticals is in the process of developing a drug known as BCX7353 to prevent an angioedema that attacks especially the patients suffering from hereditary angioedema. The drug is in Phase II stage of development and is expected to complete the study in two part depending upon the dosage of the drug. The Dyax Corporation is conducting a clinical trial on a drug which is a recombinant protein, known as Ecallantide, which is reversible inhibitor of kallikrein and it has shown to increase in case of acute attacks of hereditary angioedema. The drug also helps in preventing angiotensin converting enzyme inhibitor induced angioedema. Shire plc is also conducting a post marketing investigation of drug Firazyr in countries where it is approved for hereditary angioedema attacks.

Various collaborations are undergoing between companies, universities and healthcare organization for the development of drugs to treat hereditary angioedema. Adverum Biotechnologies, Inc. is developing a drug with the name ADVM-053, for treatment of hereditary angioedema.

Some of the companies having a pipeline of hereditary angioedema therapeutics include BioCryst Pharmaceuticals, Inc., CSL Limited, Dyax Corp., Cevec Pharmaceuticals GmbH, Global Blood Therapeutics, Inc., Isis Pharmaceuticals, Inc., iBio, Inc. Pharming Group N.V., Ra Pharmaceuticals, Inc., Shire Plc.

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