Hereditary Angioedema Market – Efficient Disease Management for Orphan Disease
Hereditary angioedema (HAE) is a rare disorder related with the immune system. The life-threatening condition is caused due to the lack of C1-esterase inhibitor (C1-INH) causing the blood vessels to dilate. The symptoms of the disease include recurrent episodes of edema in various body parts such as hands, feet, face and airways. HAE is classified into 3 types: Type I HAE, Type II HAE and Type III HAE based on the reduction in its synthesis of inhibitor or the formation of a dysfunctional protein. Although, the condition is hereditary, the absence of a family history does not rule out the diagnosis of HAE, indicating that as many as 25% of HAE cases result from a spontaneous mutation of the C1-inhibitor gene at conception.
The global hereditary angioedema market was valued at US$ 1,563.7 million in 2016 and is expected to witness a robust CAGR of 9.1% over the forecast period (2017 - 2025).
Figure 1. Global Hereditary Angioedema Market (US$ Mn), by Region, 2016
Increasing prevalence and rising awareness of hereditary angioedema to augment market growth
The increasing number of hereditary angioedema cases and the hereditary nature giving the disease an ability to pass the defective gene to the future generations is expected to propel market growth. According to the survey by International Hereditary Angioedema Organization (HAEi), 2015, the global prevalence of hereditary angioedema is estimated to be around 1 in 10,000 to 50,000 people, meaning that around a quarter million people suffer globally from this rare and life threatening deficiency condition. Also, according to National Association for Rare Disorders, 2016, the risk of passing the abnormal gene from affected parent to offspring is 50% for each pregnancy regardless of the sex of the resulting child. North America dominates the hereditary angioedema market, owing to the higher number of drugs approved in the regions, presence of major manufacturers and the presence of various organizations serving HAE patients. In 2017, the U.S. Food and Drug Administration (FDA) approved Haegarda (C1-esterase inhibitor) manufactured by CSL Behring for routine prophylaxis to prevent HAE attacks in adolescent and adult patients. However, the rare occurrence of the disease make it difficult for efficient diagnosis and either opt for alternative therapies to control the disease, thus accounting to be the major factors restraining the market growth.
Increasing support from non-profit organizations towards intensive research is expected to accelerate the market
The organizations are focusing on increasing global HAE awareness by educating and empowering patient, providing access to suitable therapy/treatment. The organizations are also working towards intensive research to find a cure. For instance, the non-profit organization named HAEi is dedicated towards spreading awareness of C1-inhibitor deficiencies and to improve diagnosis, facilitating access to reimbursement of the HAE therapies, enabling lifelong health for the global patients. The organization also initiated the Global Access Program (HAEi GAP) in 2015, to provide the access to the approved HAE medications in countries where modern therapies are otherwise unavailable. Moreover, the non-profit organization- US Hereditary Angioedema Association is committed to advance and conduct clinical research designed to improve the lives of HAE patients. The major players operating the global hereditary angioedema market include CSL Behring, Shire Plc., Pharming Healthcare, Inc., BioCryst Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., Arrowhead Pharmaceuticals, Adverum Biotechnologies and Attune Pharmaceuticals, Inc.
Hereditary angioedema (HAE) is characterized by acute skin and mucosal edema caused by an autosomal dominant mutation of the genes. The current treatments of the condition include the HAE drugs which can be administered intravenously, subcutaneously or taken orally. The disease causes swelling particularly of the face and airways and abdominal cramping. The World Allergy Organization (WAO) has set out guidelines to use the different class of drugs such as esterase inhibitors, kallikrein inhibitors and other drugs approved to treat hereditary angioedema.
The major factor driving growth of the hereditary angioedema market include intense R&D initiatives by the manufacturers to treat the condition, increasing prevalence of hereditary angioedema and the presence of promising drug pipeline. For instance, in 2017, Attune Pharmaceuticals, revealed the successful study results the first preclinical data results for ATN-249, a novel orally administered plasma kallikrein inhibitor for the treatment of Hereditary Angioedema (HAE). Also, the approval of novel therapies by the regulatory agencies is expected to be a factor augmenting market growth. For instance, in 2014, the FDA approved Ruconest manufactured by Pharming Healthcare, a recombinant C1-esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with HAE. However, lack of awareness regarding the diseases, inefficient diagnosis and high cost of drugs are the factors restraining hereditary angioedema market growth.
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.
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