Report Overview

The global hereditary angioedema therapeutics market was pegged at US$ 3.59 billion in 2021 and is expected to witness a CAGR of 16% during the forecast period.

Market Fundamental 

Hereditary angioedema (HAE) is a rare genetic disorder characterized by repeated bouts of subcutaneous and submucosal swelling that are nonpruritic, nonpitting, and lack urticarial symptoms. Due to a blood protein deficit, it results in severe discomfort, nausea, vomiting, diarrhoea, exhaustion, tingling in the muscles, headaches, stomach pain, hoarseness, and mood swings. Additionally, it causes fluid leaks because liquids are constantly building up outside the veins in the hands, feet, intestinal wall, genitals, face, tongue, and throat. For instance, Ionis Pharmaceuticals, a biotechnology business with headquarters in California, is developing an experimental antisense drug called IONIS-PKK-LRx that is ligand-conjugated (LICA). In order to treat patients with hereditary angioedema, the business is studying a pharmacological molecule to decrease the production of prekallikrein, or PKK.

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Market Dynamics 

Due to a deficiency in C1 protein, HAE prevalence is currently significantly rising among people. The need for HAE medicines has increased as a result globally. This is one of the main elements influencing the market, along with the general public's increased awareness of the therapy for HAE. Additionally, major market players are heavily funding research and development (R&D) initiatives to advance the development of orphan drugs. This, in addition to the rising need for subcutaneous injections that are extremely effective at delivering drugs like goserelin, morphine, and insulin, is boosting the market's expansion.

Market Ecosystem 

The global hereditary angioedema therapeutics market has been analysed from three perspectives by drug class, distribution channel and region. 

Hereditary Angioedema Therapeutics Market by Drug Class

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Based on drug class, the hereditary angioedema therapeutics market has been segmented into C-1 esterase inhibitors, kallikrein inhibitors, bradykinin receptor inhibitor, anabolic androgens, antifibrinolytic drugs. The kallikrein inhibitors segment dominated the market during forecast period. The ability of several kallikrein inhibitors to prevent edematous HAE attacks is currently being researched. For this relatively new class of medicinal therapy, there are a lot of interesting compounds in the pipeline, and their commercialization is anticipated to fuel market growth.

Hereditary Angioedema Therapeutics Market by Distribution Channel 

Based on distribution channel, the hereditary angioedema therapeutics market has been segmented into hospital, retail pharmacies and others. The hospital pharmacies segment dominated the market during forecast period. The section can be ascribed to the accessibility and convenience of medications.

Hereditary Angioedema Therapeutics Market by Region 

Based on the region, the global hereditary angioedema therapeutics market has been segmented into North America, Europe, Asia Pacific and Rest of the World. North America is anticipated to dominate the global hereditary angioedema market during the forecast period due to the presence of key players, expanding awareness programmes and research organisations regarding the treatment of the condition, established healthcare infrastructure, and the presence of key players. he primary growth drivers of the industry are increased government efforts, growing research alliances, and a healthy pipeline. For instance, LIONIS-PKK-LRx is an investigational antisense drug that has been ligand-conjugated (LICA) and is being developed by the California-based biotechnology business ionis Pharmaceuticals. In order to treat patients with hereditary angioedema, the business is studying a pharmacological molecule to decrease the production of prekallikrein, or PKK. Cycle Pharmaceuticals introduced SAJAZIR (Icatibant) Injection in September 2021, a fresh, US FDA-approved therapeutic option for those with hereditary angioedema (HAE).

Competitive Landscape 

Some of the prominent players in the global hereditary angioedema therapeutics market include 

• Takeda Pharmaceutical
• CSL-Bering
• Pharming Group NV
• Sanofi-Aventis
• Pfizer Inc.
• Ferring Pharmaceuticals Inc.
• Ionis Pharmaceuticals, Inc.
• BioCryst Pharmaceuticals
• KalVista Pharmaceuticals, Ltd.
• Pharvaris Netherlands B.V.
• Intellia Therapeutics
• Prothya Biosolutions
• BioMarin Pharmaceutical
• Other Notable Players 

Strategic Development 

• In June 2022, Hereditary angioedema (HAE) and fibrodysplasia ossificans progressive (FOP) are two rare diseases for which BioCryst Pharmaceuticals, Inc. creates and develops small molecule medications. Swissmedic, the Swiss surveillance authority for medicines and medical devices, recently approved ORLADEYO (berotralstat), an oral, once-daily medication, for routinely preventing HAE attacks in patients aged 12 and older.
• In March 2022, the biotechnology company Ionis Pharmaceuticals, Inc. reported that the New England Journal of Medicine had published donidalorsen's good Phase 2 findings (NEJM). Ionis Pharmaceuticals, Inc. is testing an experimental antisense drug called donidalorsen for the treatment of people with hereditary angioedema (HAE). Donidalorsen showed a 90% reduction in angioedema events in the Phase 2 study when compared to placebo at an 80 mg monthly dose. Patients who received donidalorsen experienced a significant improvement in quality of life as measured by the Angioedema Quality of Life Questionnaire (AE-QoL).
• In February 2021, the approval of ORLADEYO (Berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 and older was announced by BioCryst Pharmaceuticals Inc. The opinion was adopted by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
• In August 2018, Shire reported that TAKHZYRO, a preventive therapy for hereditary angioedema, had received FDA approval in the United States.

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