The prevalence of HAE is estimated to be approximately 1 case per 50,000. Hereditary angioedema arises from gene mutations that encode an important protease known as a C1 inhibitor belonging to a serine superfamily of proteins. More than 100 mutations have been reported to affect the C1–inhibitor gene, including up to 25? novo mutations. There are 2 main types of HAE that are inherited in an autosomal dominant manner. Type I HAE accounts for approximately 85% of cases and results from a quantitative deficiency of C1 inhibitor. Type II HAE is responsible for approximately 15% of cases and results from a dysfunctional C1 inhibitor protein. More recently, the third form of HAE has been identified as HAE with a normal C1 inhibitor. This condition, formerly known as type III HAE, occurs mostly in females in whom both quantitative and functional studies of C1 inhibitors are normal. Approximately 15% of patients with HAE have the type II form, in which mutations cause the production of a dysfunctional C1INH protein resulting in low C1INH function despite normal levels of antigenic C1INH. A registry of patients with HAE in Norway reported a prevalence of 1.75 per 100,000 inhabitants, while a Spanish registration study detected a minimal prevalence of 1.09 per 100,000.
Comprehensive insight on patient segmentation based on age, sex, types (Type I HAE, Type II HAE, & Type III HAE) which further segregated into Genetic C1-INH deficiency, FXII mutation, and unknown cause (U-HAE), clinical manifestations (Extremities, Face, Genitalia, Lip, GastroIntestinal System, Throat, Respiratory System, and others) has been provided into the epidemiology (Incidence and Prevalence) section of the Hereditary angioedema (HAE) and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China
In terms of pharmacologic therapies, there are few drug candidates are in Phase III, II, I, and pre-clinical stages of development. Key assets among these are Orladeyo from BioCryst; Garadacimab from CSL, IONIS PKK-LR from Ionis, PHVS416 from Pharvaris B.V., and KVD900 from KalVista. N terms of a major upcoming milestone, BioCryst Pharmaceuticals announced it has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of oral, once-daily berotral-stat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks. Limited or difficult vascular access is a complicated process; the use of repeated IV medication such as placement of a long-term catheter is required for therapy which poses additional risks, so the requirement of oral therapy is the critical unmet need.
Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.
Gurugram- Onework Gold Souk mall, Phase I, Sector 43, Haryana-122002, India
Contact no.: +91(124) 404-1731
183-Asylum Street Hartford, CT-06103, USA
USA +1 (267) 244-6955,