DelveInsight’s, “Hereditary Angioedema – Pipeline Insight, 2022,” report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in Hereditary Angioedema pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Hereditary Angioedema: Overview
Hereditary Angioedema is a disorder characterized by recurrent episodes of severe swelling (angioedema). The most common areas of the body to develop swelling are the limbs, face, intestinal tract, and airway. Minor trauma or stress may trigger an attack, but swelling often occurs without a known trigger. Episodes involving the intestinal tract cause severe abdominal pain, nausea, and vomiting. Swelling in the airway can restrict breathing and lead to life-threatening obstruction of the airway. About one-third of people with this condition develop a non-itchy rash called erythema marginatum during an attack.
Symptoms of Hereditary Angioedema typically begin in childhood and worsen during puberty. On average, untreated individuals have an attack every 1 to 2 weeks, and most episodes last for about 3 to 4 days. The frequency and duration of attacks vary greatly among people with Hereditary Angioedema, even among people in the same family.
There are three types of Hereditary Angioedema, called types I, II, and III, which can be distinguished by their underlying causes and levels of a protein called C1 inhibitor in the blood. The different types have similar signs and symptoms. Type III was originally thought to occur only in women, b.
Symptoms
The symptoms of Hereditary Angioedema includes:
Diagnosis
It is important to note that MOST cases of angioedema or swelling are NOT HAE or C1 Inhibitor Deficiency. Laboratory analysis of blood samples, or genetic samples, are required to establish an HAE diagnosis.
There are three specific blood tests used to confirm Hereditary Angioedema Type I or II.
Treatment
The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition
Report Highlights
This segment of the Hereditary Angioedema report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Hereditary Angioedema Emerging Drugs
BCX7353: BioCryst Pharmaceuticals
Berotralstat (BCX7353) is an oral inhibitor of plasma kallikrein in development for the prevention and treatment of Hereditary Angioedema (HAE). The US Food and Drug Administration (FDA) has accepted and filed its new drug application (NDA) for the approval of oral, once daily berotralstat (BCX7353). The Prescription Drug User Fee Act (PDUFA) date for the NDA is December 2020.
KVD900: KalVista Pharmaceuticals
KVD900 is a candidate from our oral portfolio of plasma kallikrein inhibitors. It exhibits high solubility and high permeability and is uniquely suited for on-demand treatment of HAE attacks, with rapid uptake into the plasma and high plasma concentrations. Data in phase II clinical trial for KVD900 is anticipated in the fourth quarter of 2020.
PHA121: Pharvaris
PHA121 is a novel small molecule with drug-like properties. In preclinical studies, PHA121 demonstrates highly potent and selective competitive antagonism of the B2 receptor, and shows rapid and potent activity on oral dosing in a bradykinin-mediated disease model. It is currently in phase I stage of development.
BMN 331: BioMarin Pharmaceutical
BMN 331 is a gene therapy product candidate for HAE. It is currently in preclinical stage of development.
Further product details are provided in the report……..
This segment of the report provides insights about the different Hereditary Angioedema drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 30+ key companies which are developing the therapies for Hereditary Angioedema. The companies which have their Hereditary Angioedema drug candidates in the most advanced stage, i.e. NDA include BioCryst Pharmaceuticals and others.
DelveInsight’s report covers around 30+ products under different phases of clinical development like
Hereditary Angioedema pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Products have been categorized under various Molecule types such as
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Hereditary Angioedema therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hereditary Angioedema drugs.
Current Treatment Scenario and Emerging Therapies:
Introduction
Executive Summary
Hereditary Angioedema: Overview
Pipeline Therapeutics
Therapeutic Assessment
Hereditary Angioedema – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Hereditary Angioedema Collaboration Deals
Late Stage Products (Phase III and NDA)
BCX7353: BioCryst Pharmaceuticals
Mid Stage Products (Phase II)
KVD900: KalVista Pharmaceuticals
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
PHA121: Pharvaris
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
BMN 331: BioMarin Pharmaceutical
Inactive Products
Hereditary Angioedema Key Companies
Hereditary Angioedema Key Products
Hereditary Angioedema- Unmet Needs
Hereditary Angioedema- Market Drivers and Barriers
Hereditary Angioedema- Future Perspectives and Conclusion
Hereditary Angioedema Analyst Views
Hereditary Angioedema Key Companies
Appendix
List of Table
Table 1: Total Products for Hereditary Angioedema
Table 2: Late Stage Products
Table 3: Mid Stage Products
Table 4: Early Stage Products
Table 5: Pre-clinical & Discovery Stage Products
Table 6: Assessment by Product Type
Table 7: Assessment by Stage and Product Type
Table 8: Assessment by Route of Administration
Table 9: Assessment by Stage and Route of Administration
Table 10: Assessment by Molecule Type
Table 11: Assessment by Stage and Molecule Type
Table 12: Inactive Products
List of Figures
Figure 1: Total Products for Hereditary Angioedema
Figure 2: Late Stage Products
Figure 3: Mid Stage Products
Figure 4: Early Stage Products
Figure 5: Preclinical and Discovery Stage Products
Figure 6: Assessment by Product Type
Figure 7: Assessment by Stage and Product Type
Figure 8: Assessment by Route of Administration
Figure 9: Assessment by Stage and Route of Administration
Figure 10: Assessment by Molecule Type
Figure 11: Assessment by Stage and Molecule Type
Figure 12: Inactive Products
• BioCryst Pharmaceuticals
• KalVista Pharmaceuticals
• Pharvaris
• BioMarin Pharmaceutical
• Ionis Pharmaceuticals, Inc.
• Pharming Technologies B.V.
• Adverum Biotechnologies
• Intellia Therapeutics
• Pharming Group NV
