Hereditary Angioedema - Pipeline Insight, 2024

This “Hereditary angioedema - Pipeline Insight, 2024,” report provides comprehensive insights about 20+ 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.

Geography Covered

• Global coverage

Hereditary angioedema Understanding

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.

Hereditary angioedema Emerging Drugs Chapters

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

KVD900: Kal Vista 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.Currently the drug is in Phase III stage of development for the treatment of Hereditaryangioedema.

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 II stage ofdevelopment.

BMN 331: Bio Marin Pharmaceutical BMN 331 is a gene therapy product candidate for HAE. It is currently in Phase I stageofdevelopment.

Hereditary angioedema: Therapeutic Assessment

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:

Major Players in Hereditary angioedema

There are approx. 20+ 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. Phase III include Kal VistaPharmaceuticals.

Phases

This report covers around 30+ products under different phases of clinical development like

• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage products (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hereditary angioedema: Pipeline Development Activities

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.

Hereditary angioedema Report Insights

• Hereditary angioedema Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hereditary angioedema Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hereditary angioedema drugs?
• How many Hereditary angioedema drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hereditary angioedema?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hereditary angioedema therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hereditary angioedema and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Bio CrystPharmaceuticals
• Kal VistaPharmaceuticals
• Pharvaris
• Bio MarinPharmaceutical
• Ionis Pharmaceuticals, Inc.
• Intellia Therapeutics

Key Products

• BCX7353
• KVD900
• PHA121
• BMN 331
• IONIS-PKK-LRx
• NTLA-2002

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