This “Congenital Hyperinsulinism - Pipeline Insight, 2020” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Congenital Hyperinsulinism 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
Congenital Hyperinsulinism Understanding

Congenital Hyperinsulinism: Overview

Congenital hyperinsulinism is a condition that causes individuals to have abnormally high levels of insulin, which is a hormone that helps control blood sugar levels. People with this condition have frequent episodes of low blood sugar (hypoglycemia). In infants and young children, these episodes are characterized by a lack of energy (lethargy), irritability, or difficulty feeding. Repeated episodes of low blood sugar increase the risk for serious complications such as breathing difficulties, seizures, intellectual disability, vision loss, brain damage, and coma. The severity of congenital hyperinsulinism varies widely among affected individuals, even among members of the same family.

Symptoms

It is often difficult to identify symptoms of HI because they are often confused with typical behaviors of newborns and infants. Common symptoms include irritability, sleepiness, lethargy, excessive hunger and rapid heart rate. More severe symptoms, such as seizures and coma, can occur with a prolonged or extremely low blood sugar level. Common symptoms of low blood sugar in older children and adults include feelings of shakiness, weakness, or tiredness, confusion and rapid pulse. More severe symptoms include seizures or coma.

Diagnosis

The diagnosis of Hyperinsulinism may be quite difficult if one relies on demonstrating a detectable blood insulin concentration at the time of hypoglycemia because insulin levels fluctuate widely over time in patients with Hyperinsulinism. Other signs and chemical markers must be used to provide clues to excess insulin action and are often easier to demonstrate. Hypoglycemia which occurs while an infant is on a glucose infusion is strongly suggestive of Hyperinsulinism. Other clues to excess insulin action are low free fatty acids and ketones at the time of hypoglycemia.

Treatment

Prompt treatment of hypoglycemia due to Hyperinsulinism is essential to prevent brain damage. Unlike other hypoglycemia-causing conditions in which alternative fuels, such as ketones or lactate, may be available for the brain during periods of hypoglycemia, Hyperinsulinism prevents the production of these fuels and leaves the brain without a source of energy. Hypoglycemia can be treated by giving a carbohydrate-containing drink by mouth or if severe, by giving glucose through the vein or by injecting glucagon.

Congenital Hyperinsulinism Emerging Drugs Chapters

This segment of the Congenital Hyperinsulinism report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Congenital Hyperinsulinism Emerging Drugs

ABG 023: AmideBio

ABG-023 is a solution stable, soluble glucagon analog designed to overcome the limitations of glucagon - an effective treatment for CHI, but rendered impractical for long term administration given its instability in solution. The development of ABG-023 was funded through Small Business Innovation Research (SBIR) Phase I and II grants from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Division of the National Institutes of Health (NIH). The US Food and Drug Administration (FDA) Office of Orphan Products Development granted an orphan drug designation to AmideBio's glucagon analog (ABG-023) for the treatment of congenital hyperinsulinism (CHI).

Congenital Hyperinsulinism: Therapeutic Assessment

This segment of the report provides insights about the different Congenital Hyperinsulinism drugs segregated based on following parameters that define the scope of the report.

Major Players in Congenital Hyperinsulinism

There are approx. 5+ key companies which are developing the therapies for Congenital Hyperinsulinism. The companies which have their Congenital Hyperinsulinism drug candidates in early stage, i.e. pre-clinical and Discovery include, AmideBio, Zealand Pharma etc.

Phases

This report covers around 5+ products under different phases of clinical development like
Mid-stage products (Phase II and Phase I/II)
Early-stage products (Phase I/II and Phase I) along with the details of
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates
Route of Administration
Congenital Hyperinsulinism pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Subcutaneous
Intravenous
Oral
Intramuscular
Molecule Type
Products have been categorized under various Molecule types such as
Small molecules
Gene therapy
Proteins and peptides
Hormones
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Congenital Hyperinsulinism: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Congenital Hyperinsulinism 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 Congenital Hyperinsulinism drugs.

Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Congenital Hyperinsulinism R&D. The therapies under development are focused on novel approaches to treat/improve Congenital Hyperinsulinism.
September 2020: Crinetics Pharmaceuticals received Rare Pediatric Disease Designation from FDA for CRN04777 for the treatment of congenital hyperinsulinism
Congenital Hyperinsulinism Report Insights
Congenital Hyperinsulinism Pipeline Analysis
Therapeutic Assessment
Unmet Needs
Impact of Drugs
Congenital Hyperinsulinism 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 Congenital Hyperinsulinism drugs?
How many Congenital Hyperinsulinism drugs are developed by each company?
How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Congenital Hyperinsulinism?
What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Congenital Hyperinsulinism 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 Congenital Hyperinsulinism and their status?
What are the key designations that have been granted to the emerging drugs?
Key Players
Crinetics Pharmaceuticals
Rezolute, Inc.
AmideBio
Key Products
ABG-023
Dasiglucagon
CRN04777