Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2024

This “Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Familial Lipoprotein Lipase Deficiency 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

Familial Lipoprotein Lipase Deficiency: Understanding

Familial Lipoprotein Lipase Deficiency: Overview

Familial lipoprotein lipase (LPL) deficiency is a rare genetic metabolic disorder characterized by a deficiency of the enzyme lipoprotein lipase. Deficiency of this enzyme prevents affected individuals from properly digesting certain fats and results in massive accumulation of fatty droplets called chylomicrons in the circulation (chylomicronemia) and consequently also an increase of the plasma concentration of fatty substances called triglycerides. Affected individuals often experience episodes of abdominal pain, acute recurrent inflammation of the pancreas (pancreatitis), abnormal enlargement of the liver and/or spleen (hepatosplenomegaly), and the development of skin lesions known as eruptive xanthomas. Familial LPL deficiency is caused by changes (mutations) in the lipoprotein lipase (LPL) gene and is inherited in an autosomal recessive pattern. Recently, mutations in other genes besides LPL were found to cause a clinical picture similar to LPL deficiency.

Signs & Symptoms
Most cases of familial LPL deficiency are identified during childhood, usually before the age of 10. In approximately 25 percent of patients, the disorder is identified during the first year of life. Some affected individuals may not be identified until adulthood. For example, some women may not be diagnosed until after becoming pregnant or when they begin taking contraceptive medication.

The severity of familial LPL deficiency varies depending upon the degree of chylomicronemia, which fluctuates depending upon the amount fat in an individual’s diet. The main symptoms are abdominal pain, pancreatitis, eruptive xanthomas and hepatosplenomegaly.

The most common symptom of familial LPL deficiency is episodic abdominal pain. The severity of abdominal pain can vary, ranging from mild to severe and, in some people, can be incapacitating. The pain may be located in the upper, central region (epigastric area) of the abdomen and can radiate to cause back pain. In some people, the pain may be widespread (diffuse) and can potentially resemble acute abdomen (peritonitis). In the past, this has led to unnecessary surgery.

Diagnosis
The diagnosis of LPL deficiency is established in a proband by the identification of biallelic pathogenic variants in LPL on molecular genetic testing.

Treatment
Treatment of manifestations: Treatment is based on medical nutrition therapy to maintain plasma triglyceride concentration below 1000 mg/dL. Maintenance of triglyceride levels below 2000 mg/dL prevents recurrent abdominal pain. Restriction of dietary fat to =20 g/day or 15% of a total energy intake is usually sufficient to reduce plasma triglyceride concentration and to keep the individual with familial LPL deficiency free of symptoms. An acute pancreatitis episode is treated with standard care.

Prevention of secondary complications: Prevention of recurrent acute pancreatitis decreases the risk of developing diabetes mellitus.

Surveillance: Monitoring of plasma triglycerides.

Agents/circumstances to avoid: Agents known to increase endogenous triglyceride concentration such as alcohol, oral estrogens, diuretics, isotretinoin, glucocorticoids, selective serotonin reuptake inhibitors, and beta-adrenergic blocking agents; fish oil supplements are contraindicated because they contribute to chylomicron levels.

Pregnancy management: For pregnant women with LPL deficiency, extreme dietary fat restriction to < 2 g/day during the second and third trimesters of pregnancy with close monitoring of plasma triglyceride concentration is recommended.

Other: The lipid-lowering drugs that are used to treat other disorders of lipid metabolism are not effective in individuals with familial LPL deficiency.

Familial Lipoprotein Lipase Deficiency- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Familial Lipoprotein Lipase Deficiency pipeline landscape is provided which includes the disease overview and Familial Lipoprotein Lipase Deficiency treatment guidelines. The assessment part of the report embraces, in depth Familial Lipoprotein Lipase Deficiency commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Familial Lipoprotein Lipase Deficiency collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Familial Lipoprotein Lipase Deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Familial Lipoprotein Lipase Deficiency.

Familial Lipoprotein Lipase Deficiency Emerging Drugs Chapters

This segment of the Familial Lipoprotein Lipase Deficiency 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.

Familial Lipoprotein Lipase Deficiency Emerging Drugs

Olezarsen: Ionis Pharmaceuticals, Inc.

Olezarsen, formerly known as IONIS-APOCIII-LRx and AKCEA-APOCIII-LRx, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of apoC-III for patients who are at risk of disease due to elevated triglyceride levels. ApoC-III is a protein produced in the liver that regulates triglyceride metabolism in the blood. People with severely elevated triglycerides, such as people with FCS, are at high risk for acute pancreatitis and an increased risk of cardiovascular disease, or CVD. The drug is in Phase III clinical evaluation for the treatment of familial lipoprotein lipase deficiency.

Familial Lipoprotein Lipase Deficiency: Therapeutic Assessment

This segment of the report provides insights about the different Familial Lipoprotein Lipase Deficiency drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Familial Lipoprotein Lipase Deficiency

There are approx. 5+ key companies which are developing the therapies for Familial Lipoprotein Lipase Deficiency. The companies which have their Familial Lipoprotein Lipase Deficiency drug candidates in the most advanced stage, i.e. phase III.

Phases

This report covers around 5+ products under different phases of clinical development like

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

Route of Administration

Familial Lipoprotein Lipase Deficiency pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Familial Lipoprotein Lipase Deficiency: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Familial Lipoprotein Lipase Deficiency 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 Familial Lipoprotein Lipase Deficiency drugs.

Familial Lipoprotein Lipase Deficiency Report Insights

• Familial Lipoprotein Lipase Deficiency Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Familial Lipoprotein Lipase Deficiency 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 Familial Lipoprotein Lipase Deficiency drugs?
• How many Familial Lipoprotein Lipase Deficiency drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Familial Lipoprotein Lipase Deficiency?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Familial Lipoprotein Lipase Deficiency 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 Familial Lipoprotein Lipase Deficiency and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Ionis Pharmaceuticals, Inc.
• Arrowhead Pharmaceuticals
• NorthSea Therapeutics

Key Products

• Olezarsen
• ARO-APOC3
• SEFA-6131

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