Familial Chylomicronemia Syndrome (FCS) Markets, Epidemiology and Forecast to 2030 - Featuring Emerging Drugs AKCEA-ANGPTL3-LRx, Lomitapide, AKCEA-APOCIII - LRx, and ARO-APOC3

DUBLIN, March 23, 2020 /PRNewswire/ -- The "Familial Chylomicronemia Syndrome (FCS) - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the FCS, historical and forecasted epidemiology as well as the FCS market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The FCS market report provides current treatment practices, emerging drugs, FCS market share of the individual therapies, current and forecasted FCS market size from 2017 to 2030 segmented by seven major markets. The report also covers current FCS treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Study Period: 2017-2030

Epidemiology

The total prevalent cases of FCS in the 7MM were found to be 5,801 in 2017 which is expected grow during the study period, i.e., 2017-2030.

The FCS epidemiology division provide the insights about historical and current FCS patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

The disease epidemiology covered in the report provides historical as well as forecasted FCS epidemiology [segmented as Total Prevalence of FCS, Total Diagnosed Prevalence of FCS, Age-specific Diagnosed Prevalence of FCS, Severity-Specific Diagnosed Prevalence of FCS, and Treated Patient Pool of FCS] scenario of FCS in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country-Wise Epidemiology

Estimates show that the highest prevalent population of FCS is in the United States, followed by Japan, Germany, France, and the United Kingdom in 2017.

Drug Chapters

Drug chapter segment of the Familial Chylomicronemia Syndrome report encloses the detailed analysis of Familial Chylomicronemia Syndrome marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Familial Chylomicronemia Syndrome clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Marketed Drugs

Waylivra (volanesorsen): Ionis Pharmaceuticals/Akcea Therapeutic

Waylivra (Volanesorsen) is a 2'-O-(2-methoxyethyl) antisense oligonucleotide (ASO) inhibitor of the molecular target apolipoprotein C-III (APOC-III). Volanesorsen is delivered as a subcutaneous injection of 280 mg/1.5 mL weekly in a single-use pre-filled syringe.

Waylivra is indicated as an adjunct to diet for the treatment of patients with FCS. In May 2019, Akcea and Ionis have received conditional marketing authorization from the European Commission (EC) for the treatment of FCS for Waylivra. The company has launched Waylivra in Germany, however, launch in other European countries is expected in 2020. The company has also initiated ATU (Autorisation Temporaire d'Utilisation - Temporary Use Authorization) in France. This drug is not approved by the US FDA. Also recently in January 2020, NICE rejected Waylivra for the treatment of FCS.

Waylivra is a product of Ionis' proprietary antisense technology. Antisense technology is an innovative platform for discovering first-in-class and/or best-in-class medicines for treating disease. Unlike most other drug technologies that work by affecting existing proteins in the body, antisense medicines target RNA, the intermediary that conveys genetic information from a gene to the protein synthesis machinery in the cell. By targeting RNA instead of proteins, antisense technology can be used to increase, decrease or alter the production of specific proteins.

Emerging Drugs

AKCEA-ANGPTL3-LRx: Akcea Therapeutics/Ionis Pharmaceuticals

AKCEA-ANGPTL3-LRx (ISIS 703802) is under development by Akcea and Ionis. It is a subcutaneously administered investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases.

AKCEA-ANGPTL3-LRx is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver. It is a key regulator of triglycerides, cholesterol, and glucose and energy metabolism. This drug was developed using Ionis's advanced Ligand Conjugated Antisense (LICA) technology platform.

ARO-APOC3: Arrowhead Pharmaceuticals

ARO-APOC3 which is under development by Arrowhead Pharmaceuticals, is a subcutaneously administered RNAi therapeutic targeting Apolipoprotein C-III (APOC3). The company utilizes Targeted RNAi Molecule (TRiMTM) platform for ligand-mediated delivery of this drug. This is mainly designed to enable tissue-specific targeting while being structurally simple. This mechanism offers many potential advantages in the development of disease therapies, such as the ability to target a broad range of genes and proteins with high specificity, and also the disease pathways that have proven difficult to address with traditional small molecule and biologic therapeutics.

AKCEA-APOCIII - LRx: Ionis Pharmaceuticals/Akcea Therapeutics

AKCEA-APOCIII-LRx which is under development by Ionis and Akcea, is a subcutaneously administered generation 2+ ligand-conjugated antisense (LICA) drug designed to inhibit the production of apoC-III, for patients who are at risk of disease due to elevated triglyceride levels.

In January 2020, based on the positive results of phase II trial for hypertriglyceridemia and cardiovascular diseases company announced to pursue the rapid development of AKCEA- APOCIII-LRx for FCS. Results of phase II clinical trial has shown favorable safety and tolerability in hypertriglyceridemia patients. Both primary and key secondary endpoints were met with significant reduction in apoC-III and triglyceride levels. Furthermore, more than 90% of patients achieved serum triglycerides 150 mg/dL at the highest monthly dose.

Market Outlook

The Familial Chylomicronemia Syndrome market in the 7MM is expected to change in the study period 2017-2030. The therapeutic market of Familial Chylomicronemia Syndrome in seven major markets was found to be USD 1 million in 2017 which is expected to increase during study period (2017-2030).

Current treatment choices for FCS can be fragmented down into managing acute crisis related to pancreatitis and chronic management of hypertriglyceridemia to reduce the risk of future episodes. During an episode of acute pancreatitis, complete fasting with parenteral fluid support and analgesia if required is usually very effective.

There is a lack of approved therapies for FCS in the market. As of now, no drug is approved by the United States (Food and Drug Administration) FDA for the management of FCS. Likewise, no therapy is approved in Japan as well.

Only one approved drug for the treatment FCS is currently present which was approved by the EC in Europe is Waylivra. Glybera was the another drug which was approved in October 2012 by European Comission under exceptional circumstances as a treatment for small subsets of adult patients diagnosed with familial LPL deficiency, which was confirmed by genetic testing. But later it was withdrawn from the market by uniQure in October 2017.

The United States Market Outlook

In 2017, the total market size of FCS therapies was found to be USD 0.3 million in the United States which is expected to increase in the study period (2017-2030).

EU-5 Countries: Market Outlook

In 2017, the total market size of FCS therapies was found to be USD 0.5 million in the EU-5 countries which is expected to increase in the study period (2017-2030).

Japan Market Outlook

The total market size of FCS therapies in Japan was found to be USD 0.2 million in 2017.

Pipeline Development Activities

The drugs which are in pipeline includes:
1. AKCEA-ANGPTL3-LRx (Akcea Therapeutics/Ionis Pharmaceuticals): Phase II
2. Lomitapide (Amryt Pharma/Novelion Therapeutics): Phase II
3. AKCEA-APOCIII - LRx (Ionis Pharmaceuticals/Akcea Therapeutics): Phase II
4. ARO-APOC3 (Arrowhead Pharmaceuticals): Phase I/IIa

Pipeline Development Activities

Key Points:
1. The AKCEA-ANGPTL3-LRx Phase II program was designed to include three clinical studies in patients with one of three rare hyperlipidemias, including FCS, Familial Partial Lipodystrophy (FPL), and Homozygous Familial Hypercholesterolemia (HoFH).]
2. Arrowhead Pharmaceuticals is planning to initiate pivotal studies of ARO-APOC3 in 2020 for FCS.
3. Amryt Pharma and Novelion Therapeutics is expecting the launch of Lomitapide in 2023 for the treatment of FCS as mentioned in its May 2019 annual report of the company.
4. In January 2020, based on the positive results of phase II trial for hypertriglyceridemia and cardiovascular diseases Ionis Pharmaceuticals and Akcea Therapeutics announced to pursue the rapid development of AKCEA- APOCIII-LRx for FCS.

Drugs Uptake

In February 2020, Arrowhead Pharmaceuticals reported the interim results for ARO-APOC3 which shows about 95% of reductions in the elevated triglyceride results. Based on its efficacy and safety results this therapy expected to impact the upcoming therapeutic market of FCS significantly.

Access & Reimbursement Scenario

• For Waylivra early access program is ongoing in Europe, the US and Canada and in March 2018, Medicines and Healthcare Products Regulatory Agency (MHRA) granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for the treatment of FCS. According to the decision, eligible patients with FCS are able to access volanesorsen before the EC makes a formal decision for its use in Europe.
• According to the recommendation by National Institute for Health and Care Excellence (NICE) given in January 2020. Waylivra is not recommended, within its marketing authorization, for treating FCS in adults with genetically confirmed FCS who are at high risk of pancreatitis, and when the response to diet and triglyceride-lowering therapy has been inadequate.
• It was also stated by NICE that this recommendation is not intended to affect treatment with Waylivra that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Companies Mentioned

• Ionis Pharmaceuticals/Akcea Therapeutic
• Akcea Therapeutics/Ionis Pharmaceuticals
• Arrowhead Pharmaceuticals
• Amryt Pharma/Novelion Therapeutics

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/eqg5za

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