Lipoprotein lipase deficiency

Brazilian Regulatory Authority Grants Approval for Waylivra™ for Familial Partial Lipodystrophy

Retrieved on: 
Thursday, December 1, 2022
Abdominal pain, FPL, Enzyme replacement therapy, European Medicines Agency, PTC Therapeutics, Union, Pancreatitis, TG, Triglyceride, Chylomicron, Risk, Liver disease, Insulin resistance, Anovulation, Therapy, Hypertriglyceridemia, Cognition, Brain, ANVISA, Patient, HTG, Anxiety, Diet, Brazilian Health Regulatory Agency, Disease, Adipose tissue, Diabetes, Acute pancreatitis, Depression, Cirrhosis, FCS, Dyslipidemia, Hyperandrogenism, Quality of life, PTC, Lipoprotein lipase deficiency, Woman, LPL, Liver, Pharmaceutical industry, Medical imaging, Volanesorsen, Ionis

SOUTH PLAINFIELD, N.J., Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This is the first approval globally for Waylivra for the FPL indication. Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).

Key Points: 
  • SOUTH PLAINFIELD, N.J., Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agncia Nacional de Vigilncia Sanitria), has approved Waylivra (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil.
  • This is the first approval globally for Waylivra for the FPL indication.
  • Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).
  • ANVISA's approval for Waylivra for FPL was based on results from the Phase 2/3 BROADEN study in patients with familial partial lipodystrophy.

Global Viral Vector Manufacturing Market Trajectory & Analytics Research Report 2022 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, April 6, 2022
Science, Other Science, Research, Pharmaceutical, Health, Infectious Diseases, Clinical Trials, Other Health, Virus, Cell, CAGR, Population, Human, Investment, SCID, Growth, Viral vector, AAV, Vaccine, Research, Molecular biology, Prevalence, Genome, Safety, Ageing, COVID-19, Infection, Viral, Modification, Lipoprotein lipase deficiency, Vector (mathematics and physics), Lists of diseases, Toxicity, Pet

The "Viral Vector Manufacturing - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Viral Vector Manufacturing - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.
  • Viral vectors are currently being used for vaccines development and gene therapy in addition to their use in molecular biology.
  • Another important property of viral vectors is that they are of low toxicity, having limited impact on cell physiology.
  • In the global Adeno-associated Viral Vectors segment, USA, Canada, Japan, China and Europe will drive the 19.7% CAGR estimated for this segment.

Global Viral Vector Manufacturing Market to Reach $1.2 Billion by 2026

Retrieved on: 
Wednesday, March 23, 2022
CAGR, Conditional sentence, Viral vector, Vaccine, Gesellschaft mit beschränkter Haftung, Forecasting, Research, Genetic disorder, Pharma, Adoption, Genome, Mindful Scientific Inc., Vector (mathematics and physics), Thermo Fisher Scientific, Virus, Population, Players, Marketing, SCID, Molecular biology, Ageing, Lipoprotein lipase deficiency, Toxicity, DNA, Merck Group, Investment, Growth, Gendicine, Internet, Intelligence, Lists of diseases, SAN, Cell, Human, Infection, AAV, Diosynth, BioNTech, SV40, Genelux Corporation, Prevalence, Safety, GIA, COVID-19, Viral, Pharmaceutical industry, Pet, Disease, Others, Cancer

SAN FRANCISCO, March 23, 2022 /PRNewswire/ --A new market study published by Global Industry Analysts Inc., (GIA) the premier market research company, today released its report titled "Viral Vector Manufacturing - Global Market Trajectory & Analytics" .

Key Points: 
  • SAN FRANCISCO, March 23, 2022 /PRNewswire/ --A new market study published by Global Industry Analysts Inc., (GIA) the premier market research company, today released its report titled "Viral Vector Manufacturing - Global Market Trajectory & Analytics" .
  • Amid the COVID-19 crisis, the global market for Viral Vector Manufacturing estimated at US$661.8 Million in the year 2022, is projected to reach a revised size of US$1.2 Billion by 2026, growing at a CAGR of 18.5% over the analysis period.
  • The U.S. Market is Estimated at $397 Million in 2022, While China is Forecast to Reach $49 Million by 2026
    The Viral Vector Manufacturing market in the U.S. is estimated at US$397 Million in the year 2022.
  • Global Industry Analysts, Inc., ( www.strategyr.com ) is a renowned market research publisher the world`s only influencer driven market research company.

Familial Lipoprotein Lipase Deficiency: A Rare Inherited Condition, in Which the Normal Breakdown of Fats in the Body is Affected - Global Pipeline Insight Report 2020 - ResearchAndMarkets.com

Retrieved on: 
Friday, April 2, 2021
Health, General Health, Branches of biology, Metabolic disorders, Lipids, Lipid metabolism disorders, Lipoprotein lipase deficiency, Cardiology, Lipase, Lipoprotein lipase, Lipoprotein, Triglyceride, Hyperlipidemia, Acid lipase disease

The "Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • This "Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020," report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Familial Lipoprotein Lipase Deficiency pipeline landscape.
  • Familial Lipoprotein Lipase Deficiency (LPLD) is a rare inherited condition, in which the normal breakdown of fats in the body is affected.
  • Treatment for Familial Lipoprotein Lipase Deficiency aims to control the symptoms and blood triglyceride levels with a very low-fat diet.

Arrowhead Pharmaceuticals Files IND to Begin Phase 2b Study of ARO-APOC3 in Patients with Severe Hypertriglyceridemia

Retrieved on: 
Monday, March 1, 2021
Health, FDA, Genetics, Clinical trials, Research, Science, Pharmaceutical, Health sciences, Health, Medical research, Clinical research, Design of experiments, Drug discovery, Pharmaceutical industry, Lipid metabolism disorders, Clinical trial, Apolipoprotein C-III, Dose-ranging study, Lipoprotein lipase deficiency

Based on these highly promising results, we intend to conduct Phase 2b and Phase 3 clinical studies across a wide spectrum of patient populations, pending review by regulators.

Key Points: 
  • Based on these highly promising results, we intend to conduct Phase 2b and Phase 3 clinical studies across a wide spectrum of patient populations, pending review by regulators.
  • Following the FDAs review of the IND, the company intends to initiate a Phase 2b dose-finding clinical study (ARO-APOC3-2001) in patients with severe hypertriglyceridemia (sHTG).
  • Additional clinical studies planned in 2021 include a Phase 2b dose-finding clinical study in patients with mixed dyslipidemia and a Phase 3 clinical study in patients with familial chylomicronemia syndrome (FCS).
  • Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them.

WAYLIVRA▼® (volanesorsen), the First and Only Therapy for Familial Chylomicronaemia Syndrome, an Ultra-Rare and Life-Threatening Condition, to Be Available on the NHS in Scotland

Retrieved on: 
Monday, November 9, 2020
Biotechnology, Pharmaceutical, Health, Clinical research, Health, Medicine, Therapeutic gene modulation, Antisense RNA, Orphan drugs, Volanesorsen, Lipoprotein lipase deficiency, Placebo-controlled study, National Health Service, Placebo, volanesorsen, FCS

This means that eligible patients will be able to access volanesorsen through NHS Scotland while additional clinical effectiveness data are gathered.

Key Points: 
  • This means that eligible patients will be able to access volanesorsen through NHS Scotland while additional clinical effectiveness data are gathered.
  • The SMC will review this data after three years and make a final decision on its routine use in NHS Scotland.
  • Volanesorsen, which received conditional marketing authorisation in Europe in May 2019, is the only therapy indicated for people with familial chylomicronaemia syndrome (FCS).
  • (2017) The APPROACH Study: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS).

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

Retrieved on: 
Monday, March 23, 2020
Lipid metabolism disorders, Rare diseases, Lipoprotein lipase deficiency, Drug pipeline, Health

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.

Key Points: 
  • 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.
  • The FCS epidemiology division provide the insights about historical and current FCS patient pool and forecasted trend for each seven major countries.
  • This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
  • 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.

Familial Chylomicronemia Syndrome Foundation Commends FDA Decision to Create New Advisory Group to Support Rare Disease Drug Review Processes

Retrieved on: 
Tuesday, January 21, 2020
Lipid metabolism disorders, Health, Lipoprotein lipase deficiency, Food and Drug Administration, Rare disease, Articles

SAN DIEGO, Jan. 21, 2020 /PRNewswire/ --The FCS Foundation, the nation's leading advocacy and support organization for people affected by familial chylomicronemia syndrome (FCS), today announced the foundation's support of recent comments by the U.S. Food and Drug Administration (FDA) to offer rare disease groups support across agency divisions to improve the review process for drugs to treat rare diseases.

Key Points: 
  • SAN DIEGO, Jan. 21, 2020 /PRNewswire/ --The FCS Foundation, the nation's leading advocacy and support organization for people affected by familial chylomicronemia syndrome (FCS), today announced the foundation's support of recent comments by the U.S. Food and Drug Administration (FDA) to offer rare disease groups support across agency divisions to improve the review process for drugs to treat rare diseases.
  • "We applaud the FDA for listening to us and other members of the rare disease community and for taking this bold action to improve the regulatory review process in an effort to advance more treatments to approval faster.
  • The new group is part of a reorganization of the OND at the FDA and will exist within the soon-to-be established Division of Rare Diseases and Medical Genetics (DRDMG).
  • The FCS Foundation is the only foundation dedicated to patients suffering from familial chylomicronemia syndrome.

Akcea Recognizes Landmark Year for Familial Chylomicronemia Syndrome (FCS) Community with Commemoration of 2nd Annual FCS Awareness Day

Retrieved on: 
Friday, November 1, 2019
Organ systems, Antisense RNA, Syndromes, Lipodystrophy, Lipid metabolism disorders, Ionis Pharmaceuticals, Familial partial lipodystrophy, Ionis, Lipoprotein lipase deficiency

BOSTON, Nov. 01, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today recognizes Familial Chylomicronemia Syndrome (FCS) Awareness Day.

Key Points: 
  • BOSTON, Nov. 01, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today recognizes Familial Chylomicronemia Syndrome (FCS) Awareness Day.
  • We are so encouraged that people in Europe living with FCS now have a treatment available.
  • and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL.
  • Additional information about Akcea is available at www.akceatx.com and you can follow us on twitter at @akceatx.

Familial Chylomicronemia (Type I Hyperlipoproteinemia) Pipeline Insight Report 2019 Featuring Aegerion, Catabasis, CymaBay, Kadmon, & Novartis - ResearchAndMarkets.com

Retrieved on: 
Tuesday, April 9, 2019
Health, Pharmaceutical, Lipid metabolism disorders, Hyperlipidemia, Lipoprotein lipase deficiency, Drug pipeline, Health

The "Familial Chylomicronemia (Type I Hyperlipoproteinemia) - Pipeline Insight, 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Familial Chylomicronemia (Type I Hyperlipoproteinemia) - Pipeline Insight, 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The report offers comprehensive Insight of the pipeline (under development) therapeutics scenario and growth prospects across Familial Chylomicronemia (Type I Hyperlipoproteinemia) development.
  • The report assesses the active Familial Chylomicronemia (Type I Hyperlipoproteinemia) pipeline products by developmental stage, product type, molecule type, and administration route.
  • clinical, pre-clinical and discovery stages for the Familial Chylomicronemia (Type I Hyperlipoproteinemia)
    The report provides pipeline product profiles which includes product description, developmental activities, licensors & collaborators and chemical information
    Provides pipeline assessment by monotherapy and combination therapy products, stage of development, route of administration, and molecule type for Familial Chylomicronemia (Type I Hyperlipoproteinemia)