Polyneuropathy

Alnylam Announces Preliminary* Fourth Quarter and Full Year 2021 Global Net Product Revenues and Provides Additional Updates

Retrieved on: 
Sunday, January 9, 2022
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Gene silencing, Physiology, Safety, COVID-19, RNA transfection, Novartis, Government, Polyneuropathy, Growth, Therapy, Inclisiran, Nobel Prize, Company, Private Securities Litigation Reform Act, Patient, Adult, Sirna, Cell, TTR, Goal, Science, Nobel Prize in Physiology or Medicine, Central nervous system, Intellectual property, Conference, U.S. Securities and Exchange Commission, Twitter, Medication, Security (finance), RNA interference, Disease, SEC, LinkedIn, Marketing, Instagram, Regeneron Pharmaceuticals, RNA, CNS, Biology, Risk, Drug development, Nasdaq, Bachelor of Medicine, Bachelor of Surgery, Pharmaceutical industry, Lithium, Medical device, Online gambling, Medicine, Alnylam Pharmaceuticals, RNAi Therapeutics, Alnylam Pharmaceuticals, RNAI THERAPEUTICS, ALNYLAM PHARMACEUTICALS

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced its preliminary* fourth quarter and full year 2021 global net product revenues for ONPATTRO, GIVLAARI and OXLUMO and provided additional updates on the products commercial launches.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced its preliminary* fourth quarter and full year 2021 global net product revenues for ONPATTRO, GIVLAARI and OXLUMO and provided additional updates on the products commercial launches.
  • Preliminary global net product revenues for the fourth quarter and full year 2021 were approximately $139 million and $475 million, respectively, representing quarterly and annual growth of 15% and 55% compared to Q3 2021 and full year 2020, respectively.
  • Preliminary global net product revenues for the fourth quarter and full year 2021 were approximately $41 million and $128 million, respectively, representing quarterly and annual growth of 28% and 132% compared to Q3 2021 and full year 2020, respectively.
  • Preliminary global net product revenues for the fourth quarter and full year 2021 were approximately $19 million and $60 million, respectively, representing quarterly growth of 29% compared to Q3 2021.

Alnylam Comments on FDA Approval of Leqvio®, the First siRNA (RNAi Therapeutic) Approved to Reduce LDL-C

Retrieved on: 
Wednesday, December 22, 2021
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Mortality, Sequah Medicine Company, Nobel Prize, Disease, Lists of diseases, LinkedIn, Cell, CNS, Twitter, Sirna, Private Securities Litigation Reform Act, Intellectual property, COVID-19, Regeneron Pharmaceuticals, Physiology, European Medicines Agency, Food, U.S. Securities and Exchange Commission, Drug development, RNA, Biology, Risk, Patient, Nobel Prize in Physiology or Medicine, RNA interference, Diet, Government, Polyneuropathy, LDL, SEC, ESC, Science, Goal, Marketing, Instagram, Therapy, Central nervous system, Safety, Security (finance), RNA transfection, Medication, Novartis, Nasdaq, FDA, Blackstone, Growth, ORION, EMA, Gene silencing, Pharmaceutical industry, Alnylam Pharmaceuticals, Medicine, Inclisiran

Leqvio is the fourth Alnylam-discovered medicine using its RNAi therapeutic platform to be approved to date.

Key Points: 
  • Leqvio is the fourth Alnylam-discovered medicine using its RNAi therapeutic platform to be approved to date.
  • The Leqvio approval marks the first U.S. approval of an RNAi therapeutic indicated to treat a major risk factor for a highly prevalent disease.
  • Alnylam launched its first RNAi therapeutic in 2018 with the FDA approval of ONPATTRO (patisiran) for treatment of the polyneuropathy caused by hATTR amyloidosis, a progressive and life-threatening, rare, genetic disease.
  • The approval of Leqvio, a potentially transformational medicine for lowering LDL-C is a historic event for Alnylam and its RNAi therapeutics platform.

Intellia Therapeutics Announces Expansion of Ongoing Phase 1 Study of NTLA-2001 to Include Adults with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

Retrieved on: 
Monday, November 22, 2021
Health care, Cas9, Pharmacokinetics, Amyloidosis, CRISPR, Cardiomyopathy, Risk, GLOBE, COVID-19, Nobel Prize, Interim, Regeneron Pharmaceuticals, Nerve Damage, TTR, Liver, Private Securities Litigation Reform Act, Heart, Security (finance), Polyneuropathy, Cancer, IND, U.S. Securities and Exchange Commission, United Kingdom, Patient, Royal commission, NTLA, Amyloid, Degenerative disease, Protein, The New England Journal of Medicine, Population, MHRA, Clinical trial, Human, Intellia Therapeutics, Twitter, NASDAQ, ATTR, European Commission, Form 10-K, CTA, Therapy, Tissue, Serum, Heart failure, Safety, Annual report, Medicines and Healthcare products Regulatory Agency, III, Disease, Pharmaceutical industry, Vaccine

NTLA-2001 has received orphan drug designation for the treatment of ATTR amyloidosis by both the European Commission and the U.S. FDA.

Key Points: 
  • NTLA-2001 has received orphan drug designation for the treatment of ATTR amyloidosis by both the European Commission and the U.S. FDA.
  • ATTR amyloidosis is a chronic, fatal disease that can impact different organs and tissues within the body, often manifesting as either polyneuropathy or cardiomyopathy.
  • The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or cardiomyopathy (ATTR-CM).
  • There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis.

Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis

Retrieved on: 
Thursday, October 21, 2021
Safety, The New England Journal of Medicine, Diagnosis, Transthyretin, ATTR, Disease, Cancer, Annual report, Human, Twitter, Polyneuropathy, NTLA, Security (finance), U.S. Securities and Exchange Commission, Patient, Private Securities Litigation Reform Act, NASDAQ, FDA, Amyloidosis, Food, Interim, Cardiomyopathy, Risk, Degenerative disease, Nobel Prize, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, CRISPR, Nerve Damage, European Commission, Royal commission, Protein, Clinical trial, Tissue, COVID-19, Intellia Therapeutics, Regeneron Pharmaceuticals, IND, Form 10-K, TTR, Therapy, EC, GLOBE, Liver, Heart, Heart failure, Cas9, Pharmaceutical industry, Vaccine

This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.

Key Points: 
  • This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.
  • Orphan drug designation underscores the FDAs recognition of NTLA-2001s potential promise as a single-dose, novel therapy for the treatment of ATTR amyloidosis, said Intellia President and Chief Executive Officer John Leonard, M.D.
  • NTLA-2001 is currently being studied in a Phase 1 trial in adults with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
  • The decision by the FDA follows a March 2021 decision by the European Commission (EC) to also grant NTLA-2001 orphan drug designation for the treatment of ATTR amyloidosis.

Ionis to host ATTR symposium highlighting CARDIO-TTRansform Study at 3rd European ATTR Amyloidosis meeting

Retrieved on: 
Friday, September 3, 2021
Cardiology, Form 10-K, COVID-19, Congress, European, Annual report, Polyneuropathy, SEC, Degenerative disease, View, Company, Patients Association, Biotechnology, Therapy, Multimedia, IONS, Knowledge, EU, ATTR, Safety, Genetic testing, Judgement, Doctor, LICA, Goal, RNA, Efficacy, Amyloidosis, Development, NASDAQ, Disease, Pharmaceutical industry

CARLSBAD, Calif., Sept. 3, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today it will present 14 posters and two symposiums highlighting new scientific insights from its ATTR amyloidosis portfolioat the 3rd European ATTR Amyloidosis (EU-ATTR) meeting for Patients and Doctors on September 6-8, 2021.

Key Points: 
  • CARLSBAD, Calif., Sept. 3, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today it will present 14 posters and two symposiums highlighting new scientific insights from its ATTR amyloidosis portfolioat the 3rd European ATTR Amyloidosis (EU-ATTR) meeting for Patients and Doctors on September 6-8, 2021.
  • The presentations underscore Ionis' commitment to the ATTR amyloidosis community and the company's important work toward advancing treatment options for people impacted by the disease.
  • Satellite Symposium: Sotirios "Sam" Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis, is moderating a satellite symposium about ATTR-CM and investigational antisense medicine, eplontersen, for Ionis' CARDIO-TTRansform Study, on the Doctors Channel.
  • This press release includes forward-looking statements regarding Ionis' business, and the therapeutic and commercial potential of Ionis' technologies and products in development.

Alnylam Announces U.S. Food and Drug Administration Acceptance of New Drug Application for Investigational Vutrisiran for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis

Retrieved on: 
Thursday, June 24, 2021
Health, FDA, Genetics, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Amyloidosis, Branches of biology, Organ systems, Nervous system, Neurological disorders, Protein folding, Familial amyloid polyneuropathy, Orphan drug, Peripheral neuropathy, Polyneuropathy, Wild-type transthyretin amyloid, PTC Therapeutics, Vutrisiran, the HELIOS-A Phase 3 Study, hATTR Amyloidosis, RNAi, Alnylam Pharmaceuticals, VUTRISIRAN, THE HELIOS-A PHASE 3 STUDY, HATTR AMYLOIDOSIS, RNAI, ALNYLAM PHARMACEUTICALS

If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.

Key Points: 
  • If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.
  • Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis.
  • Vutrisiran has also been granted a Fast-Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
  • Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis.

Virtis Health Expands Ambulatory Infusion Center Footprint and Infusion Management Services

Retrieved on: 
Monday, June 14, 2021
Other Health, Radiology, Pharmaceutical, Insurance, Medical devices, Infectious diseases, Hospitals, Professional services, Practice Management, Physical therapy, Nursing, Managed care, Cardiology, General Health, Biotechnology, Diabetes, Surgery, AIDS, Health, Medical specialties, Autoimmune diseases, Clinical medicine, Medicine, Neurological disorders, Chronic inflammatory demyelinating polyneuropathy, Autoimmunity, Polyneuropathy, Myasthenia gravis, Transfusion medicine

Concurrently, the Company continues to expand its comprehensive infusion management and related physician practice management services nationwide.

Key Points: 
  • Concurrently, the Company continues to expand its comprehensive infusion management and related physician practice management services nationwide.
  • Virtis Health offers a range of therapy management services and treatments for patients with autoimmune disorders, including chronic inflammatory demyelinating polyneuropathy (CIDP), multiple sclerosis and myasthenia gravis.
  • Virtis Healths Physician Services division provides comprehensive infusion center and practice management services to support physician and provider partners.
  • Additionally, the Company provides comprehensive infusion center and related practice management services to physicians, health systems and other medical entities nationwide.

argenx Announces “GO” Decision in ADHERE Trial of Efgartigimod in Chronic Inflammatory Demyelinating Polyneuropathy Following Interim Analysis

Retrieved on: 
Monday, February 1, 2021
Branches of biology, Neurological disorders, Nervous system, Organ systems, Autoimmune diseases, Chronic inflammatory demyelinating polyneuropathy, Polyneuropathy, Peripheral neuropathy, Autoimmunity, Myelin, Autoimmune disease, Neuritis

The ADHERE trial is a randomized, withdrawal study evaluating 1000mg weekly subcutaneous (SC) efgartigimod for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).

Key Points: 
  • The ADHERE trial is a randomized, withdrawal study evaluating 1000mg weekly subcutaneous (SC) efgartigimod for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).
  • The trial consists of an open-label Stage A followed by a randomized, placebo-controlled Stage B with a planned interim responder analysis after the first 30 patients enroll in Stage A.
  • Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system.
  • argenx is evaluating efgartigimod in multiple serious autoimmune diseases, and cusatuzumab in hematological cancers in collaboration with Janssen.

Global Chronic Inflammatory Demyelinating Polyneuropathy Pipeline Report 2020: Therapeutic Analysis of 8 Companies, 4 Drug Profiles & Dormant Projects

Retrieved on: 
Friday, October 16, 2020
Branches of biology, Neuroscience, Neurological disorders, Organ systems, Autoimmune diseases, Chronic inflammatory demyelinating polyneuropathy, Polyneuropathy, Peripheral neuropathy, Myelin, Neuritis

Chronic Inflammatory Demyelinating Polyneuropathy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.

Key Points: 
  • Chronic Inflammatory Demyelinating Polyneuropathy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • The Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System) pipeline guide also reviews the key players involved in therapeutic development for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and features dormant and discontinued projects.
  • The pipeline guide reviews pipeline therapeutics for Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • Formulate corrective measures for pipeline projects by understanding Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System) pipeline depth and focus of Indication therapeutics.

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Pipeline Review, H2 2020 - ResearchAndMarkets.com

Retrieved on: 
Thursday, October 8, 2020
General Health, Pharmaceutical, Health, Other Health, Branches of biology, Neuroscience, Neurological disorders, Organ systems, Autoimmune diseases, Chronic inflammatory demyelinating polyneuropathy, Polyneuropathy, Peripheral neuropathy, Myelin, Neuritis

The "Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) - Pipeline Review, H2 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) - Pipeline Review, H2 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • Chronic Inflammatory Demyelinating Polyneuropathy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • The Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System) pipeline guide also reviews the key players involved in therapeutic development for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and features dormant and discontinued projects.
  • Formulate corrective measures for pipeline projects by understanding Chronic Inflammatory Demyelinating Polyneuropathy (Central Nervous System) pipeline depth and focus of Indication therapeutics.