Efgartigimod alfa

argenx Announces Approval of VYVGART (efgartigimod alfa) in Japan for Adults with Primary Immune Thrombocytopenia

Retrieved on: 
Tuesday, March 26, 2024
Diagnosis, Blood pressure, Efgartigimod alfa, System, Urination, Urinary tract infection, MHLW, Chest pain, Rituximab, Allergy, Disease, Infection, Risk, Headache, Patient, Sore throat, IWG, History, ITP, Chills, Quality of life, Cough, Euronext, Rash, Respiratory tract infection, Splenectomy, Medicine, Skin, Fever, Antibody, Wheeze, Back pain, Shivering, Safety, Pain, Fatigue, Phlegm, Swelling, Food, Autoimmune disease, Nursing

“argenx is on a mission to deliver transformative medicines for people living with severe autoimmune disease,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “argenx is on a mission to deliver transformative medicines for people living with severe autoimmune disease,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • By reducing circulating autoantibodies, VYVGART is uniquely designed to serve as a precision intervention that targets the underlying disease biology of ITP.
  • ADVANCE successfully met its primary endpoint, demonstrating that a higher proportion of chronic ITP patients receiving VYVGART achieved a sustained platelet count response compared to placebo.
  • Do not use VYVGART if you have a serious allergy to efgartigimod alfa or any of the other ingredients in VYVGART.

argenx Reports Full Year 2023 Financial Results and Provides Fourth Quarter Business Update

Retrieved on: 
Thursday, February 29, 2024
DGF, Neonatal Fc receptor, ALS, Congenital myasthenic syndrome, CMS, CIDP, MMN, Alfa Romeo, IIP, IL-6, FDA, Inflammation, MHLW, PDUFA, Euronext, Neurology, MuSK protein, Kidney, ARDA, Health care, C2, GMG, Ministry of Health, Dermatomyositis, Patient, Efgartigimod alfa, MHRA, Medicine, Multifocal motor neuropathy, Immune system, Pharmaceutical industry

“argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • Clinically, we generated significant data through multiple study readouts, achieving key milestones for both the CIDP and MMN patient communities and importantly advancing our second molecule, empasiprubart.
  • Looking forward to 2024, we will act with a continued sense of purpose to expand our patient reach.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, PKD, Personalized medicine, EMA, Research, MAA, Investment, Marketing, Food, Arrhythmogenic cardiomyopathy, Circulation, BLA, PDUFA, Fanconi anemia, Conference, Safety, DCM, European Medicines Agency, Food and Drug Administration, Muscle weakness, Efgartigimod alfa, Pyruvate kinase deficiency, Patient, Disorder, Clinical trial, Medication, ACM, IND, AAV, CGMP, CMC, Priority review, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

argenx Announces Approval of VYVDURA® (efgartigimod alfa and hyaluronidase-qvfc) Injection for Subcutaneous Use in Japan for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, January 18, 2024
Patient, Euronext, Steroid, Immunoglobulin G, GMG, MHLW, Autoimmunity, Efgartigimod alfa, Pharmaceutical industry

Following this decision, VYVGART is now approved in Japan for both intravenous (IV) and self-administered SC use.

Key Points: 
  • Following this decision, VYVGART is now approved in Japan for both intravenous (IV) and self-administered SC use.
  • “Today’s approval of VYVDURA marks a significant milestone for the gMG community in Japan and furthers our commitment to deliver innovative treatments to autoimmune patients globally,” said Hermann Strenger, General Manager, argenx Japan.
  • ADAPT-SC established the efficacy of VYVDURA by demonstrating a reduction in percent change from baseline in total immunoglobulin G (IgG) levels comparable to VYVGART IV in adult gMG patients.
  • ADAPT-SC was a bridging study to the Phase 3 ADAPT study, which formed the basis for approval of VYVGART in Japan in January 2022.

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
Monday, January 8, 2024
Immunoglobulin therapy, GMG, FDA, PRV, Congenital myasthenic syndrome, ARDA, Research Councils UK, Euronext, Inflammation, Cohort, PGIC, NRDL, Autoimmunity, Efgartigimod alfa, Dermatomyositis, Conference, MuSK protein, IND, CIDP, Pharmacokinetics, MMN, Safety, Therapy, Patient, ALS, Immune system, IL-6, Medicine, International Myeloma Foundation, IIP, FSS, Neonatal Fc receptor, Multifocal motor neuropathy, Growth, Pharmaceutical industry

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.

argenx Reports Topline Results from ADDRESS Study of Efgartigimod SC in Pemphigus

Retrieved on: 
Wednesday, December 20, 2023
Biology, Patient, Efgartigimod alfa, Autoantibody, PV, Consistency, Literature, Pemphigus vulgaris, Conference call, Euronext, PD, Steroid, Pemphigus foliaceus, Growth, Safety, Immunoglobulin G

argenx will not pursue additional development in pemphigus and plans to prioritize clinical development of efgartigimod in its ongoing severe autoimmune indications.

Key Points: 
  • argenx will not pursue additional development in pemphigus and plans to prioritize clinical development of efgartigimod in its ongoing severe autoimmune indications.
  • “We are disappointed by today’s results, particularly for pemphigus patients who have seen little innovation in this treatment space,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer at argenx.
  • “We are grateful to the pemphigus community and all involved in the ADDRESS study, including patients, healthcare professionals, and our argenx teams,” continued Dr. Truyen.
  • Consistent pharmacodynamic (PD) effect of efgartigimod SC: Treatment with efgartigimod SC led to total immunoglobulin G (IgG) and desmoglein autoantibody (DSG-1 and DSG-3) reductions up to 75%.

argenx Announces European Commission Approval of Subcutaneous VYVGART® (efgartigimod alfa) for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, November 16, 2023
Acetylcholine receptor, European Commission, GMG, Civil service commission, Patient, Autoimmune disease, CHMP, Committee, Efgartigimod alfa, Euronext, SE

Following this decision, VYVGART is now approved in Europe for both IV and self-administered SC use.

Key Points: 
  • Following this decision, VYVGART is now approved in Europe for both IV and self-administered SC use.
  • The approval is applicable to all 27 European Union (EU) Member States plus Iceland, Norway and Liechtenstein.
  • argenx will work with local health authorities to secure patient access for VYVGART SC in the region.
  • We are proud to deliver this second formulation to the European gMG community, just 15 months after the initial approval of VYVGART IV,” said Anant Murthy, General Manager of argenx EMEA.

Rocket Pharmaceuticals Appoints R. Keith Woods to Board of Directors

Retrieved on: 
Tuesday, December 12, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Efgartigimod alfa, Senior, Disorder, Florida State University, Hematology, Patient, Eisai, Alexion Pharmaceuticals, Marketing, FA, Muscle weakness, Roche, R, Fanconi anemia, Medication, Amgen, Pharmaceutical industry, Management, Eculizumab

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.
  • Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain.
  • “I’m thrilled to welcome Keith to our Board of Directors, who brings deep industry expertise and invaluable experience to the Rocket Board as we build upon our commercial and operational infrastructure to support our anticipated first product launches for severe Leukocyte Adhesion Deficiency (LAD-I) and Fanconi Anemia (FA),” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Earlier in his career, Mr. Woods held commercial and sales positions of increasing responsibility at Roche, Amgen and Eisai.

Halozyme Announces argenx Receives European Commission Approval of VYVGART® SC with ENHANZE® for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, November 16, 2023
Acetylcholine receptor, Food, European Commission, Immunoglobulin G, GMG, Patient, CHMP, Committee, Efgartigimod alfa, Pharmaceutical industry, Vaccine, Halozyme

SAN DIEGO, Nov. 16, 2023 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") today announced that argenx received European Commission (EC) approval of VYVGART® SC (efgartigimod alfa and hyaluronidase-qvfc) co-formulated with ENHANZE® for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. The European approval of VYVGART SC provides the option for patient self-administration. The EC approval is applicable to all 27 European Union Member States plus Iceland, Norway and Liechtenstein.

Key Points: 
  • The European approval of VYVGART SC provides the option for patient self-administration.
  • The EC approval is applicable to all 27 European Union Member States plus Iceland, Norway and Liechtenstein.
  • VYVGART® SC was approved by the U.S. Food and Drug Administration in June 2023 and is marketed as VYVGART® Hytrulo.
  • ADAPT-SC established the efficacy of VYVGART® SC by demonstrating a reduction in anti-AChR antibody levels comparable to VYVGART® IV in adult gMG patients.

argenx Highlights Data Evaluating VYVGART in Neuromuscular Autoimmune Disease at AANEM and MGFA Scientific Sessions

Retrieved on: 
Wednesday, November 1, 2023
Hives, Headache, Incidence, Alfa Romeo, Erythema, Vaccine, Immunization, Itch, Respiratory tract infection, Infection, Quality of life, Periodic breathing, Bruise, Milk, Infant, Rash, Vaccination, Acetylcholine receptor, Antibody, Risk, Immunoglobulin G, Angioedema, Urinary tract infection, Efgartigimod alfa, Safety, Pain, Pharmaceutical industry, Health care, Birth control, Patient, Life, Tolerability, Neurology

Delay the administration of VYVGART or VYVGART HYTRULO in patients with an active infection until the infection has resolved; monitor for clinical signs and symptoms of infections.

Key Points: 
  • Delay the administration of VYVGART or VYVGART HYTRULO in patients with an active infection until the infection has resolved; monitor for clinical signs and symptoms of infections.
  • If serious infection occurs, administer appropriate treatment and consider withholding treatment with VYVGART or VYVGART HYTRULO until the infection has resolved.
  • Because VYVGART and VYVGART HYTRULO cause a reduction in immunoglobulin G (IgG) levels, vaccination with live-attenuated or live vaccines is not recommended during treatment with VYVGART or VYVGART HYTRULO.
  • If a hypersensitivity reaction occurs during VYVGART or VYVGART HYTRULO administration, discontinue use and institute appropriate supportive measures if needed.