Eculizumab

Onco360 Has Been Selected as the Sole National Specialty Pharmacy Partner for Voydeya® (danicopan)

Retrieved on: 
Monday, April 8, 2024
Immune system, PNH, Specialty pharmacy, Patient, Ravulizumab, FDA, Eculizumab, Paroxysmal nocturnal hemoglobinuria, EVH, Blood, Alexion Pharmaceuticals

LOUISVILLE, Ky., April 08, 2024 (GLOBE NEWSWIRE) -- Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a national pharmacy partner by Alexion Pharmaceuticals for Voydeya® (Danicopan) as a first class oral, Factor D inhibitor.

Key Points: 
  • LOUISVILLE, Ky., April 08, 2024 (GLOBE NEWSWIRE) -- Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a national pharmacy partner by Alexion Pharmaceuticals for Voydeya® (Danicopan) as a first class oral, Factor D inhibitor.
  • It is FDA-approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) in combination with C5 inhibitor, Ultomiris® (Ravulizumab) or Soliris® (Eculizumab) for patients that experience clinically significant extravascular hemolysis (EVH).1 Voydeya® is an oral, Factor D inhibitor that acts in the alternative complement pathway of the immune system, providing control of red blood cell destruction within and outside the blood vessels.
  • “Onco360 appreciates the opportunity to partner further with the team at Alexion and become the sole national specialty pharmacy provider for Voydeya®”, said Benito Fernandez, Chief Commercial Officer.
  • “We are proud to add this first in class oral Factor D inhibitor treatment option for PNH patients.”

VOYDEYA™ approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular hemolysis in adults with the rare disease PNH

Retrieved on: 
Monday, April 1, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Factor D, Arthralgia, Chest pain, Confusion, Neuropsychological test, Infection, Eculizumab, Risk, Headache, ALPHA, PNH, C5, Patient, Orphan, Mitigation, Vaccine, Light, Paroxysmal nocturnal hemoglobinuria, EVH, Bacteria, University, Chills, MD, Breakthrough therapy, Cough, European Medicines Agency, Fatigue, Nausea, Rash, Fever, Priority, Safety, Immune system, REMS, Division, Vomiting, Hematology, Patient safety, Medicine

VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.

Key Points: 
  • VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.
  • You must complete or update meningococcal vaccine(s) and streptococcus vaccine(s) at least 2 weeks before your first dose of VOYDEYA.
  • If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting VOYDEYA.
  • Carry it with you at all times during treatment and for 1 week after your last VOYDEYA dose.

EMPAVELI® (pegcetacoplan) Provided Long-Term Control of PNH in New Data Presented at ASH Annual Meeting

Retrieved on: 
Monday, December 11, 2023
PEGASUS, Risk, PNH, Eculizumab, Disease, APLS, LDH, Trial of the century, Society, Standard of care, Head, Duke University, ASH, Infection, PRINCE, Patient, Safety, Bacteria, Medicine, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting.

Key Points: 
  • The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting.
  • After starting treatment with EMPAVELI, key markers of disease rapidly improved and were sustained in both treatment-naïve patients and patients previously treated with eculizumab.
  • Improvements in hemoglobin reached normal or near-normal levels, and mean lactate dehydrogenase (LDH) was maintained below the upper limit of normal.
  • “By achieving and maintaining normal clinical measures over time, EMPAVELI has the potential to elevate the standard of care for all adults with PNH."

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.

BioCryst R&D Day Highlights New Diversified Pipeline of First-in-Class/Best-in-Class Therapies with Five Programs Expected in Clinical Development in Next 24 Months

Retrieved on: 
Friday, November 3, 2023
GMG, BioCryst Pharmaceuticals, FDA, Research, Patient, Drug design, IgA nephropathy, Plasma, CAD, Lupus nephritis, KLK5, Genetic disorder, RESEARCH, C2, DME, Eculizumab, Infection, Injection, Skin, Diabetes, Autoantibody, Ryan Murphy, Edema, Netherton, R, Autoimmune hemolytic anemia, HAE, Kallikrein, Growth, Disease, Dehydration, Muscle weakness, Immune system, Hemolysis, Solubility, Netherton syndrome, Clinical trial, Paratriathlon, Eye, Ravulizumab, Cold agglutinin disease, PARK, IP, SPINK5, Fine chemical, Pharmaceutical industry, Vaccine

A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .

Key Points: 
  • A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .
  • The team is excited to share our approach, and our expanded pipeline of new programs, at our R&D Day,” said Jon Stonehouse, president and chief executive officer of BioCryst.
  • The disease is caused by the deficiency of a natural inhibitor (SPINK5) of KLK5, a serine protease responsible for regulating skin shedding.
  • Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections.

FDA Accepts Application for Genentech’s Crovalimab for the Treatment of PNH, a Rare Life-Threatening Blood Condition

Retrieved on: 
Wednesday, September 6, 2023
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Eculizumab, Injection, AE, Paroxysmal nocturnal hemoglobinuria, SC, European Hematology Association, ROG, RO, Fatigue, Anemia, PNH, Phase, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, BLA, Food, Kidney disease, C5, Roche, Pharmaceutical industry, Vaccine, Genentech

The acceptance was based on results from the pivotal Phase III COMMODORE 2 study, which demonstrated that in people with PNH, crovalimab achieved disease control and was well-tolerated.

Key Points: 
  • The acceptance was based on results from the pivotal Phase III COMMODORE 2 study, which demonstrated that in people with PNH, crovalimab achieved disease control and was well-tolerated.
  • Results from the Phase III COMMODORE 1 study, demonstrating the consistent benefit-risk profile of crovalimab, also supported the application.
  • “Crovalimab could provide an option to self-administer as infrequently as every four weeks, thereby reducing clinic visits for people with this lifelong condition.”
    PNH is a rare and life-threatening blood condition, which affects approximately 20,000 people worldwide.
  • In PNH, red blood cells are destroyed by the complement system – part of the innate immune system.

AstraZeneca urges re-examination of unintended consequences of Inflation Reduction Act on American cancer and rare disease patients

Retrieved on: 
Friday, August 25, 2023
Other Health, Research, General Health, Professional Services, Pharmaceutical, Oncology, Healthcare Reform, Science, Legal, Congressional News, Views, Health, Public Policy, Government, Orphan Drug Act of 1983, Eculizumab, REMS, Central nervous system, Drug development, Vaccination, Prostate cancer, GMG, Infection, Attenuated vaccine, Fever, JAMA, Cancer, Light, Neuromyelitis optica spectrum disorder, Breast, Medicine, NMOSD, IRA, AstraZeneca, Rash, American Medical Association, Neisseria meningitidis, Vomiting, Inflation, ODA, Low-probability-of-intercept radar, Meningococcal vaccine, Patient, Risk, Autoimmune disease, PNH, Immune system, Nausea, Confusion, Inflation Reduction Act of 2022, Rare, Headache, Patient safety, Death, Disease, Pharmaceutical industry, Vaccine

To help protect access to medicines for cancer and rare disease patients, AstraZeneca today has filed a legal challenge to critical aspects of the drug price negotiation provisions of the Inflation Reduction Act (IRA).

Key Points: 
  • To help protect access to medicines for cancer and rare disease patients, AstraZeneca today has filed a legal challenge to critical aspects of the drug price negotiation provisions of the Inflation Reduction Act (IRA).
  • Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Rare disease and cancer patients depend upon high-risk, low-probability drug development that takes many years to develop and aims for cure.
  • One example is LYNPARZA® (olaparib), a small-molecule cancer medicine approved in 2014 in the US for a small group of late-line ovarian cancer patients.
  • Additional trials added small groups of breast and pancreatic cancer patients, with the most recent indication in prostate cancer approved just this year – nine years later.

What’s on Your Must-Read List? If You Work in Pharma, Add Spherix Global Insights’ Generalized Myasthenia Gravis Patient Chart Audit to that List

Retrieved on: 
Thursday, August 10, 2023
Cholinesterase, GMG, Efgartigimod alfa, Myasthenia gravis, Neurology, Genentech, UCB, Patient, Physician, Ocular myasthenia, Diagnosis, Neonatal Fc receptor, Horizon, Cholinesterase inhibitor, Pharmaceutical industry, Eculizumab, Ravulizumab

US-based neurologists completed this HIPAA compliant, online chart audit survey among 607 patients with gMG using information exclusively found in their practice’s electronic medical records.

Key Points: 
  • US-based neurologists completed this HIPAA compliant, online chart audit survey among 607 patients with gMG using information exclusively found in their practice’s electronic medical records.
  • Spherix’s sweeping research paints a picture of the gMG treatment journey and the patients who are coping with this devastating neuromuscular condition.
  • The RealWorld Dynamix chart audit study was completed by nearly 150 neurologists and neuromuscular specialists across all regions of the US.
  • Patient charts were submitted by general neurologists seeing as few as 10-20 gMG patients and specialists managing more than 100 gMG patients.

Muscular Dystrophy Association Celebrates FDA Approval of UCB’s RYSTIGGO for the Treatment of Generalized Myasthenia Gravis

Retrieved on: 
Tuesday, June 27, 2023
Communication, US Food Sovereignty Alliance, Disease, MuSK protein, Doctor of Philosophy, Thymectomy, GMG, Efgartigimod alfa, Fc receptor, Antibody, Immunoglobulin G, Enzyme inhibitor, Ravulizumab, UCB, Patient, MG, Muscular Dystrophy Association, Acetylcholine receptor, Neonatal Fc receptor, MDA, FDA, Pharmaceutical industry, Vaccine, Eculizumab

New York, June 27, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of RYSTIGGO (rozanolixizumab-noli) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

Key Points: 
  • New York, June 27, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of RYSTIGGO (rozanolixizumab-noli) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
  • RYSTIGGO is the only FDA-approved treatment in adults for both anti-AChR and anti-MuSK antibody-positive gMG, the two most common subtypes of gMG, and will be administered as an injection for subcutaneous infusion.
  • Although treatment with RYSTIGGO will not cure gMG, it could lead to functional improvements that improve the daily life of individuals with gMG.
  • “Rystiggo is a welcome addition to the targeted therapeutic options available for the treatment of generalized myasthenia gravis,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA.

Muscular Dystrophy Association Celebrates FDA Approval of argenx’s Vyvgart Hytrulo Injection for Treatment of Generalized Myasthenia Gravis

Retrieved on: 
Wednesday, June 21, 2023
Communication, US Food Sovereignty Alliance, Disease, Travel, University, IV, Doctor of Philosophy, Public policy, Thymectomy, GMG, Myasthenia gravis, Quality of life, Research, Antibody, Enzyme inhibitor, MG, MD, UCSF, Patient, Muscular Dystrophy Association, Immunoglobulin G, Acetylcholine receptor, Hospital, Neonatal Fc receptor, MDA, FDA, Pharmaceutical industry, Eculizumab, Efgartigimod alfa

New York, June 20, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of subcutaneous (SC) Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc),for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

Key Points: 
  • New York, June 20, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of subcutaneous (SC) Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc),for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.
  • Vyvgart Hytrulo is the first FDA-approved SC injectable for gMG and will be made available and marketed in the United States by argenx.
  • The new SC formulation can now be administered as a single injection (1,008 mg fixed dose) at home or in a physician’s office.
  • Although treatment with Vyvgart will not cure gMG, it could lead to functional improvements that improve the daily life of individuals with gMG.