Histiocytosis

New Data Demonstrates Impact of Emapalumab in Patients With Macrophage Activation Syndrome (MAS)

Retrieved on: 
Tuesday, June 18, 2019
Branches of biology, Medical specialties, Immunology, Antivirals, Cytokines, Histiocytosis, Hemophagocytic lymphohistiocytosis, Emapalumab, Interferon, Macrophage

The study showed that treatment with emapalumab led to rapid neutralisation of interferon gamma (IFN) and a complete response in all patients who participated in the study.

Key Points: 
  • The study showed that treatment with emapalumab led to rapid neutralisation of interferon gamma (IFN) and a complete response in all patients who participated in the study.
  • Emapalumab has previously been shown to induce rapid and sustained responses in patients with primary HLH.
  • Emapalumab is FDA-approved for paediatric (newborn and older) and adult patients with primary HLH with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.
  • It is classified as a secondary form of HLH and is caused by excessive activation and expansion of T cells and macrophages.

Video Alert: New Video Illustrates Mechanism of Action for Gamifant® (emapalumab-lzsg)

Retrieved on: 
Monday, June 3, 2019
Health, Biotechnology, Pharmaceutical, Other Health, Research, Science, Viruses, Medical specialties, Microbiology, Viral diseases, RTT, Infectious causes of cancer, Histiocytosis, Betaherpesvirinae, Hemophagocytic lymphohistiocytosis, Epstein–Barr virus, Cytomegalovirus, Vaccine

Gamifant represents a new approach to helping critically ill primary HLH patients reach haematopoietic stem cell transplant.

Key Points: 
  • Gamifant represents a new approach to helping critically ill primary HLH patients reach haematopoietic stem cell transplant.
  • During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated.
  • Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.
  • Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least four weeks after the last dose of Gamifant.

Sobi and Novimmune Selected as Honourees for 2019 National Organization for Rare Disorders (NORD) Rare Impact Award in Industry Innovation for Gamifant® (emapalumab)

Retrieved on: 
Monday, March 25, 2019
Hemophagocytic lymphohistiocytosis, Histiocytosis, Rare disease, Swedish Orphan Biovitrum, Nord, National Organization for Rare Disorders, Clavia, Medical specialties, Health, Companies

STOCKHOLM, March 25, 2019 /PRNewswire/ -- Sobi and Novimmune SA have both been selected as honourees by the National Organization for Rare Disorders (NORD) for the 2019 Rare Impact Awards.

Key Points: 
  • STOCKHOLM, March 25, 2019 /PRNewswire/ -- Sobi and Novimmune SA have both been selected as honourees by the National Organization for Rare Disorders (NORD) for the 2019 Rare Impact Awards.
  • The Rare Impact Awards recognise individuals, organisations and industry innovators for outstanding work in support of the rare disease community.
  • The 2019 Rare Impact Awards ceremony will take place on Saturday, 22 June, as part of NORD's 2019 Living Rare, Living Stronger NORD Patient and Family Forum in Houston.
  • "We are honoured to be recognised by NORD for the work we have done to bring Gamifant to primary HLH patients.

FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease

Retrieved on: 
Tuesday, November 20, 2018
Immunology, Medicine, Hemophagocytic lymphohistiocytosis, Histiocytosis, Health, Cytokine release syndrome, Immunosuppressants, Food and Drug Administration, Monoclonal antibodies

This FDA approval is the first for a drug specifically for HLH.

Key Points: 
  • This FDA approval is the first for a drug specifically for HLH.
  • "We are committed to continuing to expedite the development and review of therapies that offer meaningful treatment options for patients with rare conditions."
  • HLH is a condition in which the body's immune cells do not work properly.
  • Common side effects reported by patients receiving Gamifant in clinical trials included infections, hypertension, infusion-related reactions, low potassium and fever.

FDA Approves Gamifant® (emapalumab), the First and Only Treatment for Primary Haemophagocytic Lymphohistiocytosis (HLH)

Retrieved on: 
Tuesday, November 20, 2018
Medicine, Clinical medicine, Health, Cancer treatments, Antineoplastic drugs, Hemophagocytic lymphohistiocytosis, Histiocytosis, Oncology, Food and Drug Administration, Chemotherapy

Gamifant represents a major advance in the treatment of these patients through a targeted mode of action.

Key Points: 
  • Gamifant represents a major advance in the treatment of these patients through a targeted mode of action.
  • The FDA approval is based on data from the pivotal phase 2/3 study which enrolled patients with primary HLH.
  • The approval of Gamifant as the first treatment for primary HLH is a significant step in our journey of helping these patients."
  • The current conventional treatment prior to transplant includes steroids and chemotherapy and are not specifically approved to treat primary HLH.