Emapalumab

Annals of Rheumatic Diseases Publishes Results from Phase 2 Study of emapalumab in Patients with Secondary HLH/Macrophage Activation Syndrome

Retrieved on: 
Tuesday, April 4, 2023
Medical Affairs Bureau, Research and development, Swedish Orphan Biovitrum, Pediatrics, Glucocorticoid, Emapalumab, Macrophage, Annals of the Rheumatic Diseases, HLH, MAS, Cincinnati Children's Hospital Medical Center, AOSD, Tocilizumab, Development, Annals, Patient, Division, Bambino, Hospital, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Laboratory, Food, Safety, Disease, Pharmaceutical industry, Rheumatology

In the published study, 14 patients with sJIA or AOSD and sHLH/MAS who did not respond to high-dose glucocorticosteroids received emapalumab.

Key Points: 
  • In the published study, 14 patients with sJIA or AOSD and sHLH/MAS who did not respond to high-dose glucocorticosteroids received emapalumab.
  • All 14 patients completed the trial, entered long-term follow-up and were alive at the end of follow-up.
  • Based on the results of this study, Sobi decided to continue to evaluate emapalumab in this patient population and initiated the EMERALD phase 3 study, which is ongoing.
  • “We remain committed to evaluating emapalumab as a potential new treatment option for patients affected by this severe condition.

Sobi to Present Data from REAL-HLH Study at the ACR Convergence 2022 Meeting

Retrieved on: 
Thursday, October 27, 2022
Yee, Severe cutaneous adverse reactions, Infection, American College, ACR, Virus, Priority, Rash, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Death, Life, Hemophagocytic lymphohistiocytosis, Tachycardia, Risk, Patient, Bacteria, Food, Vaccine, Shingles, Hypokalemia, MPH, TB, Irritability, Lymphocytosis, ODD, CMV, Rheumatology, Abdominal pain, Diarrhea, Fungus, Swedish Orphan Biovitrum, Adult, GLOBE, Full, FDA, Degenerative disease, Hematopoietic stem cell transplantation, Tuberculosis, Histoplasmosis, Cough, Constipation, Hypertension, HLH, Pharmaceutical industry, Medical device, Dentistry, MD, Fever, Tachypnea, Emapalumab

Since its approval, no study has evaluated the use of Gamifant in a larger cohort of patients in the real-world clinical setting.

Key Points: 
  • Since its approval, no study has evaluated the use of Gamifant in a larger cohort of patients in the real-world clinical setting.
  • Currently, Gamifant is not approved by the FDA for the treatment of patients with secondary HLH.
  • The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899).
  • To report suspected adverse reactions, contact Sobi North America at 1-866-773-5274 or FDA at 1-800-FDA-1088.

Results From Pivotal Phase 2/3 Study of Emapalumab in Patients With Primary HLH Published in New England Journal of Medicine

Retrieved on: 
Thursday, May 7, 2020
Health, Drugs, Hemophagocytic lymphohistiocytosis, Emapalumab, HLH, Clinical trial, Swedish Orphan Biovitrum, Histiocytosis

STOCKHOLM, May 7, 2020 /PRNewswire/ -- Sobi announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020.

Key Points: 
  • STOCKHOLM, May 7, 2020 /PRNewswire/ -- Sobi announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020.
  • Emapalumab is the first therapy approved by the US Food & Drug Administration (FDA) for primary HLH and is under review by the European Medicines Agency (EMA).
  • "The results further advance our understanding of primary HLH and the role of interferon gamma in its pathogenesis.
  • This pivotal clinical study is the first study in primary HLH to prospectively assess and report treatment responses using predefined comprehensive objective clinical and laboratory criteria.

Data to be Highlighted at the American Society of Hematology 2019 Annual Meeting

Retrieved on: 
Thursday, December 5, 2019
Health, Drugs, Breakthrough therapy, Monoclonal antibodies, Hemophagocytic lymphohistiocytosis, Histiocytosis, Emapalumab, Food and Drug Administration, Pediatric Trials Network, Phases of clinical research

The adaptive design of a study evaluating the efficacy, safety and pharmacokinetics of emapalumab in adult patients with secondary HLH will be presented.

Key Points: 
  • The adaptive design of a study evaluating the efficacy, safety and pharmacokinetics of emapalumab in adult patients with secondary HLH will be presented.
  • The initial phase will enrol 10 adult patients and the study is expected to open later this year.
  • The latest clinical data and ongoing trials with the potential to impact the care of patients with HLH will also be reviewed.
  • The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899).

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.