Hemophagocytic lymphohistiocytosis

Emapalumab's efficacy confirmed by sensitivity analysis presented at ESID2020

Saturday, October 17, 2020 - 1:33pm

"We remain committed to making emapalumab accessible to patients affected by this severe condition around the world."

Key Points: 
  • "We remain committed to making emapalumab accessible to patients affected by this severe condition around the world."
  • A high unmet medical need exists among these patients despite today's standard-of-care therapy, glucocorticoids and etoposide, with or without cyclosporine.
  • The pivotal study used clinically objective response criteria to define the primary endpoint of overall response rate to emapalumab in primary HLH.
  • The sensitivity analysis supports the use of the clinically objective ORR as a primary endpoint in studies of primary HLH.

Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH)

Monday, August 24, 2020 - 3:44pm

Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3).

Key Points: 
  • Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3).
  • "We are excited to announce this expansion of our gene and cell therapy pipeline beyond our lead stem cell lentiviral gene therapy candidate for hemophilia A that is entering Phase 1 clinical testing.
  • Proof of concept for stem cell lentiviral gene therapy of FHL3 was demonstrated using primary patient cells and a genetic mouse model of FHL3.
  • Third, with stem cell-based lentiviral gene therapy there will be no wait time to find a sufficiently human leukocyte antigen-matched donor," said Trent Spencer, Ph.D., President of Expression Therapeutics.

Hemophagocytic Lymphohistiocytosis (HLH) Markets, 2017-2030 - Epidemiology, Drugs, Pipeline, Reimbursement Scenario, Competitive Intelligence

Wednesday, July 29, 2020 - 12:45pm

The Hemophagocytic Lymphohistiocytosis epidemiology chapters provide insights about historical and current Hemophagocytic Lymphohistiocytosis patient pool and forecasted trend for every seven major countries.

Key Points: 
  • The Hemophagocytic Lymphohistiocytosis epidemiology chapters provide insights about historical and current Hemophagocytic Lymphohistiocytosis patient pool and forecasted trend for every seven major countries.
  • Hemophagocytic Lymphohistiocytosis epidemiology is segmented by Incidence of Hemophagocytic Lymphohistiocytosis (Familial and secondary HLH), Familial HLH cases by Mutation Types and Secondary HLH cases by Etiologies.
  • Drug chapter segment of the Hemophagocytic Lymphohistiocytosis report encloses the detailed analysis of Hemophagocytic Lymphohistiocytosis pipeline drug.
  • The report covers the detailed information of Hemophagocytic Lymphohistiocytosis collaborations, acquisition and merger, licensing, patent details and other information for Hemophagocytic Lymphohistiocytosis emerging therapies.

Hemophagocytic Lymphohistiocytosis Market Insights, Epidemiology, and Forecast to 2030 - ResearchAndMarkets.com

Tuesday, July 28, 2020 - 2:33pm

The "Hemophagocytic Lymphohistiocytosis Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Hemophagocytic Lymphohistiocytosis Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The Hemophagocytic Lymphohistiocytosis epidemiology chapters provide insights about historical and current Hemophagocytic Lymphohistiocytosis patient pool and forecasted trend for every seven major countries.
  • Hemophagocytic Lymphohistiocytosis epidemiology is segmented by Incidence of Hemophagocytic Lymphohistiocytosis (Familial and secondary HLH), Familial HLH cases by Mutation Types and Secondary HLH cases by Etiologies.
  • The Drug chapter segment of the Hemophagocytic Lymphohistiocytosis report encloses the detailed analysis of Hemophagocytic Lymphohistiocytosis pipeline drug.

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

Thursday, May 7, 2020 - 6:20am

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.

AB2 Bio Appoints Michael Soldan CEO

Tuesday, March 3, 2020 - 6:00am

Prior to Fresenius Kabi, Dr. Soldan was with Merck Group, where he spent two years heading the biosimilars business.

Key Points: 
  • Prior to Fresenius Kabi, Dr. Soldan was with Merck Group, where he spent two years heading the biosimilars business.
  • I am very pleased to welcome Michael Soldan to AB2 Bio, where he will bring the benefit of his impressive and wide-ranging career in the pharmaceuticals industry.
  • AB2 Bio is conducting a pivotal Phase 3 clinical trial of Tadekinigalfa in primary, interleukin-18 driven Hemophagocytic Lymphohistiocytosis (HLH) patientswith NLRC4 mutation or XIAP deficiency.
  • AB2 Bio Ltd ( http://www.ab2bio.com/ ) is a private advanced clinical-stage biotech company located in the Innovation Park at the Ecole polytechnique federale de Lausanne (EPFL), Switzerland.

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

Thursday, December 5, 2019 - 12:20pm

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)

Tuesday, June 18, 2019 - 8:18am

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)

Monday, June 3, 2019 - 1:00pm

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)

Monday, March 25, 2019 - 8:19am

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.