Emmaus 
Welcome,         Profile    Billing    Logout  
  Products    Diseases    Products    Trials    News 
  • ||||||||||  Oxbryta (voxelotor) / Pfizer, Adakveo (crizanlizumab-tmca) / Novartis, Endari (L-glutamine) / Emmaus
    Prescribing Trends of Newly Approved Sickle Cell Disease-Modifying Medications () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_296;    
    Between 2016 and 2021, the dispensing rates for DMTs increased slightly, especially after the approval of Adakveo and Oxbryta. However, the overall dispensing rates of newly approved DMTs remained low.
  • ||||||||||  Lyfgenia (lovotibeglogene autotemcel) / bluebird bio, Casgevy (exagamglogene autotemcel) / Vertex, CRISPR Therap, Molecular Templates
    Journal, Gene therapy:  Casgevy and Lyfgenia: Two gene therapies for sickle cell disease. (Pubmed Central) -  Jan 11, 2024   
    However, the overall dispensing rates of newly approved DMTs remained low. No abstract available
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Trial completion, Enrollment change:  L-glutamine Treatment in Patients With Diverticulosis (clinicaltrials.gov) -  Feb 16, 2023   
    P1,  N=9, Completed, 
    Acute chest syndrome, Vasoocclusive crisis, Sickle cell disease, Red blood cell Active, not recruiting --> Completed | N=15 --> 9
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Journal, Real-world evidence:  Real-World data on efficacy of L-glutamine in preventing sickle cell disease-related complications in pediatric and adult patients. (Pubmed Central) -  Aug 22, 2022   
    The primary objective was to confirm the efficacy of L-glutamine (Endari) therapy in pediatric and adult patients with SCD at follow-up time points of 24, 48 and 72 weeks...No patient reported treatment-related adverse events. Although the sample size was small, our data clearly demonstrated that L-glutamine therapy was safe and significantly improved clinical outcomes and hemolysis parameters in patients with SCD.
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Trial completion:  Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients (clinicaltrials.gov) -  Aug 2, 2022   
    P4,  N=13, Completed, 
    Although the sample size was small, our data clearly demonstrated that L-glutamine therapy was safe and significantly improved clinical outcomes and hemolysis parameters in patients with SCD. Recruiting --> Completed
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Trial completion date, Trial primary completion date:  L-glutamine Treatment in Patients With Diverticulosis (clinicaltrials.gov) -  Jul 26, 2022   
    P1,  N=15, Active, not recruiting, 
    Recruiting --> Completed Trial completion date: Jun 2022 --> Dec 2022 | Trial primary completion date: May 2022 --> Oct 2022
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Journal:  A re-analysis of pain crises data from the pivotal l-glutamine in sickle cell disease trial. (Pubmed Central) -  Nov 29, 2021   
    The pivotal Endari trial in sickle cell disease showed a reduction in pain crises events...The observed 45% difference in the VOC rate is comparable to what was reported in other sickle cell therapeutics used to reduce the incidence of pain. The results presented in this communication are informative for clinicians evaluating treatment effects across available SCD therapeutic options based on studies that utilized VOC as the primary endpoint.
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Safety Profile of L-Glutamine in Patients with Sickle Cell Disease: Data from Post-Marketing Surveillance () -  Nov 24, 2021 - Abstract #ASH2021ASH_6853;    
    The most common side effects observed during clinical trials were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain (Table 1). These same side effects have been observed in the post-marketing phase with the exception of "cough." Side effects reported during the post-marketing phase that were not reported in the clinical trials were abdominal discomfort, diarrhea, malaise, and pain.
  • ||||||||||  Oxbryta (voxelotor) / Global Blood Therap, Adakveo (crizanlizumab) / Novartis, Endari (L-glutamine) / Emmaus
    Review, Journal:  Research in Sickle Cell Disease: From Bedside to Bench to Bedside. (Pubmed Central) -  Jun 8, 2021   
    HU has other salutary effects, reduction of cellular adhesion to the vascular endothelium and inflammation, and dissecting these mechanisms has informed bench-both cellular and animal-research for development of the 3 recently approved agents: endari, voxelotor, and crizanlizumab; truly, a bidirectional bench to bedside translation. Decades of research to understand the mechanisms of fetal to adult hemoglobin have also culminated in promising anti-sickling genetic therapies and the first-in-human studies of reactivating an endogenous (γ-globin) gene HBG utilizing innovative genomic approaches.
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Enrollment open, Metastases:  Trial of First-line L-glutamine with Gemcitabine and Nab-paclitaxel in Advanced Pancreatic Cancer (clinicaltrials.gov) -  Apr 18, 2021   
    P1,  N=16, Recruiting, 
    Decades of research to understand the mechanisms of fetal to adult hemoglobin have also culminated in promising anti-sickling genetic therapies and the first-in-human studies of reactivating an endogenous (γ-globin) gene HBG utilizing innovative genomic approaches. Not yet recruiting --> Recruiting
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Trial termination:  L-Glutamine Therapy for Sickle Cell Anemia (clinicaltrials.gov) -  Sep 2, 2020   
    P2,  N=15, Terminated, 
    These findings, combined with computational prediction of GLN benefit vs. Completed --> Terminated; Inadequate clinical supplies and Emmaus business decision.
  • ||||||||||  Journal:  Two drugs for sickle cell disease. (Pubmed Central) -  May 19, 2020   
    Completed --> Terminated; Inadequate clinical supplies and Emmaus business decision. No abstract available
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Rheological Effects of L-Glutamine in Patients with Sickle Cell Disease (Hall B, Level 2 (Orange County Convention Center)) -  Nov 7, 2019 - Abstract #ASH2019ASH_5277;    
    RBCs incubated with L-glutamine also had significantly higher EImin, meaning deoxygenated RBCs were more flexible and deformable. Whole blood viscosity at 45s-1 and 225s-1 did not change significantly following incubation with L-glutamine; %DRBCs also did not change significantly (Table 1).
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Barriers to the Use of Endari™ in an Urban Adult Sickle Cell Center (Hall B, Level 2 (Orange County Convention Center)) -  Nov 7, 2019 - Abstract #ASH2019ASH_3439;    
    From our report, it is critical to evaluate and mitigate barriers to initiation and adherence to Endari, to ensure it is available to and accepted by the patient population it gained approval and was intended for. N Engl J Med.2018 Jul 19;379(3):226-235
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Journal:  Antisickling Drugs Targeting βCys93 Reduce Iron Oxidation and Oxidative Changes in Sickle Cell Hemoglobin. (Pubmed Central) -  Aug 10, 2019   
    ...Molecules that specifically bind to or modify βCys93, such as 4,4'-di(1,2,3-triazolyl) disulfide (TD-3) and hydroxyurea (HU) were contrasted with molecules that target other sites on Hb including 5-hydroxymethyl-2-furfural (5-HMF) and L-glutamine...Increases in the delay times prior to polymerization of HbS under hypoxia were in the following order: TD-3 > HU > 5-HMF = L-glutamine. Designing antisickling agents that can specifically target βCys93 may provide a dual antioxidant and antisickling therapeutic benefits in treating this disease.
  • ||||||||||  Endari (L-glutamine) / Emmaus
    Trial completion:  L-Glutamine Therapy for Sickle Cell Anemia and Sickle  (clinicaltrials.gov) -  Aug 8, 2017   
    P2,  N=81, Completed, 
    Designing antisickling agents that can specifically target βCys93 may provide a dual antioxidant and antisickling therapeutic benefits in treating this disease. No longer recruiting --> Completed