- |||||||||| Review, Journal: Impact of Disease-modifying Therapies on Respiratory Function in People with Neuromuscular Disorders. (Pubmed Central) - Aug 3, 2024
The paradigm of care is changing as new disease-modifying therapies are altering disease trajectory, outcomes, expectations, as well as patient and caregiver experiences. This article provides an overview on therapeutic advances for SMA and DMD in the last 10 years, with a focus on the effects of disease-modifying therapies on respiratory function.
- |||||||||| Exondys 51 (eteplirsen) / Sarepta Therap
Journal: Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study. (Pubmed Central) - Jul 22, 2024 This article provides an overview on therapeutic advances for SMA and DMD in the last 10 years, with a focus on the effects of disease-modifying therapies on respiratory function. This exploratory study indicated that most caregivers perceived improvements or maintenances in aspects of their child's physical functioning, ADLs, and HRQoL since eteplirsen initiation, which they perceived to be a positive outcome.
- |||||||||| Exondys 51 (eteplirsen) / Sarepta Therap
Journal, Adverse events: Adverse events associated with eteplirsen: A disproportionality analysis using the 2016-2023 FAERS data. (Pubmed Central) - Jul 19, 2024 This has contributed to a deeper understanding of the complex interrelations between adverse reactions and the use of eteplirsen. The findings underscore the critical importance of ongoing monitoring and sustained observation to promptly detect and effectively manage AEs, thereby enhancing the overall safety and well-being of patients treated with eteplirsen for DMD.
- |||||||||| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
Review, Journal, Gene therapy: How safe is gene therapy? : Second death after Duchenne therapy (Pubmed Central) - May 30, 2024 As more data become available, the impact of eteplirsen on survival will be further elucidated. Although gene therapy applications of AAV vectors are generally considered safe, the systemic administration of high vector doses can lead to severe side effects with a
- |||||||||| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
Clinical, Journal, Video: Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy. (Pubmed Central) - May 16, 2024 P1/2 Participants in this analysis received delandistrogene moxeparvovec (as part of SRP-9001-101 [Study 101; NCT03375164] or SRP-9001-102 [Study 102; NCT03769116]) or were randomized to receive placebo (in Part 1 of Study 102)...The results showed that scores from remote functional assessment of patients with Duchenne muscular dystrophy strongly correlated with those obtained in person. These findings demonstrate congruence between live stream remote and in-person functional assessment and suggest that remote assessment has the potential to reduce the burden on a family by supplementing in-clinic visits.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi, Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Imlifidase for Kidney Transplantation of Highly Sensitized Patients with a Positive Crossmatch: The French Consensus Guidelines () - May 12, 2024 - Abstract #EFI2024EFI_259; Frequent post-transplant testing for DSA and systematic surveillance kidney biopsies are strongly recommended to monitor post-transplant DSA rebound and detect subclinical rejection. These French guidelines aim to provide valuable insights into refining the use and implementation of Imlifidase, a major breakthrough in kidney transplantation.
- |||||||||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap, Rituxan (rituximab) / Roche
Delisting Strategy to Enable DSA Positive and Cross-Match Negative Kidney Transplantation in Hypersensitized Patients with 100% cPRA (108-AB, Level 1) - May 6, 2024 - Abstract #ATC2024ATC_2896; Upon kidney offer, if flow-cytometry (FC-XM) and complement-dependent cytotoxicity (CDC-XM) cross-match were negative, patients were transplanted and received post-transplant desensitization with rituximab, apheresis and immunoglobulins.* From June 2022 to February 2023, we delisted HLA-antigens from 14 patients in the waiting list with a cPRA=100% who had waited in the prioritization Spanish program (PATHI) for a median time of 5.8[4.6-9.1] years...Only one patient had a positive FC-XM and was desensitized before transplantation with imlifidase and discarded from the analysis... Since DSA are not invariably associated with early graft failure due to rejection, delisting strategy and post-transplant desensitization may be considered for hypersensitized transplant candidates with 100% cPRA who have not any other options of finding a compatible match.
- |||||||||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Long-Term Follow Up of Imlifidase Desensitized Kidney Transplant Recipients: 5 Year Pooled Analysis (108-AB, Level 1) - May 6, 2024 - Abstract #ATC2024ATC_2895; Since DSA are not invariably associated with early graft failure due to rejection, delisting strategy and post-transplant desensitization may be considered for hypersensitized transplant candidates with 100% cPRA who have not any other options of finding a compatible match. At 5 years, XM+ imlifidase enabled transplant recipients continue to demonstrate expected patient and graft survival and despite the high-risk immunological profile of these patients, the relative stability of allograft function indicates imlifidase is a viable treatment option for patients whose breadth and depth of sensitization may preclude them from receiving a compatible transplant.
- |||||||||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
IgM Cleaving Enzyme on Anti-Pig Xenoreative IgM and IgG in Nonhuman Primate Model (114 - Nutter Theater, Level 1) - May 6, 2024 - Abstract #ATC2024ATC_2446; Repeat administration of IceM in macaques enables selective, robust and transient (or prolonged) clearance of circulating IgM. This approach will be useful in treating preformed natural IgM and rebound IgM during antibody-mediated rejection in xenotransplantation.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi, Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
A Phase II Study Investigating DSA Rebound in Highly Sensitized Living Donor Kidney Transplant Recipients Treated with Imlifidase (107-AB, Level 1) - May 6, 2024 - Abstract #ATC2024ATC_1767; Imlifidase is effective at converting a positive crossmatch to negative, enabling living donor kidney transplantation in highly sensitized patients who were previously unable to be transplanted with other modalities with substantial time in paired exchange. The proposed desensitization regimen appears safe and effective at suppressing DSA rebound and reducing the risk of early AMR.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Low Molecular Evolution of SARS-CoV-2 in Kidney Transplant Recipients (118-ABC, Level 1) - May 6, 2024 - Abstract #ATC2024ATC_1192; CDC guidelines recommending 20 days of isolation in immunosuppressed individuals seems to be too conservative for most KTR, with the exception of those within 1 year after transplantation, receiving T-cell depletive induction. There were no warning signals of molecular evolution and all KTRs cleared the virus, including those with high immunosuppressive burden such as imlifidase treatment.
- |||||||||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Reestablishment of COVID-Specific IgG Antibodies after Imlifidase Treatment (Poster Hall, Exhibit Hall A, Level 2) - May 6, 2024 - Abstract #ATC2024ATC_911; However, it should be noted that other components of the immune system such as IgM and IgD are left intact and able to respond to active infection during imlifidase treatment. This was highlighted by the subject who was infected with COVID during the study.
- |||||||||| Translarna (ataluren) / PTC Therap, Exondys 51 (eteplirsen) / Sarepta Therap, Emflaza (deflazacort) / Marathon, PTC Therap
Retrospective data, Review, Journal, IO biomarker: Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis. (Pubmed Central) - May 3, 2024 Glucocorticoid therapy was associated with delayed loss of ambulation (overall meta-analysis HR deflazacort/prednisone/prednisolone: 0.44 [95% CI: 0.40-0.48]) (n?=?25 studies)...Treatment with ataluren (n?=?2 studies) and eteplirsen (n?=?3 studies) were associated with prolonged ambulation...In total, 33% of studies exhibited some risk of bias. Our synthesis of predictors of loss of ambulation in DMD contributes to the understanding the natural history of disease and informs the design of new trials of novel therapies targeting this heavily burdened patient population.
- |||||||||| scAAV1.tMCK.NTF3 / Sarepta Therap
Trial completion date, Trial primary completion date: Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A (clinicaltrials.gov) - May 1, 2024 P1/2, N=3, Suspended, Percent dystrophin positive fibers also rose from 14% Trial completion date: Jul 2024 --> Apr 2030 | Trial primary completion date: Mar 2024 --> Apr 2028
- |||||||||| Amondys 45 (casimersen) / Sarepta Therap
Review, Journal: Casimersen (AMONDYS 45 (Pubmed Central) - Apr 27, 2024 While casimersen is currently continuing in phase III of clinical trials in various countries, it was granted approval by the FDA under the accelerated approval program due to its observed increase in dystrophin production. This article discusses the pathophysiology of DMD, summarizes available treatments thus far, and provides a full drug review of casimersen (AMONDYS 45TM).
- |||||||||| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
Review, Journal: Duchenne muscular dystrophy: promising early-stage clinical trials to watch. (Pubmed Central) - Mar 11, 2024 The approval of Elevidys, a micro-dystrophin therapy, is reflected in a recent trend toward gene transfer therapies in phase I DMD clinical trials, but their safety and efficacy are being established in this phase of development. Other Phase I clinical-stage approaches are diverse, but have a range in efficacy, safety, and endpoint measures.
- |||||||||| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap, Emflaza (deflazacort) / Marathon, PTC Therap, Viltepso (viltolarsen) / Nippon Shinyaku
Why It Is Important to Ensure the Price Is Right: Access Restrictions for Therapies Treating Duchenne Muscular Dystrophy () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2137; This importance only intensifies as more therapies become available. While most plans currently cover DMD therapies, if value and price are perceived to be misaligned, stakeholders can attempt to restrict access to who they determine as the most appropriate patients for treatment (i.e., population studied in the trial) or only approve on a case-by-case basis (i.e., non-formulary).
- |||||||||| Review of Gene Therapy Access Landscape () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_1812;
Recently launched ultra-high-cost therapies face additional restrictions compared to lower-priced (<$3M) gene therapies. Therapies on the market longer (e.g., Luxturna, Zolgensma) employ more types of contracting strategies and face less restrictive management.
- |||||||||| Journal: Characteristics of Patients Receiving Novel Muscular Dystrophy Drugs in Trials vs Routine Care. (Pubmed Central) - Jan 24, 2024
These findings raise questions about the translation of DMD drug trial findings to routine care settings, with patients in routine care discontinuing the treatment within 1 year and payers incurring substantial expenses for these medications. More data are needed on whether these high costs are accompanied by corresponding clinical benefits.
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