Featured data include several accepted abstracts and an Oral Presidential Symposium supporting the safety and efficacy of atidarsagene autotemcel (formerly OTL-200 which was recently approved as Lenmeldy™ in the
“Our presentations at ASGCT add to the compendium of evidence supporting the transformative impact and broad applicability of our approach,” said
Details of the oral presentations are follows (all times in EDT):
- Title: Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal Cross-correction Mechanisms
Date/Time:Tuesday, May 7 at1:30 p.m.
Presenter:Maria Ester Bernardo
- Title: Atidarsagene autotemcel (Hematopoietic Stem Cell Gene Therapy) Preserves Cognitive and
Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-up (Oral Presidential Symposium)
Date/Time:Wednesday, May 8 at11:15 a.m.
Presenter: Alessandro Aiuti
- Title: Restoring Macrophage Immune Functions by Transplantation of Gene-modified HSCs: a Therapeutic Approach to NOD2 Crohn’s Disease (Invited Oral Presentation)
Date/Time:Thursday, May 9 at9:18 a.m.
Presenter:Pervinder Sagoo
- Title: Somatic Mutation Tracking in Hematopoietic Stem Cell Gene Therapy Reveals Absence of Clonal Hematopoiesis
Date/Time:Saturday, May 11 at11:45 a.m.
Presenter:Francesco Gazzo
Details of the poster presentations are as follows (all times in EDT):
- Title: Development of an Ex
Vivo Hematopoietic Stem Cell Gene Therapy for Frontotemporal Dementia (FTD)
Date/Time:Thursday, May 9 fromnoon to 1:30 p.m. and from5:30 to 7:00 p.m.
Presenter:Yuri Ciervo
Poster #1136
- Title: Lentiviral Hematopoietic Stem Cell Gene Therapy for Late Juvenile Metachromatic leukodystrophy
Date/Time:Friday, May 10 fromnoon to 1:30 p.m. and from5:30 to 7:00 p.m.
Presenter:Valeria Calbi
Poster #1905
- Title: Non-neurological, Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene therapy in Hurler Patients: Retrospective Comparison with Allogeneic Hematopoietic Stem Cell Transplantation
Date/Time:Friday, May 10 fromnoon to 1:30 p.m. and from5:30 to 7:00 p.m.
Presenter:Maria Ester Bernardo
Poster #1904
- Title: Interim Analysis on Neurological Outcomes in Hurler Syndrome Patients Treated with Autologous Ex Vivo Hematopoietic Stem Cell Gene Therapy
Date/Time:Friday, May 10 fromnoon to 1:30 p.m. and from5:30 to 7:00 p.m.
Presenter:Maria Ester Bernardo
Poster #1903
Early skeletal outcomes from OTL-203 PoC study in MPS-IH published in Science Translational Medicine
In addition to the data presented at ASGCT, Orchard’s collaborators at the
The manuscript, titled, “Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome,” was published in the
About Lenmeldy
Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, is the only approved therapy in the
For additional details about Lenmeldy, please refer to the full Prescribing Information.
In
The program was originated by and developed in partnership with the
INDICATION
LENMELDYTM (atidarsagene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Thrombosis and Thromboembolic Events:
Treatment with LENMELDY may increase the risk of thrombosis and thromboembolic events. A child with PSEJ MLD died after experiencing a left hemisphere cerebral infarction secondary to a thrombotic event in a large blood vessel approximately 1 year after treatment with LENMELDY. Evaluate the risk factors for thrombosis prior to and after LENMELDY infusion according to best clinical practice.
Encephalitis:
Treatment with LENMELDY may increase the risk of encephalitis. A child with ESEJ developed a serious event of encephalitis after treatment with LENMELDY. The etiology of this event is unclear but attribution to LENMELDY cannot be ruled out. Treatment with LENMELDY may trigger a relapsing-remitting pattern of disease progression. No other events related to encephalitis have been reported during the clinical development of LENMELDY. Monitor children for signs or symptoms of encephalitis after LENMELDY treatment.
Serious Infection:
In the period between start of conditioning and within 1 year after LENMELDY treatment, severe Grade 3 infections occurred in 39% of all children (21% bacterial, 5% viral, 5% bacterial and viral or bacterial and fungal, and 8% unspecified). Grade 3 febrile neutropenia developed within 1 month after LENMELDY infusion in 82% of children. In the event of febrile neutropenia, monitor for signs and symptoms of infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated. Monitor children for signs and symptoms of infection after myeloablative conditioning and LENMELDY infusion and treat appropriately. Administer prophylactic antimicrobials according to best clinical practice.
Veno-Occlusive Disease:
Three children (8%) treated in clinical trials of LENMELDY developed veno-occlusive disease (VOD) with one Grade 4 SAE and two Grade 3 AEs. None of these three events met Hy’s Law criteria. Monitor children for signs and symptoms of VOD including liver function tests in all children during the first month after LENMELDY infusion. Consider prophylaxis for VOD with anti-thrombotic agents based on risk factors for VOD and best clinical practice.
Delayed Platelet Engraftment (DPE):
DPE has been observed with LENMELDY treatment. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in children with prolonged thrombocytopenia. In clinical trials of LENMELDY, 4 (10%) children had delayed platelet engraftment after day 60 (range day 67-109), with 3 children requiring platelet transfusions until engraftment occurred. Patients should be informed of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding until platelet engraftment and recovery are achieved.
Neutrophil Engraftment Failure:
There is a potential risk of neutrophil engraftment failure after treatment with LENMELDY. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a child treated with LENMELDY, provide rescue treatment with the unmanipulated back-up collection of CD34+ cells.
Insertional Oncogenesis:
There is a potential risk of LVV-mediated insertional oncogenesis after treatment with LENMELDY. Children treated with LENMELDY may develop hematologic malignancies and should be monitored life-long. Monitor for hematologic malignancies with a complete blood count (with differential) annually and integration site analysis as warranted for at least 15 years after treatment with LENMELDY. In the event that a malignancy occurs, contact
Hypersensitivity Reactions:
The dimethyl sulfoxide (DMSO) in LENMELDY may cause hypersensitivity reactions, including anaphylaxis which is potentially life-threatening and requires immediate intervention. Hypersensitivity including anaphylaxis can occur in children with and without prior exposure to DSMO. Monitor for hypersensitivity reactions during infusion and after infusion.
Anti-Retroviral Use:
Children should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization, or for the expected duration of time needed for the elimination of the medications. Anti-retroviral medications may interfere with the manufacturing of LENMELDY. If a child requires antiretrovirals for HIV prophylaxis, initiation of LENMELDY treatment should be delayed until confirmation of a negative test for HIV.
Interference With Serology Testing:
Due to the likelihood of a false-positive test for HIV, children who have received LENMELDY should not be screened for HIV infection using a PCR-based assay.
USE IN SPECIFIC POPULATIONS
Females and Males of Reproductive Potential
Pregnancy Testing
As a precautionary measure, a negative serum pregnancy test must be confirmed prior to the start of mobilization, and reconfirmed prior to conditioning procedures, and before administration of LENMELDY in females of childbearing potential.
Contraception
Consult the Prescribing Information of the mobilization and conditioning agents for information on the need for effective contraception. Males capable of fathering a child and females of childbearing age should use an effective method of contraception from start of mobilization through at least 6 months after administration of LENMELDY.
Infertility
There are no data on the effects of LENMELDY on fertility.
Data are available on the risk of infertility with myeloablative conditioning. In clinical trials of LENMELDY, seven children (50% of females) developed ovarian failure. Advise children of the option to cryopreserve semen or ova before treatment, if appropriate.
For additional safety information, please see the full Prescribing Information.
About
Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.
For more information, please visit www.orchard-tx.com.
About Kyowa Kirin
Kyowa Kirin aims to discover novel medicines with life-changing value. As a
ContactBenjamin Navon +1 857-248-9454 Benjamin.Navon@orchard-tx.com
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