San Raffaele Hospital

Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy

Retrieved on: 
Wednesday, March 20, 2024
Encephalitis, ICER, Bone marrow, Patient, DSM-IV codes, Urinary tract infection, Travel, Child, Health technology assessment, Therapy, Children's Hospital of Philadelphia, Central nervous system, Risk, MLD, Qualification, Health, WAC, NBS, Nerve, Mutation, UCSF, Disease, Federation, Research, Acquisition, Genome, Rash, Stem cell, HSC, Hospital, Family, Swelling, FDA, Diagnosis, HTA, Caregiver, Traffic barrier, Viral, Fever, Kyowa Kirin, PSLI, Liver, Government, San Raffaele Hospital, ARSA, Pharmaceutical industry

TOKYO, LONDON and BOSTON, March 20, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the details of its U.S. commercial launch of Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, the first FDA-approved therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile, (PSEJ), or early symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

Key Points: 
  • Prior to Lenmeldy, there were no treatment options in the U.S. for early-onset MLD beyond supportive and end-of-life care.
  • This approach has the potential to restore enzymatic function to stop or slow disease progression with a single treatment.
  • Utilizing results from such studies, a multi-stakeholder working group is finalizing a nomination to add MLD to the U.S.
  • Based on current timelines and assumptions, Orchard Therapeutics expects the nomination will be submitted in mid-year 2024.

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

Retrieved on: 
Monday, March 18, 2024
Gastroenteritis, Genomics, Metabolism, Neutropenia, Brain, Nerve, Mutation, RPD, Disease, Incidence, Partnership, Central nervous system, Genetics, GSK, Stomatitis, Patient, Research, Fats, PRV, Traffic barrier, Child, Viral, Gene therapy, SAN, Genome, Urinary tract infection, Rash, Hepatomegaly, Priority review, Stem cell, Infection, Therapy, MLD, Kyowa Kirin, Febrile neutropenia, Fever, PSLI, Prognosis, Health, Family, FDA, San Raffaele Hospital, ARSA, Orchard, Food, Pharmaceutical industry

“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Retrieved on: 
Monday, February 5, 2024
Partnership, Mortality, Hurler, Orchard, Growth, Congress, Cell, Division, Heart, ERT, Glycosaminoglycan, GAG, Health, Disease, Patient, Safety, Transplant, FDA, EMA, Șaeș, San Raffaele Hospital, ESGCT, HSCT, Food, POC, Therapy, MPS, Hurler syndrome, Kyowa Kirin, Acquisition, Enzyme replacement therapy, European Medicines Agency, Gene, University, Eye, IDUA, HSC, RPD, Pharmaceutical industry

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH

Retrieved on: 
Thursday, November 30, 2023
Quality of life, Glycosaminoglycan, Food, ESGCT, Health, Antibody, FDA, Cell, Disease, Mortality, Tissue, Congress, Gene, HSC, POC, Therapy, RPD, Heart, Hurler syndrome, Eye, San Raffaele Hospital, Death, San Raffaele, ERT, European Medicines Agency, Cognitive development, IDUA, Growth, HSCT, Patient, Safety, Diagnosis, Hurler, Fast Track, Pharmaceutical industry

BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs. Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.

Key Points: 
  • Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs.
  • Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.
  • “We are encouraged that OTL-203 has been granted Fast Track designation because new treatment options are urgently needed for children with MPS-IH, as the current standard of care is associated with significant morbidity and mortality.
  • Throughout the PoC study, treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen.

Orchard Therapeutics Announces Presentation of Additional Positive Data from Proof-of-concept Study of OTL-203 in MPS-IH at ESGCT 2023

Retrieved on: 
Thursday, October 26, 2023
Glycosaminoglycan, Heart, Hurler syndrome, Hearing loss, Congress, Photophobia, POC, ERT, IDUA, Cell, Disease, MPS, Antibody, Gene, San Raffaele Hospital, HSC, The New England Journal of Medicine, ESGCT, Eye, Safety, European Society for Primary Immunodeficiencies, Therapy, Patient, Diagnosis, Death, Hurler, San Raffaele, European Society of Gene and Cell Therapy, HSCT, Pharmaceutical industry, Medical device, Medical imaging

The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.

Key Points: 
  • The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.
  • “The complications associated with MPS-IH involve multiple organ systems and have an adverse impact on patients’ quality of life.
  • Importantly, following treatment with OTL-203, no patients reported photophobia (light sensitivity), or any other ophthalmological symptoms typically associated with MPS-IH.
  • Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability.

CatalYm commences Dosing in a Phase 2 Study with Visugromab in Combination with Neoadjuvant Immunotherapy in Treatment-naive Muscle Invasive Bladder Cancer Patients

Retrieved on: 
Thursday, October 26, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Clinical Trials, Principal, Biomarker, Nivolumab, MIBC, Cancer, San Raffaele Hospital, Immunotherapy, Safety, Patient, Pharmaceutical industry

CatalYm today announced that the first patient has been dosed in the Phase 2 clinical trial, GDFather-NEO ( NCT06059547 ), evaluating its lead anti-GDF-15 antibody candidate, visugromab, in combination with neoadjuvant immunotherapy (nivolumab, aPD-1 inhibitor) in muscle invasive bladder cancer (MIBC).

Key Points: 
  • CatalYm today announced that the first patient has been dosed in the Phase 2 clinical trial, GDFather-NEO ( NCT06059547 ), evaluating its lead anti-GDF-15 antibody candidate, visugromab, in combination with neoadjuvant immunotherapy (nivolumab, aPD-1 inhibitor) in muscle invasive bladder cancer (MIBC).
  • The exploratory study expands visugromab’s clinical Phase 2 evaluation to a therapeutic front-line setting, building on the positive results from the ongoing GDFather-2 ( NCT04725474 ) trial in last-line metastatic patients with a range of advanced, treatment-resistant solid tumors.
  • In addition, the study will provide data on potential response-predictive biomarkers for patient stratification.
  • The trial will be led from Milan, Italy, and will enroll 30 patients in a 1:1 setting at four sites in Italy.

Orchard Therapeutics Announces Acceptance of Biologics License Application for OTL-200 in MLD and Receives Priority Review

Retrieved on: 
Monday, September 18, 2023
Priority review, Society, Natural history, Orchard, MLD, San Raffaele Hospital, Death, Prescription Drug User Fee Act, Patient, Data, Child, Busulfan, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Report of the 1st Annual Symposium on Relaxed Improvisation, DSM-IV codes, Food, Disease, Pharmaceutical industry, Metabolism, PDUFA

BOSTON and LONDON, Sept. 18, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.

Key Points: 
  • The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.
  • “We look forward to collaborating with the FDA throughout the review and evaluation of our application.
  • With more than a cumulative 250 patient-years of follow-up, treatment with OTL-200 was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • The full clinical results comprising the BLA dataset were recently presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem.

FONDAZIONE TELETHON AND ORCHARD THERAPEUTICS COMPLETE TRANSFER OF MARKETING AUTHORIZATION OF STRIMVELIS FOR ADA-SCID IN EUROPE

Retrieved on: 
Tuesday, September 12, 2023
Gene, Civil service commission, Marketing, SAN, European Medicines Agency, Research, EMA, Patient, San Raffaele Hospital, ADA-SCID, Therapy, European Commission, Adenosine deaminase, Vaccine, Medical device

MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the completion of the transfer of the marketing authorization for Strimvelis, a gene therapy approved by the European Medicines Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Key Points: 
  • The marketing authorization transfer was approved on July 17th by the European Commission[1] following a positive opinion from the European Medicines Agency (EMA).
  • The European manufacturing and distribution rights have been fully transferred to Fondazione Telethon from its former holder, Orchard Therapeutics, which previously announced it would discontinue investment in and seek strategic alternatives for its programs in rare primary immune deficiencies, including Strimvelis.
  • A total of 45 patients from over 20 countries worldwide have been treated with Strimvelis in clinical trials and commercially, to date.
  • Fondazione Telethon will continue to make Strimvelis available to eligible patients through the San Raffaele Hospital in Milan, Italy.

FONDAZIONE TELETHON AND ORCHARD THERAPEUTICS COMPLETE TRANSFER OF MARKETING AUTHORIZATION OF STRIMVELIS FOR ADA-SCID IN EUROPE

Retrieved on: 
Tuesday, September 12, 2023
Gene, Civil service commission, Marketing, SAN, European Medicines Agency, Research, EMA, Patient, San Raffaele Hospital, ADA-SCID, Therapy, European Commission, Adenosine deaminase, Vaccine, Medical device

MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the completion of the transfer of the marketing authorization for Strimvelis, a gene therapy approved by the European Medicines Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Key Points: 
  • The marketing authorization transfer was approved on July 17th by the European Commission[1] following a positive opinion from the European Medicines Agency (EMA).
  • The European manufacturing and distribution rights have been fully transferred to Fondazione Telethon from its former holder, Orchard Therapeutics, which previously announced it would discontinue investment in and seek strategic alternatives for its programs in rare primary immune deficiencies, including Strimvelis.
  • A total of 45 patients from over 20 countries worldwide have been treated with Strimvelis in clinical trials and commercially, to date.
  • Fondazione Telethon will continue to make Strimvelis available to eligible patients through the San Raffaele Hospital in Milan, Italy.

Orchard Therapeutics Announces Presentation of Data Comprising the Clinical Package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023

Retrieved on: 
Thursday, August 31, 2023
Natural history, MLD, San Raffaele Hospital, Locomotion, SMF 120.9, Patient, Composite, FDA, Therapy, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Disease, Pharmaceutical industry, Metabolism

BOSTON and LONDON, Aug. 31, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced long-term results from an updated integrated analysis of 39 patients with metachromatic leukodystrophy (MLD) treated with investigational OTL-200 in the clinical development program. The data were presented yesterday at the ongoing Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem.

Key Points: 
  • The data were presented yesterday at the ongoing Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem.
  • “MLD is a devastating and ultimately fatal disease for which there are no alternative treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.
  • Importantly, use of sMFS was discussed with the U.S. Food and Drug Administration (FDA) who agreed it is clinically meaningful.
  • Orchard Therapeutics has requested priority review, which if granted, would put OTL-200 on track for a potential U.S. approval in the first half of 2024.