Drugs / ATIDARSAGENE AUTOTEMCEL
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | metachromatic leukodystrophy | 2023-08-03 | “The company has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for OTL-200” orchard-tx.com ↗ |
| Filed | US (FDA) | — | metachromatic leukodystrophy | 2023-05-15 | “I am delighted to announce the initiation of our OTL-200 rolling BLA submission with the filing of the first module” orchard-tx.com ↗ |
| Filed | EU (EMA) | — | metachromatic leukodystrophy | 2019-12-02 | “today announced that the European Medicines Agency (EMA) has validated the company’s Marketing Authorization Application (MAA) for OTL-200” orchard-tx.com ↗ |
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT01560182 | Apr 2010 → Apr 2018 | metachromatic leukodystrophy | Orchard Therapeutics | Completed | Met primary |
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT03392987 | Jan 2018 → Apr 2022 | metachromatic leukodystrophy | Orchard Therapeutics | Completed | Met primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT04283227 | Jan 2022 → Jan 2026 overdue | metachromatic leukodystrophy | Orchard Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 23
| Date | Issuer | Release |
|---|---|---|
| 2025-10-28 | Orchard Therapeutics | Regulatory Kyowa Kirin and Orchard Therapeutics Announce OTL-200 Granted Orphan Regenerative Medicine Product Designation for Early-onset MLD in Japan orchard-tx.com ↗ |
| 2024-03-18 | Orchard Therapeutics | Regulatory Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S. orchard-tx.com ↗
today announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, 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). |
| 2023-12-11 | Orchard Therapeutics | Regulatory Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD). |
| 2023-11-13 | Orchard Therapeutics | Regulatory Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments orchard-tx.com ↗
With our BLA file for OTL-200 in MLD accepted by the FDA under Priority Review, we are one step closer to potentially bringing this important therapy to families in the U.S. |
| 2023-09-18 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces Acceptance of Biologics License Application for OTL-200 in MLD and Receives Priority Review orchard-tx.com ↗
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. |
| 2023-08-31 | Orchard Therapeutics | Results Orchard Therapeutics Announces Presentation of Data Comprising the Clinical Package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023 orchard-tx.com ↗
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. |
| 2023-08-03 | Orchard Therapeutics | Regulatory Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA orchard-tx.com ↗ |
| 2023-05-15 | Orchard Therapeutics | Regulatory Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of OTL-200 for Metachromatic Leukodystrophy to U.S. FDA orchard-tx.com ↗ |
| 2023-02-24 | Orchard Therapeutics | Results Orchard Therapeutics Announces Presentation of Updated Integrated Analysis of OTL-200 in MLD and Reports Progress with Newborn Screening Efforts at the 19th Annual WORLDSymposium™ orchard-tx.com ↗
The three patient deaths were considered unrelated to treatment with OTL-200. |
| 2022-01-21 | Orchard Therapeutics | Results Orchard Therapeutics Announces Publication in The Lancet of Long-term Clinical Outcomes with Libmeldy for the Treatment of Children with Early-onset MLD orchard-tx.com ↗
Twenty-six of 29 patients (90%) were alive with median follow-up of 3.2 years (range 0.64 to 7.51 years) in all participants. |
| 2021-02-09 | Orchard Therapeutics | Results Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH) orchard-tx.com ↗
Updated clinical data for OTL-200 in patients with MLD were also presented, including results in early-symptomatic early-juvenile (ES-EJ) patients as well as new data from a cryopreserved formulation of OTL-200. |
| 2021-01-14 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic Leukodystrophy (MLD) orchard-tx.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200 |
| 2020-12-23 | Guangzhou Reforgene Medicine Co., Ltd. | Regulatory 欧盟批准自体造血干细胞基因疗法治疗异染性脑白质营养不良 reforgene.com ↗
Libmeldy的营销授权在欧盟的所有27个成员国以及英国，冰岛，列支敦士登和 挪威有效。 |
| 2020-12-21 | Orchard Therapeutics | Regulatory Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) orchard-tx.com ↗
today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy |
| 2020-11-19 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces FDA Clearance of IND Application for OTL-200 for Metachromatic Leukodystrophy (MLD) orchard-tx.com ↗ |
| 2020-10-16 | Orchard Therapeutics | Regulatory Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy |
| 2019-12-02 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces the Filing and Validation of Marketing Authorization Application by European Medicines Agency for OTL-200 for the Treatment of Metachromatic Leukodystrophy orchard-tx.com ↗
Validation of the MAA confirms that the submission is sufficiently complete to begin the formal review process. |
| 2019-11-18 | Orchard Therapeutics | Regulatory European Medicines Agency Grants Orchard Therapeutics Accelerated Assessment of OTL-200 for Patients with Metachromatic Leukodystrophy orchard-tx.com ↗ |
| 2019-11-06 | Orchard Therapeutics | Results Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting orchard-tx.com ↗
We now have two supportive data sets — one from our OTL-101 program in ADA-SCID and one from our OTL-200 program in metachromatic leukodystrophy — that demonstrate cryopreserved formulations are engrafting as expected, similar to the fresh formulation. |
| 2019-10-22 | Orchard Therapeutics | Results Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. |
| 2019-09-04 | Orchard Therapeutics | Results Orchard Therapeutics Presents an Integrated Data Analysis Demonstrating Sustained Clinical Benefit of OTL-200 for the Treatment of Metachromatic Leukodystrophy orchard-tx.com ↗
The results, which demonstrate positive clinical effects of OTL-200 in the treatment of MLD and a consistent safety profile, were featured today in an oral presentation at the Society for the Study of Inborn Errors of Metabolism (SSIEM) symposium in Rotterdam, the Netherlands. |
| 2019-03-27 | Orchard Therapeutics | Results Orchard Therapeutics Presents New Registrational Data Demonstrating Sustained Clinical Benefit of OTL-200 for the Treatment of MLD orchard-tx.com ↗
Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, presented the full registrational dataset from a 20 patient trial evaluating the efficacy and safety of OTL-200, an ex vivo autologous hematopoietic stem cell-based (HSC) gene therapy for the treatment of metachromatic leukodystrophy (MLD). |
| 2018-05-03 | Orchard Therapeutics | Regulatory Orchard Therapeutics’ OTL-200 Receives Rare Pediatric Disease Designation from FDA for Treatment of Metachromatic Leukodystrophy orchard-tx.com ↗ |
All press releases naming this drug 34 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ATIDARSAGENE AUTOTEMCEL | ChEMBL registry synonym — accepted as the source's own label CHEMBL4802262 ↗ |
| Known as | Lenmeldy | ChEMBL registry synonym — accepted as the source's own label CHEMBL4802262 ↗ |
| Known as | Libmeldy | ChEMBL registry synonym — accepted as the source's own label CHEMBL4802262 ↗ |
| Known as | OTL-200 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01560182 ↗ |
| Action | Restore | “a functional ARSA cDNA is introduced” NCT01560182 ↗ |
| Route | Intravenous | “intravenous (IV) infusion of OTL-200 gene therapy” NCT03392987 ↗ |
| Target | ARSA | “lentiviral vector that encodes for the human ARSA cDNA sequence” NCT03392987 ↗ |