In late 2020, the FDA cleared the company's Investigational New Drug (IND) application for OTL-200, and the therapy also recently was approved in the
'Receipt of RMAT designation for OTL-200 underscores both the severe nature of MLD and the transformative potential of the therapy for young patients suffering from this devastating, fatal neurodegenerative condition,' said
Established under the 21st Century Cures Act, the RMAT designation program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition. The FDA granted Orchard RMAT designation for OTL-200 based on data submitted on 39 patients, including 9 patients from the
'We look forward to continued engagement with the FDA in the coming months to discuss the comprehensive data set we have already collected in the OTL-200 clinical development program and agree on the potential next steps on the regulatory path to approval for this innovative gene therapy,' said
About Libmeldy / OTL-200
Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, has been approved by the
The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
About Orchard
Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets,
Forward-Looking Statements
This press release contains certain forward-looking statements about Orchard's strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchard's business strategy and goals, the therapeutic potential of Libmeldy (OTL-200), the likelihood that data from clinical trials will support further clinical development and regulatory approval of OTL-200, and the outcome of planned FDA interactions regarding the potential approval pathway for OTL-200. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of OTL-200, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that OTL-200 or any one or more of Orchard's product candidates will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchard's ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the delay of any of Orchard's regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchard's product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchard's ability to commercialize OTL-200, if approved, or Libmeldy in the EU; the risk that the market opportunity for Libmeldy, or any of Orchard's product candidates, may be lower than estimated and the severity of the impact of the COVID-19 pandemic on Orchard's business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard include those identified under the heading 'Risk Factors' in Orchard's quarterly report on Form 10-Q for the quarter ended
Contact:
Tel: +1 862-242-0764
Email: Renee.Leck@orchard-tx.com
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