{"id":775054,"date":"2023-08-03T10:31:38","date_gmt":"2023-08-03T14:31:38","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/orchard-therapeutics-completes-submission-of-biologics-license-application-for-otl-200-in-mld-to-u-s-fda\/"},"modified":"2023-08-03T10:31:38","modified_gmt":"2023-08-03T14:31:38","slug":"orchard-therapeutics-completes-submission-of-biologics-license-application-for-otl-200-in-mld-to-u-s-fda","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/orchard-therapeutics-completes-submission-of-biologics-license-application-for-otl-200-in-mld-to-u-s-fda\/","title":{"rendered":"Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA"},"content":{"rendered":"
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\n Reported $6.6M in Q2\u201923 Libmeldy net sales, representing the highest quarter to date<\/em>
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\n $34.0M of additional capital from second closing of strategic financing extends runway to mid-2025<\/em>\n <\/p>\n

\n Four MLD patients identified from ~150,000 newborns screened in prospective studies suggests significantly higher incidence than previously estimated in the medical literature<\/em>\n <\/p>\n

\n Six presentations at ASGCT demonstrate the ability of HSC gene therapy to address neurometabolic and CNS disorders, as well as larger indications<\/em>\n <\/p>\n

\n Company to host conference call and live webcast today at 8:00 a.m. EDT<\/em>\n <\/p>\n

BOSTON and LONDON, Aug. 03, 2023 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced several business accomplishments along with its financial results for the quarter ended June 30, 2023.<\/p>\n

\u201cWith the completion of the rolling BLA submission for OTL-200 to the FDA, we are now one significant step closer to bringing this important therapy to families in the U.S. affected by MLD who currently have no treatment options beyond supportive care,\u201d said Bobby Gaspar, M.D., Ph.D., chief executive officer. \u201cWe look forward to working with the agency throughout the filing and review process and expect to hear from the FDA regarding acceptance of the BLA in the third quarter of this year.\u201d<\/p>\n

Dr. Gaspar continued, \u201cIn addition, the $34 million in proceeds from the second closing of our strategic financing ensures we are well-capitalized to progress U.S. launch preparations, continue investing in initiatives aimed at accelerating commercial growth in Europe, and advance our next-in-line neurometabolic programs in MPS disorders. The next 12 months have the potential to provide Orchard Therapeutics several breakout opportunities as we work to cement our leadership position in the HSC gene therapy field.\u201d<\/p>\n

\n BLA Submission Completed for OTL-200 in the U.S.<\/strong>\n <\/p>\n

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, in children with early-onset metachromatic leukodystrophy (MLD). OTL-200 previously received both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. 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.<\/p>\n

OTL-200 is approved as Libmeldy\u00ae<\/sup> (atidarsagene autotemcel) by the European Commission (EC) and Medicines and Healthcare products Regulatory Agency (MHRA). The clinical development program for Libmeldy comprises data spanning 39 children and encompassing more than 10 years follow-up in the earliest treated patients.<\/p>\n

\n Second Closing of Strategic Financing Resulted in $34.0M of New Capital<\/strong>\n <\/p>\n

In June 2023, the company completed the second closing<\/a> of its previously announced strategic financing, resulting in $34.0 million of new capital before placement agent and transaction fees, bringing the total raised to $68.0 million. These securities were sold at a significant premium to the company\u2019s share price on the date of issuance. Orchard Therapeutics expects funding from the first and second closing to provide cash runway to mid-2025.<\/p>\n

The company could bring in up to an additional $120 million in proceeds in 2024 at a price of $11.00 per American Depositary Share (ADS) following potential U.S. approval of OTL-200 if all warrants sold as part of the securities purchase agreement are exercised by participating investors.<\/p>\n

\n Libmeldy Commercial Momentum and Newborn Screening Updates<\/strong>\n <\/p>\n