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Orchard (ORTX) Gets FDA's RMAT Status for Gene Therapy in MLD

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Orchard Therapeutics plc (ORTX - Free Report) announced that the FDA has granted Regenerative Medicine Advanced Therapy (“RMAT”) designation to its investigational ex-vivo autologous hematopoietic stem cell gene therapy, OTL-200, for the treatment of early onset metachromatic leukodystrophy (“MLD”), a rare and life-threatening neurodegenerative condition.

The RMAT designation was created under the 21st Century Cures Act and is granted to speed up the development and review of regenerative therapies that target serious or life-threatening conditions.

Per the press release, the RMAT tag was based on data from a study which evaluated 39 patients, including 9 from the United States who were treated with OTL-200 as part of clinical studies and compassionate use programs conducted at the San Raffaele-Telethon Institute for Gene Therapy in Italy.

The company plans to interact with the FDA authorities by the middle of this year to decide the regulatory path forward for OTL-200 and file a biologics license application in the United States.

Shares of Orchard Therapeutics were up 12.5% on Thursday following the announcement of the news. However, the stock has plunged 52.6% in the past year against the industry’s increase of 8.2%.

price chart for ORTX


We note that in November 2020, the FDA cleared Orchard Therapeutics’ investigational new drug application for OTL-200 to treat MLD.

Last month, the European Commission granted market authorization to OTL-200 for the treatment of MLD. The drug is marketed under the trade name Libmeldy and is the first therapy to be approved for eligible patients with early-onset MLD.

Libmeldy is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy based in Italy.

Zacks Rank & Stocks to Consider

Orchard Therapeutics currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Novavax, Inc. (NVAX - Free Report) , Blueprint Medicines Corporation (BPMC - Free Report) and Arcus Biosciences, Inc. (RCUS - Free Report) , all carrying a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Novavax’s earnings estimates have been revised 30.7% upward for 2021 over the past 60 days. The stock has skyrocketed 2619.2% in the past year

Blueprint Medicines’ loss per share estimates have narrowed 1.6% for 2021 over the past 60 days. The stock has rallied 26.6% in the past year.

Arcus Biosciences’ loss per share estimates have narrowed 5.5% for 2021 over the past 60 days. The stock has rallied 271.2% in the past year.

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