Vita Therapeutics receives Orphan Drug Designation from FDA for new novel treatment

Jul 23, 2020, 09:00 ET

BALTIMORE, July 23, 2020 /PRNewswire/ -- Vita Therapeutics, Inc. announced today that it received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for VTA-110, a novel regenerative therapy for the treatment of Duchenne's Muscular Dystrophy (DMD).

VTA-110, is a potential first-in-class allogenic iPSC-based therapy that has shown the ability to repair and regenerate healthy muscle in preclinical studies and has the potential to benefit patients with DMD. The company plans to continue to progress this treatment towards clinical trials and plans to bring it forward for other congenital muscular dystrophy diseases as well. This therapy was exclusively licensed from technology originally invented at Johns Hopkins University and the Kennedy Krieger Institute in the labs of Dr. Kathryn Wagner and Dr. Gabsang Lee.

Douglas Falk, M.S., Chief Executive Officer of Vita Therapeutics commented, "We are very pleased the FDA granted orphan drug designation for VTA-110. This represents an important step in the right direction for developing innovative treatments for patients with an incredibly high unmet medical need. We believe VTA-110 has the potential to be a long-term disease-modifying treatment for patients living with DMD and other types of muscular dystrophy."    

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people annually. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA's implementing regulations. Orphan designation qualifies the sponsor of the drug for various development incentives including tax credits for qualified clinical testing and 7 years of marketing exclusivity if regulatory approval is ultimately received.

About DMD
DMD is a lethal, rapidly progressive degenerative neuromuscular disorder caused by mutations in the dystrophin gene that result in absent or insufficient functional dystrophin, a cytoskeletal protein that enables the strength, stability, and functionality of myofibers within muscle. Affected patients can gain strength and motor skills until about 6 years of age. After this stage, progressive deterioration in strength occurs. Individuals with DMD lose ambulation in teenage years and die from cardiac or respiratory failure in early adulthood. There is no cure for DMD, only supportive therapies such as corticosteroids are available.

About Vita Therapeutics
Vita Therapeutics is a cell engineering company that aims to develop life-transformative treatments. It was founded out of Johns Hopkins University in 2019 by Douglas Falk, M.S. and Peter Andersen, PhD. The company utilizes induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defected in patients. Earlier this year the company completed a $2M seed round and received a $300k grant from the Maryland Stem Cell Research Foundation for the development of its lead asset, VTA-110. The company is currently in progress towards completing Series A funding. More information about Vita Therapeutics is available at  

Douglas Falk
CEO, Vita Therapeutics