Celularity Submits Request to U.S. FDA for Orphan Drug Designation for PDA-002 Asset Treating Facioscapulohumeral Muscular Dystrophy
Company Has Already Received IND Clearance from the FDA and Plans to Begin Phase 1/2 Study in 2024 with Off-the-Shelf, Placental-Derived Cell Therapy
FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).
Orphan drug designation is a status given to drugs that show the potential to treat, prevent or diagnose serious or life-threatening diseases that affect fewer than people in the U.S. Earlier, Celularity received FDA Investigational New Drug Application clearance for PDA-002 in FSHD and plans to commence a phase 1/2 study in the second half of 2024. The trial serves as an important component for submitting a Biologics License Application (BLA) to the FDA in the future as a potential treatment for FSHD and other types of muscular dystrophy.
Affecting approximately people, FSHD is one of the most common types of muscular dystrophy, characterized by severe progressive, asymmetrical muscle atrophy that affects facial, upper and lower limb, and shoulder skeletal muscles. There is currently no cure or disease-modifying treatments for FSHD. The disease can cause significant lifetime morbidity, with approximately of patients at risk of wheelchair use within six years of their diagnosis.
“Celularity is committed to developing best-in-class therapies for patients who suffer from challenging diseases who have few, if any, currently available treatment options,” said Robert Hariri, M.D., Ph.D., Celularity CEO, Chairman and Founder. “Having already received IND clearance from the FDA, we intend to leverage our cell therapy platform to improve outcomes for people living with FSHD and potentially other neurodegenerative disorders and believe these cellular immunotherapies have breakthrough potential. We look forward to commencing our Phase 1/2 study as we continue to push forward our extensive cell therapy portfolio, including T-cells, natural killer (NK) cells, mesenchymal stem cells (MSCs), and exosomes.”
About Celularity
(NASDAQ: CELU) is a cell therapy and regenerative medicine company developing allogeneic, cryopreserved, placental-derived cell therapies, including therapeutic programs using mesenchymal-like adherent stromal cells (MLASCs), T-cells engineered with CAR (CAR T-cells) and genetically modified and unmodified natural killer (NK) cells. These therapeutic programs target aging-related diseases, including degenerative diseases, cancer, and immune disorders. In addition, Celularity develops, manufactures, and commercializes advanced biomaterial products also derived from the postpartum placenta. Celularity believes that, by harnessing the placenta’s unique biology and ready availability, it can develop therapeutic solutions that address significant unmet global needs for effective, accessible and affordable therapies.
Forward-Looking Statements
This press release includes “forward-looking statements” within the meaning of The U.S. Private Securities Litigation Reform Act of 1995, as well as within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “can,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “intends,” “may,” “might,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “seek,” “should,” “strive,” “target,” “will,” “would” and the negative of terms like these or other comparable terminology, and other words or terms of similar meaning. The forward-looking statements in this press release include express or implied statements regarding the ability of Celularity to obtain orphan drug designation for PDA-002 for the treatment of FSHD, commence a Phase 1/2 clinical trial in FSHD, as well as to advance our pipeline to address other neurodegenerative diseases, among others. Many factors could cause actual results to differ materially from those described in these forward-looking statements, including but not limited to: Celularity’s liquidity situation; the volatility in Celularity’s stock price; inherent risks in biotechnological development, including with respect to the development of novel cellular therapies; and the clinical trial and regulatory approval process; along with those risk factors set forth under the caption “Risk Factors” in Celularity’s annual report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 31, 2023, and other filings with the SEC. If any of these risks materialize or underlying assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that Celularity does not presently know, or that Celularity currently believes are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, these forward-looking statements reflect Celularity’s current expectations, plans, or forecasts of future events and views as of the date of this communication. Subsequent events and developments could cause assessments to change. Accordingly, forward-looking statements should not be relied upon as representing Celularity’s views as of any subsequent date, and Celularity undertakes no obligation to update forward-looking statements to reflect events or circumstances after the date hereof, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.
Investor Contact:
Carlos Ramirez
Senior Vice President, Celularity Inc.
Media Contacts:
Ali Nagy / Patrick Maddox
KCSA Strategic Communications
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