Celularity Submits Request to U.S. FDA for Orphan Drug Designation for PDA-002 Asset Treating Facioscapulohumeral Muscular Dystrophy
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The submission for orphan drug designation by Celularity Inc. for its cell therapy PDA-002 represents a strategic move in the biotechnology and pharmaceutical industry. The orphan drug status is significant because it provides companies with certain benefits, including market exclusivity, tax credits and assistance with clinical research. The designation is particularly important for drugs targeting rare diseases, such as Facioscapulohumeral Muscular Dystrophy (FSHD), which affects a relatively small patient population. Given the lack of current treatments for FSHD, the development of PDA-002 could fill a substantial unmet medical need, potentially leading to improved patient outcomes and quality of life.
From an investment perspective, the progression of PDA-002 into clinical trials could be a positive indicator for Celularity's future revenue streams and market positioning. However, investors should be aware of the inherent risks associated with clinical development, particularly in the early phases. The success of PDA-002 in clinical trials will be pivotal for Celularity's valuation and investor confidence. It's also noteworthy that the company is looking to expand the application of its cell therapy platform to other neurodegenerative disorders, which could further diversify its portfolio and increase its market potential.
The biotech sector is driven by innovation and the potential for breakthrough therapies. Celularity's announcement concerning the FDA's IND clearance and the upcoming phase 1/2 study for PDA-002 places the company at an important juncture. The success of this study could catalyze further interest and investment in regenerative medicine, particularly in allogeneic cell therapies derived from placental cells. As a market analyst, observing the company's trajectory, the implications for the stock could be substantial if the therapy demonstrates efficacy and safety. This would not only benefit Celularity but could also have a ripple effect across the sector, encouraging investment in similar regenerative approaches.
Additionally, the potential for PDA-002 to address other types of muscular dystrophy could broaden the therapy's impact and market size. This multiplicity of applications is a key factor that could drive the company's long-term growth. Stakeholders should monitor the development of PDA-002 closely, as positive clinical data could lead to significant stock appreciation, while any setbacks might have the opposite effect.
The pursuit of orphan drug designation by Celularity for its PDA-002 therapy underscores the economic incentives in place to encourage the development of treatments for rare diseases. As a healthcare economist, it's important to highlight that these incentives are designed to offset the high costs and lower market returns associated with serving smaller patient populations. The societal benefit of developing treatments for conditions like FSHD, which currently lack effective therapies, is substantial, as it addresses both direct healthcare costs and indirect costs, such as loss of productivity and caregiver burden.
In the long run, if PDA-002 is successful, it could lead to significant cost savings for the healthcare system by reducing the need for supportive care and delaying disease progression. However, the pricing of orphan drugs is often a topic of debate, given the high costs typically associated with them. It is essential to balance the financial sustainability of healthcare systems with the need to incentivize the development of rare disease therapies. The impact of PDA-002's pricing strategy on insurance coverage and patient access will be important factors in its overall success and adoption.
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) -- Celularity Inc. (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 200,000 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 one in 8,000 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 24 percent 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
Celularity Inc. (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.
Carlos.ramirez@celularity.com
Media Contacts:
Ali Nagy / Patrick Maddox
KCSA Strategic Communications
anagy@kcsa.com / pmaddox@kcsa.com
FAQ
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