- The Mikaela Naylon Give Kids a Chance Act reauthorizes the Pediatric Priority Review Voucher (PPRV) Program and extends eligibility for medicines achieving U.S. FDA approval before September 2029
- The Pediatric Priority Review Voucher program encourages the development of treatments for rare pediatric diseases
- U.S. FDA has awarded Longeveron’s laromestrocel Hypoplastic Left Heart Syndrome (HLHS) program Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation
- With the extension of the PPRV Program, Longeveron may be granted a Priority Review Voucher if laromestrocel’s Biological License Application (BLA) is approved by the FDA before September 2029
MIAMI, Feb. 05, 2026 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapy for life-threatening, rare pediatric and chronic aging-related conditions, supports and applauds Congress passing the Mikaela Naylon Give Kids a Chance Act (H.R. 1262).
The Mikaela Naylon Give Kids a Chance Act reauthorizes the Rare Pediatric Disease Priority Review Voucher Program, which encourages the development of treatments for rare pediatric diseases. Companies pursuing treatments for rare pediatric diseases can receive a priority review voucher upon FDA approval of a therapy, and then either use it to secure a speedier FDA review of a future therapy or sell it to another company. Since August 2024, vouchers have been sold for $150-200 million each. Since the program’s inception, more than 60 PRVs have been awarded for 40 pediatric diseases, all at no cost to taxpayers.
“We are extremely gratified by the renewal of the PRV program given the tremendous incentives it has brought to bear in the past to enhance the approval of treatments for rare pediatric diseases, a category of diseases with enormous unmet medical need,” said Dr. Joshua Hare, Chief Science Officer and co-founder of Longeveron. “We are at the late stages of clinical development of laromestrocel for children with HLHS, a very severe congenital heart condition, in which only 50% of children survive past the age of 15. The chance to increase the survival of these children is our primary focus, and we welcome any initiative that could enhance our development program.”
Longeveron is evaluating its stem cell therapy laromestrocel (Lomecel-B™) as a potential adjunct therapy for Hypoplastic Left Heart Syndrome (HLHS), a rare pediatric congenital heart birth defect in which the left ventricle (one of the pumping chambers of the heart) is either severely underdeveloped or missing. Even with current standard of care surgeries, only 50% of infants survive to adolescence due to right ventricular failure.
ELPIS II is Longeveron’s Phase 2b clinical trial evaluating laromestrocel for HLHS. The clinical trial enrolled 40 pediatric patients at twelve premiere infant and children’s treatment institutions across the country. Top-line trial results are anticipated in the third quarter of 2026. If ELPIS II results are positive, the Company would plan to submit a Biological License Application (BLA) for full traditional approval for laromestrocel for HLHS. With the reauthorization approved by Congress this week, the Company will now remain eligible to receive a PRV upon BLA approval through September 30, 2029. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH).
About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a congenital birth defect in which the left ventricle (one of the pumping chambers of the heart) is either severely underdeveloped or missing. As a consequence, infants born with this condition have severely diminished systemic blood flow, which requires them to undergo a complex, three stage heart reconstruction surgery process over the course of the first 5 years of their lives. Although surgical advances have enabled survival into adulthood for some patients, early mortality remains substantial in this population, due to right ventricular failure. As such, there remains an important unmet medical need to improve right ventricular function in these patients in order to support both short-term and long-term outcomes.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company’s lead investigational product is laromestrocel (LOMECEL-B®), an allogeneic mesenchymal stem cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Laromestrocel has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease (AD), and Pediatric Dilated Cardiomyopathy (DCM). Laromestrocel development programs have received five distinct and important FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program - Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn, X, and Instagram.
Forward-Looking Statements
Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management’s current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve known and unknown risks, uncertainties, and other important factors that could cause actual results, performance, or achievements to differ materially from those anticipated, expressed, or implied by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expects,” “intend,” “looks to,” “may,” “on condition,” “plan,” “potential,” “predict,” “preliminary,” “project,” “see,” “should,” “target,” “will,” “would,” or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, our cash position and need to raise additional capital, the difficulties we may face in obtaining access to capital, and the dilutive impact it may have on our investors; our financial performance, and ability to continue as a going concern; the period over which we estimate our existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; the ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and focus of our ongoing and future preclinical studies and clinical trials, and the reporting of data from those studies and trials; the size of the market opportunity for certain of our product candidates, including our estimates of the number of patients who suffer from the diseases we are targeting; our ability to scale production and commercialize the product candidate for certain indications; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our ability to obtain and maintain regulatory approval of our product candidates in the U.S. and other jurisdictions; our plans relating to the further development of our product candidates, including additional disease states or indications we may pursue; our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and our ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and our ability to attract and retain such personnel; and our estimates regarding expenses, future revenue, capital requirements and needs for additional financing.
Further information relating to factors that may impact the Company’s results and forward-looking statements are disclosed in the Company’s filings with the Securities and Exchange Commission, including Longeveron’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 28, 2025, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The Company operates in highly competitive and rapidly changing environment; therefore, new factors may arise, and it is not possible for the Company’s management to predict all such factors that may arise nor assess the impact of such factors or the extent to which any individual factor or combination thereof, may cause results to differ materially from those contained in any forward-looking statements. The forward-looking statements contained in this press release are made as of the date of this press release based on information available as of the date of this press release, are inherently uncertain, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.
Investor and Media Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com
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