Longeveron Granted Orphan Drug Designation by FDA for Lomecel-B to Treat Infants with Hypoplastic Left Heart Syndrome (HLHS)
December 06 2021 - 8:00AM
Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a
clinical stage biotechnology company developing cellular therapies
for chronic aging-related and certain life-threatening conditions,
announced today that the U.S. Food and Drug Administration (FDA)
has granted Orphan Drug Designation (ODD) for Lomecel-B for the
treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and
life-threatening congenital heart defect in infants.
ODD is intended to assist and encourage
companies to develop safe and effective therapies for the treatment
of rare diseases or conditions. ODD positions Longeveron to be able
to potentially leverage a range of financial and regulatory
benefits, including government grants for conducting clinical
trials, waiver of FDA user fees for the potential submission of a
marketing application, and certain tax credits. Receiving ODD may
also result in the product receiving seven years market exclusivity
upon approval for use in the rare disease or condition for which
the product was designated if all of the statutory and regulatory
requirements are met.
“Adding to the Rare Pediatric Disease (RPD)
designation already granted to Lomecel-B for treatment of HLHS, the
FDA’s decision to grant ODD to Lomecel-B for this indication
indicates the ongoing and unmet need for new therapies to treat
infants with HLHS,” said Geoff Green, Chief Executive Officer at
Longeveron. “Building on results from our completed Phase 1
safety-focused trial, we believe Lomecel-B has potential to improve
outcomes for these severely impacted infants by way of repairing
cardiac tissue and improving ventricular function. The combination
of both RPD and ODD allows us to potentially move more efficiently
through clinical development and regulatory review, and Lomecel-B
may be eligible for a period of marketing exclusivity upon approval
for this indication.”
Approximately 1,000 babies are born with HLHS
each year in the U.S. HLHS babies have an underdeveloped left
ventricle, which impairs the heart’s ability to pump blood
throughout the body. HLHS is often fatal without surgical
intervention, in which three surgical procedures are performed over
the period of about 5 years, to allow the right ventricle to be
configured to pump blood to the body. Longeveron is evaluating the
safety of Lomecel-B injection into the right ventricle during the
second surgery (4 – 6 months of age), and the effect on cardiac
function and other health status endpoints. Longeveron recently
reported clinical results from its safety-focused Phase 1 clinical
study of Lomecel-B in HLHS patients. When cardiac surgeons injected
Lomecel-B directly into the babies’ hearts at the time of the
second surgery, the cells were well tolerated with no major adverse
cardiac events and no infections considered to be related to the
investigational treatment. One hundred percent of the babies
enrolled in the Phase 1 trial (n=10) were alive and had not
required a transplant between 2-3.5 years post-surgery. Other
measurements of the babies’ health, such as weight gain and growth
pattern, matched that of normal healthy babies. Longeveron is
currently enrolling ELPIS II, a 38-subject, Phase 2 randomized,
double-blind, controlled clinical trial examining the effect of
Lomecel-B in HLHS affected infants. ELPIS II is being funded in
part by a grant from the National Institute of Health’s National
Heart, Lung, and Blood Institute (NHLBI; Grant number
1UG3HL148318), in collaboration with Longeveron, and is led by
Principal Investigator Sunjay Kaushal, MD, PhD, Division Head,
Cardiovascular-Thoracic Surgery, Ann and Robert H. Lurie Children’s
Hospital of Chicago.
“We are gratified to receive both rare pediatric
disease and orphan drug designations from FDA for this clinical
trial program,” stated Sunjay Kaushal, M.D, Ph.D. study Principal
Investigator and Division Head of Cardiovascular Thoracic Surgery
at Lurie Children’s Hospital of Chicago. “This is a severe
condition and a significant unmet medical need, and these important
designations will be vital to facilitating a potentially more rapid
development program. My colleagues around the country and I are
extremely excited about the progress of the current trial, and we
look forward to seeing this program move through the regulatory and
clinical pathway.”
About Longeveron Inc.
Longeveron is a clinical stage biotechnology
company developing cellular therapies for specific aging-related
and certain life-threatening conditions. The Company’s lead
investigational product is the LOMECEL-B™ cell-based therapy
product (“Lomecel-B”), which is derived from culture-expanded
medicinal signaling cells (MSCs) that are sourced from bone marrow
of young, healthy adult donors. Longeveron believes that by using
the same cells that promote tissue repair, organ maintenance, and
immune system function, it can develop safe and effective therapies
for some of the most difficult disorders associated with the aging
process and other medical disorders. Longeveron is currently
sponsoring Phase 1 and 2 clinical trials in the following
indications: Aging Frailty, Alzheimer’s disease, the Metabolic
Syndrome, Acute Respiratory Distress Syndrome (ARDS), and
hypoplastic left heart syndrome (HLHS). The Company’s mission is to
advance Lomecel-B and other cell-based product candidates into
pivotal Phase 3 trials, with the goal of achieving regulatory
approvals, subsequent commercialization and broad use by the
healthcare community. Additional information about the Company is
available at www.longeveron.com.
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characteristics, safety, efficacy and therapeutic effects of our
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results and forward-looking statements are disclosed in the
Company's filings with the Securities and Exchange Commission,
including our Quarterly Report on Form 10-Q for the period ended
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Contact:Brendan PayneStern Investor
RelationsTel: (212) 362-1200Email: Brendan.payne@sternir.com
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