Vericel Corporation (NASDAQ:VCEL), a leading developer of
patient-specific expanded cellular therapies for the treatment of
severe diseases and conditions, today announced that the U.S. Food
and Drug Administration has approved the Company’s Humanitarian
Device Exemption (HDE) supplement for Epicel® (cultured epidermal
autografts) to revise the labeled indications of use to
specifically include pediatric patients and to add pediatric
labeling. The revised product label also specifies that the
probable benefit of Epicel, mainly related to survival, was
demonstrated in two Epicel clinical experience databases and a
randomized, controlled, independent physician-sponsored study
comparing outcomes in patients with severe burns treated with
Epicel and standard care compared to standard care alone.
“We are very pleased with the FDA’s decision to expand the
labeled indications for use of Epicel to reflect the experience of
pediatric patients with severe burns who have been treated
successfully with Epicel,” said David Recker, M.D., chief medical
officer of Vericel. “We believe that the revised label
provides valuable information describing the safety and clinical
use of Epicel in this vulnerable pediatric patient population, as
well as the probable survival benefit for adult and pediatric
patients with life-threatening injuries resulting from severe
burns.”
Data from a clinical database used to support the original
Epicel HDE application, which included 552 patients treated with
Epicel from 1989 to 1996 (mean TBSA burns of 68.6%), including 205
pediatric patients aged 21 years and younger, demonstrated a
survival rate of 86.6% for all patients and 89.3% for pediatric
patients at three months post-initial surgery. Data from the
Epicel Medical Device Tracker, a post-approval registry of 402
patients treated with Epicel from October 2007 to June 2015 (mean
TBSA burns of 66.0%), including 120 pediatric patients,
demonstrated a survival rate of 81.3% for all patients and 88.3%
for pediatric patients.
Data from the randomized, controlled, independent,
physician-sponsored study of severe burn patients followed over a
seven-year period demonstrated a survival rate of 90% for patients
treated with Epicel (n=20, mean TBSA burns of 69.1%) and standard
care, meaning excision plus allografting and/or split-thickness
autografting, compared to a survival rate of 37.5% for patients in
the control group (n=24, mean TBSA burns of 62.9%) who received
standard care alone.
About EpicelEpicel® (cultured epidermal
autografts) is a permanent skin replacement indicated for use in
adult and pediatric patients who have deep dermal or full thickness
burns comprising a total body surface area greater than or equal to
30%. Epicel may be used in conjunction with split-thickness
autografts or alone in patients for whom split-thickness autografts
may not be an option due to the severity and extent of their
burns. The probable benefit of Epicel, mainly related to
survival, was demonstrated in two Epicel databases and one
physician-sponsored study. Epicel has been used in the United
States and internationally to treat severely burned patients since
1988, and was approved in the United States in 2007 as a
Humanitarian Use Device (HUD) under a Humanitarian Device Exemption
(HDE).
Important Safety InformationEpicel is
contraindicated in patients with known hypersensitivity to
vancomycin, amikacin, or amphotericin. Epicel should not be
used in patients with sensitivities to materials of bovine or
murine origin. Epicel is contraindicated for use on
clinically infected wounds.
Because Epicel is manufactured with and contains residual
amounts of murine cells, the FDA considers it a xenotransplantation
product. Therefore, recipients should not donate whole blood,
blood components, source plasma, source leukocytes, tissue, breast
milk, ova, sperm or other body parts for use in humans because
there is a potential risk of carrying an infection that is
transmitted from mouse cells to humans. In addition, the risk
of disease transmission from Epicel is unknown.
Squamous cell carcinoma (SCC) has been reported in patients with
burn injury after being grafted with Epicel. The most common
adverse reactions, occurring in ≥ 2% of patients were infection,
graft shear, blister, drainage, sepsis, graft detachment and renal
failure.
Patient information supplied by treating physicians and
attending burn teams from 1989 to 1996 included 552 patients, 205
children (age 21 years and younger) and 347 adults reported death
(13%) and the adverse reactions of highest incidence as: infection
(13.8%), graft shear (7.8%), blister (4.2%) and drainage
(3.3%).
From June 1998 through September, 2015, over 1,662 patients,
including 589 children (age 21 and younger) and 1,073 adults were
tracked through spontaneous reports via medical device reports,
reports from burn sites and published literature. Adverse
reactions were similar to the previously identified adverse
reactions. Events that were reported in ≥ 2% of patients
included death (8.8%), and adverse reactions of multi-organ
failure, sepsis, infection and graft procedure complications.
Because of the potential underreporting of adverse reactions from
these sources, the percentages of adverse reactions should be
interpreted with caution.
Epicel is intended solely for autologous use.
The effectiveness of Epicel has not been proven in clinical
studies. The long-term safety of Epicel is unknown. The
safety of Epicel has not been studied in pregnant and nursing
women.
About Humanitarian Use Devices and the Humanitarian
Device ExemptionHumanitarian Use Devices (HUD) are medical
devices intended to benefit patients in the treatment or diagnosis
of diseases or conditions that affect fewer than 4,000 individuals
in the United States per year. Devices that receive HUD
designation from the Office of Orphan Products Development of the
FDA may be eligible for marketing approval under a Humanitarian
Device Exemption (HDE) application based, among other criteria, on
evidence of safety and probable benefit to health from use of the
device. FDA approval of an HDE application authorizes the
applicant to market the device, subject to certain profit and use
restrictions.
Except in certain circumstances, HUDs approved under an HDE
cannot be sold for an amount that exceeds the costs of research and
development, fabrication, and distribution of the device (i.e., for
profit). A HUD is eligible to be sold for profit after
receiving HDE approval if the device meets certain eligibility
criteria, including where the device is intended for the treatment
of a disease or condition that occurs in pediatric patients and
such device is labeled for use in pediatric patients.
Based on the approval of this HDE supplement, Epicel is
permitted to be sold for profit as long as the number of devices
distributed in any calendar year does not exceed its annual
distribution number (ADN), which is defined as the number of
devices reasonably needed to treat a population of 4,000
individuals per year in the United States. The FDA has
determined that the ADN for Epicel is 360,400.
About Vericel CorporationVericel Corporation is
a leader in developing patient-specific expanded cellular therapies
for use in the treatment of patients with severe diseases and
conditions. The company markets two autologous cell therapy
products in the U.S.: Carticel® (autologous cultured chondrocytes),
an autologous chondrocyte implant for the treatment of cartilage
defects in the knee, and Epicel® (cultured epidermal autografts), a
permanent skin replacement for the treatment of patients with
deep-dermal or full-thickness burns comprising greater than or
equal to 30% of total body surface area. Vericel is also
developing MACI™, a third-generation autologous chondrocyte implant
for the treatment of cartilage defects in the knee, and
ixmyelocel-T, a patient-specific multicellular therapy for the
treatment of advanced heart failure due to ischemic dilated
cardiomyopathy. For more information, please visit the
company’s website at www.vcel.com.
Epicel® and Carticel® are registered trademarks and MACI™ is a
trademark of Vericel Corporation. © 2016 Vericel
Corporation. All rights reserved.
This document contains forward-looking statements, including,
without limitation, statements concerning anticipated progress,
objectives and expectations regarding the commercial potential of
our products, clinical activity timing and regulatory pathway and
timing, and objectives and expectations regarding our company
described herein, all of which involve certain risks and
uncertainties. These statements are often, but are not always, made
through the use of words or phrases such as "anticipates,"
"intends," "estimates," "plans," "expects," "we believe," "we
intend," and similar words or phrases, or future or conditional
verbs such as "will," "would," "should," "potential," “can
continue,” "could," "may," or similar expressions. Actual results
may differ significantly from the expectations contained in the
forward-looking statements. Among the factors that may result in
differences are the inherent uncertainties associated with
competitive developments, clinical trial and product development
activities, regulatory approval requirements, the availability and
allocation of resources among different potential uses, estimating
the commercial potential of our products and product candidates,
market demand for our products, and our ability to supply or meet
customer demand for our products. These and other significant
factors are discussed in greater detail in Vericel’s Annual Report
on Form 10-K for the year ended December 31, 2014, filed with the
Securities and Exchange Commission ("SEC") on March 25, 2015,
Quarterly Reports on Form 10-Q and other filings with the SEC.
These forward-looking statements reflect management's current views
and Vericel does not undertake to update any of these
forward-looking statements to reflect a change in its views or
events or circumstances that occur after the date of this release
except as required by law.
CONTACT:
Chad Rubin
The Trout Group
crubin@troutgroup.com
(646) 378-2947
or
Lee Stern
The Trout Group
lstern@troutgroup.com
(646) 378-2922
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