First Tissue-Engineered Autologous
Cellularized Scaffold Product Approved by the FDA
Vericel Corporation (NASDAQ:VCEL), a leading developer of expanded
autologous cell therapies for the treatment of patients with
serious diseases and conditions, today announced that the U.S. Food
and Drug Administration (FDA) has approved MACI® (autologous
cultured chondrocytes on porcine collagen membrane) for the repair
of symptomatic single or multiple full-thickness cartilage defects
of the knee with or without bone involvement in adults.
“The treatment of articular cartilage defects in
the knee is challenging because articular cartilage in adults has
minimal capacity to repair itself,” said David Recker, MD, chief
medical officer of Vericel. “While orthopedic surgeons have
long understood that autologous chondrocyte implantation can
regenerate cartilage tissue, the previous surgical procedure was
technically complex and time consuming, and the indicated patient
population was limited. MACI is the first product to show a
statistically significantly greater improvement in KOOS pain and
function scores compared to microfracture, a commonly performed
alternative surgical treatment for cartilage repair, in a
well-controlled Phase 3 clinical study. With the introduction
of MACI, orthopedic surgeons will have a simplified treatment
option available for a broader patient population supported by
solid clinical evidence.”
The approval of MACI is based on the SUMMIT study
(Superiority of MACI implant versus Microfracture Treatment in
patients with symptomatic articular cartilage defects in the knee),
a two-year prospective, multicenter, randomized, open-label,
parallel-group study which demonstrated a statistically
significantly (p=0.001) greater improvement in KOOS pain and
function (SRA) scores in the MACI group compared to the
microfracture group at two years. Patients from the two-year
SUMMIT study had the option to enroll in a three-year follow-up
study (extension study). A majority of the patients who
completed the SUMMIT study also participated in the extension
study. Overall efficacy data support a long-term clinical
benefit from the use of MACI in patients with cartilage defects of
the knee.
MACI is the first FDA-approved cellularized scaffold product
that applies tissue engineering processes to grow cells on
scaffolds using healthy cartilage tissue from the patient’s own
knee.
“Bringing an important new therapy to orthopedic surgeons and
patients is a significant milestone for Vericel, and I would like
to thank the FDA for working closely with us to make MACI available
for these patients,” said Nick Colangelo, president and CEO of
Vericel. "We believe that the introduction of MACI, along with
investments to expand our commercial organization and implement new
patient support programs, positions Vericel to generate significant
growth in 2017 and beyond."
Conference Call InformationToday's conference
call will be available live at 9:00am (EST) in the Investors
section of the Vericel website at
http://investors.vcel.com/events.cfm. Please access the site at
least 15 minutes prior to the scheduled start time in order to
download the required audio software if necessary. To participate
in the live call by telephone, please call (877) 312-5881 and
reference Vericel Corporation investor call. If calling from
outside the U.S., please use the international phone number (253)
237-1173. If you are unable to participate in the live call, the
webcast will be available until December 13, 2017. A replay of the
call will also be available until 12:00pm (EDT) on December 18,
2016 by calling (855) 859-2056, or from outside the U.S. (404)
537-3406. The conference ID is 39558058.
About MACIMACI (autologous cultured
chondrocytes on porcine collagen membrane) is an autologous
cellular scaffold product that is indicated for the repair of
symptomatic single or multiple full-thickness cartilage defects of
the knee with or without bone involvement in adults. The MACI
implant consists of autologous cultured chondrocytes seeded onto a
resorbable Type I/III collagen membrane. Autologous cultured
chondrocytes are human-derived cells which are obtained from the
patient's own cartilage for the manufacture of MACI.
Clinical DataThe FDA approval is supported by
the results of SUMMIT trial1, a Phase 3 two‑year, prospective,
multicenter, randomized, open-label, parallel-group study that
enrolled a total of 144 patients, ages 18 to
54 years, with at least one symptomatic Outerbridge
Grade III or IV focal cartilage defect on the medial
femoral condyle, lateral femoral condyle, and/or the
trochlea. The co-primary efficacy endpoint was change from
baseline to Week 104 for the subject’s Knee injury and
Osteoarthritis Outcome Score (KOOS) in 2 subscales: Pain
and Function (Sports and Recreational Activities [SRA]).2 At
Week 104, KOOS pain and function (SRA) had improved from baseline
in both treatment groups, but the improvement was statistically
significantly (p<0.001) greater in the MACI group compared with
the microfracture group. In a responder analysis, the
proportion of subjects with at least a 10‑point improvement in both
KOOS pain and function (SRA) was greater in the MACI® group
(63/72 = 87.5%; 95% CI [77.6%, 94.6%]) compared with the
microfracture group (49/72 = 68.1%; 95% CI [56.0%,
78.6%]).
The most frequently occurring adverse reactions (≥5%) reported
for MACI® in the 2‑year randomized, controlled clinical trial were
arthralgia, tendonitis, back pain, joint swelling, and joint
effusion. Serious adverse reactions reported for MACI were
arthralgia, cartilage injury, meniscus injury, treatment failure,
and osteoarthritis.
Important Safety Information
- MACI is contraindicated in patients with a known history of
hypersensitivity to gentamicin, other aminoglycosides, or products
of porcine or bovine origin. MACI is also contraindicated for
patients with severe osteoarthritis of the knee, inflammatory
arthritis, inflammatory joint disease, or uncorrected congenital
blood coagulation disorders. MACI is also not indicated for use in
patients who have undergone prior knee surgery in the past six
months, excluding surgery to procure a biopsy or a concomitant
procedure to prepare the knee for a MACI implant.
- MACI is contraindicated in patients who are unable to follow a
physician-prescribed post-surgical rehabilitation program.
- The safety of MACI in patients with malignancy in the area of
cartilage biopsy or implant is unknown. Expansion of present
malignant or dysplastic cells during the culturing process or
implantation is possible.
- Patients undergoing procedures associated with MACI are not
routinely tested for transmissible infectious diseases. A cartilage
biopsy and MACI implant may carry the risk of transmitting
infectious diseases to healthcare providers handling the tissue.
Universal precautions should be employed when handling the biopsy
samples and the MACI product.
- Final sterility test results are not available at the time of
shipping. In the case of positive sterility results, health care
provider(s) will be contacted.
- To create a favorable environment for healing, concomitant
pathologies that include meniscal pathology, cruciate ligament
instability and joint misalignment, must be addressed prior to or
concurrent with the implantation of MACI.
- Local treatment guidelines regarding the use of
thromboprophylaxis and antibiotic prophylaxis around orthopaedic
surgery should be followed. Use in patients with local
inflammations or active infections in the bone, joint, and
surrounding soft tissue should be temporarily deferred until
documented recovery.
- The MACI implant is not recommended during pregnancy. For
implantations post-pregnancy, the safety of breast feeding to
infant has not been determined.
- Use of MACI in pediatric patients or patients over 55 years of
age has not been assessed.
- The most frequently occurring adverse reactions reported for
MACI (≥5%) were arthralgia, tendonitis, back pain, joint swelling,
and joint effusion.
- Serious adverse reactions reported for MACI were arthralgia,
cartilage injury, meniscus injury, treatment failure, and
osteoarthritis.
About Articular Cartilage Defects of the
KneeArticular cartilage is a highly organized avascular
tissue composed of chondrocytes embedded within an extracellular
matrix of collagens, proteoglycans and noncollagenous proteins.
Its primary function is to enable the smooth articulation of
joint surfaces, and to cushion compressive, tensile and shearing
forces. Articular cartilage damage is caused by both acute
and repetitive trauma resulting in knee pain, effusion or
mechanical symptoms such as catching and locking, and
swelling. Since articular cartilage is avascular it has
little capacity to repair itself or regenerate. Articular
cartilage lesions that are left untreated may progress to
debilitating joint pain, dysfunction, and osteoarthritis3.
The prevalence rate for cartilage lesions in the knee has been
reported to be 63% in patients undergoing investigational
arthroscopies4.
About Vericel CorporationVericel develops,
manufactures, and markets expanded autologous cell therapies for
the treatment of patients with serious diseases and
conditions. The company currently markets two cell therapy
products in the United States. Carticel® (autologous cultured
chondrocytes) is an autologous chondrocyte implant for the
treatment of cartilage defects in the knee in patients who have had
an inadequate response to a prior arthroscopic or other surgical
repair procedure. Epicel® (cultured epidermal autografts) is
a permanent skin replacement for the treatment of patients with
deep dermal or full thickness burns greater than or equal to 30% of
total body surface area. Vericel also plans to
market MACI® (autologous cultured chondrocytes on porcine
collagen membrane), an autologous cellularized scaffold product
indicated for the repair of symptomatic, single or multiple
full-thickness cartilage defects of the knee with or without bone
involvement in adults, which has just been approved by the
FDA. Vericel is also developing ixmyelocel‑T, an autologous
multicellular therapy intended to treat advanced heart failure due
to ischemic dilated cardiomyopathy (DCM). For more
information, please visit the company's website at
www.vcel.com.
Epicel®, Carticel®, and MACI® are registered trademarks 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
MACI® and our other products, 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, 2015, filed with the Securities and Exchange
Commission ("SEC") on March 14, 2016, 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.
References
1Saris D, Price A, Widuchowski W, Bertrand-Marchand M, Caron J,
Drogset JO, et al. Matrix-applied characterized autologous cultured
chondrocytes versus microfracture: two-year follow-up of a
prospective randomized trial. Am J Sports Med. 2014
Jun;42(6):1384-94.
2Roos EM, Lohmander LS. The Knee injury and Osteoarthritis
Outcome Score (KOOS): from joint injury to osteoarthritis. Health
Qual Life Outcomes. 2003;1:64.
3Bedi A, Feeley BT, Williams RJ. Management of articular
cartilage defects of the knee. J Bone Joint Surg Am.
2010;92(4):994‑1009.
4Curl WW, Krome J, Gordon ES, Rushing J, Smith BP, Poehling GG.
Cartilage injuries: a review of 31,516 knee arthroscopies.
Arthroscopy. 1997;13(4):456-60.
CONTACT:
Chad Rubin
The Trout Group crubin@troutgroup.com
(646) 378-2947
or
Lee Stern
The Trout Group lstern@troutgroup.com
(646) 378-2922
Vericel (NASDAQ:VCEL)
Historical Stock Chart
From Apr 2024 to May 2024
Vericel (NASDAQ:VCEL)
Historical Stock Chart
From May 2023 to May 2024