SILVER
SPRING, Md., Aug. 2, 2024
/PRNewswire/ -- Today, the U.S. Food and Drug Administration
approved Tecelra (afamitresgene autoleucel), a gene therapy
indicated for the treatment of adults with unresectable or
metastatic synovial sarcoma who have received prior chemotherapy,
are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P
positive, and whose tumor expresses the MAGE-A4 antigen as
determined by FDA authorized companion diagnostic devices.
Synovial sarcoma is a rare form of cancer in which malignant
cells develop and form a tumor in soft tissues of the body. This
type of cancer can occur in many parts of the body, most commonly
developing in the extremities. The cancerous cells may also spread
to other parts of the body. Each year, synovial sarcoma impacts
about 1,000 people in the U.S. and most often occurs in adult males
in their 30s or younger. Treatment typically involves surgery to
remove the tumor and may also include radiotherapy and/or
chemotherapy if the tumor is larger, returns after being removed or
has spread beyond its original location.
"Potentially life-threatening cancers such as synovial
sarcoma continue to have a devastating impact on individuals,
especially those for whom standard treatments have limited efficacy
due to tumor growth and progression," said Peter Marks, M.D., Ph.D., director of the FDA's
Center for Biologics Evaluation and Research (CBER). "The approval
of this state-of-the-art immunotherapy technology provides a
critical new option for a patient population in need and
demonstrates the FDA's dedication to the advancement of beneficial
cancer treatments."
Tecelra is also the first FDA-approved T cell receptor (TCR)
gene therapy. The product is an autologous T cell immunotherapy
composed of a patient's own T cells. T cells in Tecelra are
modified to express a TCR that targets MAGE-A4, an antigen
(substance that normally triggers your immune system) expressed by
cancer cells in synovial sarcoma. The product is administered as a
single intravenous dose.
Tecelra was approved using the Accelerated Approval
pathway, under which the FDA may approve drugs for serious or
life-threatening diseases or conditions where there is an unmet
medical need and the drug is shown to have an effect on a surrogate
endpoint that is reasonably likely to predict a clinical benefit to
patients (improving how patients feel or function, or whether they
survive longer). This pathway can allow earlier approval while the
company conducts further trials to verify the predicted clinical
benefit. A confirmatory trial is ongoing to verify Tecelra's
clinical benefit.
"Adults with metastatic synovial sarcoma, a life-threatening
form of cancer, often face limited treatment options in addition to
the risk of cancer spread or recurrence," said Nicole Verdun, M.D., director of the Office of
Therapeutic Products in CBER. "Today's approval represents a
significant milestone in the development of an innovative, safe and
effective therapy for patients with this rare but potentially fatal
disease."
The safety and effectiveness of Tecelra was evaluated in a
multicenter, open-label clinical trial including patients with
inoperable and metastatic synovial sarcoma who had received prior
systemic therapy and whose tumor expressed the MAGE-A4 tumor
antigen. Effectiveness was evaluated based on overall response rate
and the duration of response to treatment with Tecelra. Among the
44 patients in the trial who received Tecelra, the overall response
rate was 43.2% and the median duration of response was six
months.
The most common adverse reactions associated with Tecelra
included nausea, vomiting, fatigue, infections, fever,
constipation, dyspnea (shortness of breath), abdominal pain,
non-cardiac chest pain, decreased appetite, tachycardia (abnormally
fast heart rate), back pain, hypotension, diarrhea and edema
(swelling due to buildup of fluid in body tissues).
Patients treated with Tecelra may experience cytokine release
syndrome (CRS), which is a dangerous type of aggressive immune
system response, including potentially life-threatening reactions.
CRS was observed following administration of Tecelra during
clinical trials. A Boxed Warning is included in the label
containing information about this risk.
Patients may also exhibit Immune Effector Cell-associated
Neurotoxicity Syndrome (ICANS), an immune system-related syndrome
that can occur following some immunotherapies, infections,
secondary malignancies, or hypersensitivity reactions, and severe
cytopenia (an abnormally low level of blood cells) for several
weeks following lymphodepleting chemotherapy and Tecelra infusion.
Patients receiving this product should be monitored for signs and
symptoms of infection and are advised not to drive or engage in
hazardous occupations or activities for at least four weeks after
receiving Tecelra.
The FDA granted Tecelra Orphan Drug, Regenerative Medicine
Advanced Therapy and Priority Review designations for this
indication.
This application was reviewed using a coordinated, cross-agency
approach, including CBER, the FDA's Oncology Center of Excellence
and the Center for Devices and Radiological Health.
The FDA granted the approval of Tecelra to Adaptimmune, LLC.
Media Contact: Carly
(Kempler) Pflaum, (240) 672-8872
Consumer Inquiries: Email or 888-INFO-FDA
The FDA, an agency within the U.S. Department
of Health and Human Services, protects the public health by
assuring the safety, effectiveness, and security of human and
veterinary drugs, vaccines and other biological products for human
use, and medical devices. The agency also is responsible for the
safety and security of our nation's food supply, cosmetics, dietary
supplements, radiation-emitting electronic products, and for
regulating tobacco products.
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SOURCE U.S. Food and Drug Administration