SILVER SPRING, Md.,
Nov. 20, 2018 /PRNewswire/ -- The
U.S. Food and Drug Administration today approved Gamifant
(emapalumab) for the treatment of pediatric (newborn and above) and
adult patients with primary hemophagocytic lymphohistiocytosis
(HLH) who have refractory, recurrent or progressive disease or
intolerance with conventional HLH therapy. This FDA approval is the
first for a drug specifically for HLH.
"Primary HLH is a rare and life-threatening condition typically
affecting children and this approval fills an unmet medical need
for these patients," said Richard
Pazdur, M.D., director of the FDA's Oncology Center of
Excellence and acting director of the Office of Hematology and
Oncology Products in the FDA's Center for Drug Evaluation and
Research. "We are committed to continuing to expedite the
development and review of therapies that offer meaningful treatment
options for patients with rare conditions."
HLH is a condition in which the body's immune cells do not work
properly. The cells become overactive releasing molecules, which
leads to inflammation. The immune cells start to damage the body's
own organs, including the liver, brain and bone marrow. It can be
inherited, which is known as primary or "familial" HLH. It can also
have non-inherited causes. People with primary HLH usually develop
symptoms within the first months or years of life. Symptoms may
include fever, enlarged liver or spleen and decreased number of
blood cells.
The efficacy of Gamifant was studied in a clinical trial of 27
pediatric patients with suspected or confirmed primary HLH with
either refractory, recurrent or progressive disease during
conventional HLH therapy or who were intolerant of conventional HLH
therapy. The median age of the patients in the trial was 1 year
old. The study showed that 63 percent of patients experienced a
response and 70 percent were able to proceed to stem cell
transplant.
Common side effects reported by patients receiving Gamifant in
clinical trials included infections, hypertension, infusion-related
reactions, low potassium and fever. Patients receiving Gamifant
should not receive any live vaccines and should be tested for
latent tuberculosis. Patients should be closely monitored and
treated promptly for infections while receiving Gamifant.
The FDA granted this application Priority Review and
Breakthrough Therapy designation. Gamifant also
received Orphan Drug designation, which provides
incentives to assist and encourage the development of drugs for
rare diseases.
The FDA granted the approval of Gamifant to Novimmune SA.
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, products that
give off electronic radiation, and for regulating tobacco
products.
Media Inquiries: Amanda
Turney, 301-796-2969, amanda.turney@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA
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SOURCE U.S. Food and Drug Administration