U.S. Food & Drug Administration sets
Prescription Drug User Fee Act action date for Sept. 3, 2019
Celgene Corporation (NASDAQ:CELG) today announced the U.S. Food
and Drug Administration (FDA) has accepted the company’s New Drug
Application (NDA) for fedratinib and granted a Priority Review.
Fedratinib is a highly selective JAK2 inhibitor intended for the
treatment of patients with myelofibrosis, a serious bone marrow
disorder that disrupts the body’s normal production of blood
cells.1 Under the Prescription Drug User Fee Act, the FDA has set
its action date as Sept. 3, 2019.
“The acceptance of the NDA and granting of Priority Review for
fedratinib represent the first potential new treatment option after
many years for patients affected by myelofibrosis.” said Jay
Backstrom, M.D., Chief Medical Officer for Celgene. “Patients with
myelofibrosis, including the number who are ineligible for or
failed existing therapy continues to increase, representing a
well-defined unmet medical need. We believe fedratinib can play an
important role in the treatment of myelofibrosis and we look
forward to working with the FDA as the review process
advances.”
The NDA for fedratinib is based on results from a randomized,
placebo-controlled, phase 3 trial (JAKARTA) in patients with
primary or secondary myelofibrosis, as well as a single-arm,
open-label phase 2 trial (JAKARTA2) in patients with primary or
secondary myelofibrosis previously exposed to ruxolitinib, the only
FDA-approved treatment for the disease. Results of these two trials
have been previously published in peer-reviewed journals. The FDA
has also provided fedratinib Orphan Drug designation for the
treatment of secondary and primary myelofibrosis.
Celgene also plans to evaluate fedratinib in combination with
luspatercept.
Fedratinib is an investigational compound that is not approved
for any use in any country.
About JAKARTA
JAKARTA is a pivotal phase 3, multicenter, randomized,
double-blind, placebo-controlled trial evaluating the efficacy of
daily oral doses (400 mg or 500 mg) of fedratinib compared with
placebo in patients with intermediate-2 or high-risk primary
myelofibrosis, post-polycythemia vera myelofibrosis or
post-essential thrombocythemia myelofibrosis with splenomegaly. The
study included 289 subjects across 94 sites in 24 countries.
The primary endpoint was spleen response rate, defined as the
proportion of patients who had a reduction in spleen volume of at
least 35% after six one-month treatment cycles with a magnetic
resonance imaging (MRI) or computerized tomography (CT) scan four
weeks later. Secondary endpoints included symptom response rate,
defined as the proportion of patients with a 50% or greater
reduction in Total Symptom Score after six one-month treatment
cycles as measured by the modified Myelofibrosis Symptoms
Assessment Form (MFSAF) v2.0 diary.
About JAKARTA2
JAKARTA2 is a phase 2, multicenter, open label, single-arm trial
evaluating the efficacy of a once daily dose of fedratinib (400 mg
starting dose) in patients previously treated with ruxolitinib and
with a diagnosis of intermediate-1 with symptoms, intermediate-2 or
high-risk primary myelofibrosis, post-polycythemia vera
myelofibrosis or post-essential thrombocythemia myelofibrosis. The
study included 97 subjects across 40 sites in 10 countries.
The primary endpoint was spleen response rate, defined as the
proportion of patients who had a reduction in spleen volume of at
least 35% as measured by MRI or CT scan after six one-month
treatment cycles. Secondary endpoints included symptom response
rate, defined as the proportion of patients with a 50% or greater
reduction in Total Symptom Score after six one-month treatment
cycles as measured by the modified Myelofibrosis Symptoms
Assessment Form (MFSAF) v2.0 diary.
FDA placed a clinical hold on the fedratinib program in November
2013 after potential cases of Wernicke’s encephalopathy were
reported among subjects (approximately 1%) in clinical trials. The
FDA removed the clinical hold in August 2017.
About Fedratinib
Fedratinib is an oral kinase inhibitor with activity against
wild type and mutationally activated Janus Associated Kinase 2
(JAK2) and FMS-like tyrosine kinase 3 (FLT3). Fedratinib is a
JAK2-selective inhibitor with higher potency for JAK2 over family
members JAK1, JAK3 and TYK2. Abnormal activation of JAK2 is
associated with myeloproliferative neoplasms (MPNs), including
myelofibrosis and polycythemia vera. In cell models expressing
mutationally active JAK2 or FLT3, fedratinib reduced
phosphorylation of signal transducer and activator of transcription
(STAT3/5) proteins, inhibited cell proliferation, and induced
apoptotic cell death. In mouse models of JAK2V617F-driven
myeloproliferative disease, fedratinib blocked phosphorylation of
STAT3/5, increased survival and improved disease-associated
symptoms, including reduction of white blood cells, hematocrit,
splenomegaly, and fibrosis.
About Myelofibrosis
Myelofibrosis is a serious and rare bone marrow disorder that
disrupts the body’s normal production of blood cells. Bone marrow
is gradually replaced with fibrous scar tissue, which limits the
ability of the bone marrow to make red blood cells.1 The disorder
can lead to anemia, weakness, fatigue and swelling of the spleen
and liver, among other symptoms.1 Myelofibrosis is classified as a
myeloproliferative neoplasm, a group of rare blood cancers that
derive from blood-forming stem cells.2 In the U.S. myelofibrosis
occurs in 1.5 of every 100,000 people each year.3 Both men and
women are affected and, while the disease can affect people of all
ages, the median age at diagnosis ranges from 60 to 67 years4,5
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey,
is an integrated global biopharmaceutical company engaged primarily
in the discovery, development and commercialization of innovative
therapies for the treatment of cancer and inflammatory diseases
through next-generation solutions in protein homeostasis,
immuno-oncology, epigenetics, immunology and neuro-inflammation.
For more information, please visit www.celgene.com.
Follow Celgene on Social Media: @Celgene,
LinkedIn and Facebook.
Forward-Looking Statements
This press release contains forward-looking statements, which
are generally statements that are not historical facts.
Forward-looking statements can be identified by the words
"expects," "anticipates," "believes," "intends," "estimates,"
"plans," "will," "outlook" and similar expressions. Forward-looking
statements are based on management's current plans, estimates,
assumptions and projections, and speak only as of the date they are
made. Celgene undertakes no obligation to update any
forward-looking statement in light of new information or future
events, except as otherwise required by law. Forward-looking
statements involve inherent risks and uncertainties, most of which
are difficult to predict and are generally beyond each company's
control. Actual results or outcomes may differ materially from
those implied by the forward-looking statements as a result of the
impact of a number of factors, many of which are discussed in more
detail in the Annual Report on Form 10-K and other reports of each
company filed with the Securities and Exchange Commission,
including factors related to the proposed transaction between
Bristol-Myers Squibb and Celgene, such as, but not limited to, the
risks that: management’s time and attention is diverted on
transaction related issues; disruption from the transaction makes
it more difficult to maintain business, contractual and operational
relationships; pending legal proceedings or any future litigation
instituted against Bristol-Myers Squibb, Celgene or the combined
company could delay or prevent the proposed transaction; and
Bristol-Myers Squibb, Celgene or the combined company is unable to
retain key personnel.
1 Mayo Clinic. Myelofibrosis. Available at
https://www.mayoclinic.org/diseases-conditions/myelofibrosis/symptoms-causes/syc-20355057.
Accessed January 2019.
2 Leukemia & Lymphoma Society. Myelofibrosis. Available at
https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis.
Accessed January 2019.
3 Leukemia & Lymphoma Society. Myelofibrosis facts.
Available at
https://www.lls.org/sites/default/files/file_assets/FS14_Myelofibrosis_Fact%20Sheet_Final9.12.pdf.
Accessed January 2019.
4 Abdel-Wahab O and Levine R. Primary myelofibrosis: Updates on
Definition, Pathogenesis and Treatment. Annual Review of Medicine.
2009;60:233-245.
5 Mesa R, Niblack J, Wadleigh M, et al. The burden of fatigue
and quality of life in myeloproliferative disorders (MPDs). Cancer.
2007;109:68-76.
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