Portola Pharmaceuticals Receives FDA Orphan Drug Designation for Cerdulatinib, an Oral Syk/JAK Inhibitor for the Treatment of...
25 Septembre 2018 - 10:38PM
Portola Pharmaceuticals (Nasdaq: PTLA) today announced that the
U.S. Food and Drug Administration (FDA) has granted Orphan Drug
Designation to cerdulatinib, an investigational, oral Syk/JAK
inhibitor for the treatment of peripheral T-cell lymphoma (PTCL).
“We are pleased that the FDA has granted
cerdulatinib Orphan Drug Designation, as it recognizes its
potential to provide a significant clinical benefit to a group of
patients with limited treatment options,” said John Curnutte, M.D.,
Ph.D., Portola’s interim co-president and head of research and
development. “We look forward to presenting additional data from
the Phase 2a trial at a scientific congress early next year and to
continuing discussions with the FDA regarding next steps for the
development of cerdulatinib, including the potential for an
accelerated approval pathway.”
The FDA's Office of Orphan Products Development
grants orphan status to support development of medicines for the
treatment of rare diseases that affect fewer than 200,000 people
in the United States. Orphan Drug Designation may provide
certain benefits, including a seven-year period of market
exclusivity if the drug is approved, tax credits for qualified
clinical trials and an exemption from FDA market application
fees.
About PTCLPTCL is a group of rare and often
fast-growing lymphomas classified as a subtype of non-Hodgkin’s
lymphoma. There are approximately 6,000 to 10,000 cases of PTCL
annually in the United States. The three most common types of PTCL
are anaplastic large cell lymphoma (ALCL), angioimmunoblastic
T-cell lymphoma (AITL), and PTCL not otherwise specified
(PTCL-NOS), but many other rarer types exist. PTCL is most often
treated with a combination of chemotherapies and the options for
patients that fail front-line therapy are limited.
About Cerdulatinib Cerdulatinib
is an investigational oral, dual spleen tyrosine kinase (Syk) and
janus kinase (JAK) inhibitor that uniquely inhibits two key cell
signaling pathways implicated in certain hematologic malignancies
and autoimmune diseases. There is a strong rationale for inhibiting
both Syk (B-cell receptor pathway) and JAK (cytokine receptors) in
B-cell malignancies where both targets have been shown to promote
cancer cell growth and survival. In addition, pre-clinical data
suggest an important role for Syk and JAK in peripheral T-cell
lymphoma (PTCL) tumor survival.
In addition to PTCL, cerdulatinib is being
evaluated in an ongoing Phase 2a study among patients with other
specific subtypes of B-cell and T-cell Non-Hodgkin Lymphoma (NHL),
including relapsed/refractory follicular lymphoma (FL) and chronic
lymphocytic lymphoma/small lymphocytic lymphoma (CLL/SLL). The
Company reported new data from this study in June at both the
2018 American Society of Clinical Oncology (ASCO) Annual
Meeting and at the 23rd Congress of the European Hematology
Association (EHA). Cerdulatinib demonstrated broad clinical
activity including an objective response rate of 47 percent in all
patients, and was generally well tolerated. Additionally,
seven of the 20 patients in the PTCL cohort achieved a complete
response at the time of presentation.
About Portola Pharmaceuticals,
Inc.Portola Pharmaceuticals is a commercial-stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel therapeutics that could significantly
advance the fields of thrombosis and other hematologic diseases.
The Company’s two FDA-approved medicines are Andexxa®
[coagulation factor Xa (recombinant), inactivated-zhzo], the first
and only antidote for patients treated with rivaroxaban and
apixaban when reversal of anticoagulation is needed due to
life-threatening or uncontrolled bleeding, and
Bevyxxa® (betrixaban), the first and only oral, once-daily
Factor Xa inhibitor for the prevention of VTE in adult patients
hospitalized for an acute medical illness. The company also is
advancing cerdulatinib, a Syk/JAK inhibitor for the treatment of
hematologic cancers.
Forward-Looking StatementsThis
announcement contains forward-looking statements, including
statements regarding next steps for the development of
cerdulatinib, including the potential for an accelerated approval
pathway in the U.S. for certain tumor subtypes. Risks that
contribute to the uncertain nature of the forward-looking
statements include: failure to obtain FDA agreement to an
accelerated or other regulatory approval pathway, regulatory
developments in the United States and foreign countries; our
expectation that we will incur losses for the foreseeable future
and will need additional funds to finance our operations; the
accuracy of our estimates regarding our ability to initiate and/or
complete our clinical trials and the timing and expense of these
trials; the results of our clinical trials related to the efficacy
and safety of our product candidates; our potential inability to
manufacture our product candidates on a commercial scale in a
timely or cost-efficient manner; the accuracy of our estimates
regarding expenses and capital requirements; our ability to
successfully build a hospital-based sales force and commercial
infrastructure; our ability to obtain and maintain intellectual
property protection for our product candidates; and our ability to
retain key scientific or management personnel. These and other
risks and uncertainties are described more fully in our most recent
filings with the Securities and Exchange Commission, including our
most recent quarterly report on Form 10-Q. All forward-looking
statements contained in this press release speak only as of the
date on which they were made. We undertake no obligation to update
such statements to reflect events that occur or circumstances that
exist after the date on which they were made.
Investor Contact:
Cara Miller
Portola PharmaceuticalsIR@portola.com
Media Contact:Patrick RyanPure
Communicationspryan@purecommunications.com
Portola Pharmaceuticals (NASDAQ:PTLA)
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