NEW YORK, July 20, 2015 /PRNewswire/ -- Delcath
Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and
medical device company focused on oncology with an emphasis on the
treatment of primary and metastatic liver cancers, announced today
that the U.S. Food and Drug Administration (FDA) Office of Orphan
Products Development (OOPD) has granted Orphan Drug Designation
for melphalan for the treatment of
cholangiocarcinoma. The OOPD is tasked with evaluating
the scientific and clinical data submissions from sponsors to
identify and designate products as promising for rare diseases and
to further advance scientific development of such promising medical
products.
Orphan drug designation provides certain exclusivity benefits,
tax credits for certain research and a waiver of the New Drug
Application user fee. Cholangiocarcinoma is recognized by the
FDA as an orphan disease, usually defined as a condition that
affects fewer than 200,000 people nationwide.
Intrahepatic cholangiocarcinoma (ICC), a sub-category of
cholangiocarcinoma, is a tumor in the bile duct that arises within
the liver. It is the second most common primary liver tumor
and represents approximately 15% of new HCC cases diagnosed
annually. Surgical resection, the standard of care, is not possible
for an estimated 80% to 90% of patients diagnosed with ICC.
The Company recently announced the expansion of its global Phase
2 clinical study in primary liver cancer (HCC) to include an ICC
cohort, which is investigating the safety and efficacy of
Melphalan/HDS treatment in patients with unresectable ICC confined
to the liver. The study is being conducted at the same
hospitals in Europe participating
in the Company's Phase 2 HCC trial, and is expected to enroll 11
patients. The ICC cohort will evaluate tumor response (objective
response rate) as measured by modified Response Evaluation Criteria
in Solid Tumor (mRECIST), and will assess progression-free survival
and safety. Additional analyses will be conducted to characterize
the systemic exposure of melphalan administered by Melphalan/HDS,
as well as to assess patient-reported clinical outcomes, or
quality-of-life.
"We are pleased with the receipt of orphan drug designation for
melphalan in the treatment of patients with cholangiocarcinoma as
it is a key milestone that supports our broader regulatory and
development strategy for our Melphalan/Hepatic Delivery System
(Melphalan/HDS) as a therapy for primary and metastatic liver
cancers," said Jennifer Simpson,
Ph.D., M.S.N., C.R.N.P., President and Chief Executive Officer of
Delcath. "ICC is a disease of significant unmet medical need and
our Melphalan/HDS treatment may offer clinical benefit for ICC
patients who face limited treatment options."
About Delcath Systems
Delcath Systems, Inc. is a
specialty pharmaceutical and medical device company focused on
oncology with an emphasis on the treatment of primary and
metastatic liver cancers. Our proprietary Melphalan Hydrochloride
for Injection for use with the Delcath Hepatic Delivery System
(Melphalan/HDS) is designed to administer high-dose chemotherapy to
the liver while controlling systemic exposure. In April 2012 we obtained authorization to affix a
CE Mark to our second-generation system, which is currently
marketed in Europe as a device
under the trade name Delcath Hepatic CHEMOSAT® Delivery System for
Melphalan (CHEMOSAT). In the U.S. the Melphalan/HDS system is
considered a combination drug and device product, and is regulated
as a drug by the U.S. Food and Drug Administration (FDA). The
Melphalan/HDS system has not been approved for sale in the U.S. We
have commenced a global Phase 2 clinical trial in Europe and the U.S. to investigate the
Melphalan/HDS system for the treatment of primary liver cancer
(HCC) and intrahepatic cholangiocarcinoma (ICC), and expect to
initiate a global Phase 3 trial in ocular melanoma (OM) that has
metastasized to the liver.
Private Securities Litigation Reform Act of 1995 provides a
safe harbor for forward-looking statements made by the Company or
on its behalf. This news release contains forward-looking
statements, which are subject to certain risks and uncertainties
that can cause actual results to differ materially from those
described. Factors that may cause such differences include, but are
not limited to, uncertainties relating to: future patient outcomes
and clinical trial results consistent with the data contained in
the SSO abstract, acceptance and publication of the Phase 3 trial
manuscript and the impact of publication to support the Company's
efforts, the timing and results of the Company's
clinical trials including without limitation the HCC, ICC and
OM clinical trial programs timely enrollment and
treatment of patients in the global Phase 2 HCC and ICC clinical
trial, FDA approval of the global Phase 3 OM clinical trial
protocol, IRB or ethics committee clearance of the Phase 2
HCC/ICC and/or Phase 3 OM protocols from
participating sites and the timing of site activation and subject
enrollment in each trial, the impact of the presentations at ESSO
and future clinical results consistent with the data presented,
approval of Individual Funding Requests for reimbursement of the
CHEMOSAT procedure, the impact, if any of Value 4 status on
potential CHEMOSAT product use and sales in Germany, clinical adoption, use and resulting
sales, if any, for the CHEMOSAT system to deliver and filter
melphalan in Europe including the
key markets of Germany and the UK,
the Company's ability to successfully commercialize the Melphalan
HDS/CHEMOSAT system and the potential of the Melphalan HDS/CHEMOSAT
system as a treatment for patients with primary and metastatic
disease in the liver, our ability to obtain reimbursement for the
CHEMOSAT system in various markets, the Company's ability to
satisfy the requirements of the FDA's Complete Response Letter and
provide the same in a timely manner, approval of the current or
future Melphalan HDS/CHEMOSAT system for delivery and filtration of
melphalan or other chemotherapeutic agents for various indications
in the U.S. and/or in foreign markets, actions by the FDA or other
foreign regulatory agencies, the Company's ability to successfully
enter into strategic partnership and distribution arrangements in
foreign markets and the timing and revenue, if any, of the same,
uncertainties relating to the timing and results of research and
development projects, our ability to maintain NASDAQ listing, and
uncertainties regarding the Company's ability to obtain financial
and other resources for any research, development, clinical trials
and commercialization activities. These factors, and others, are
discussed from time to time in our filings with the Securities and
Exchange Commission. You should not place undue reliance on these
forward-looking statements, which speak only as of the date they
are made. We undertake no obligation to publicly update or revise
these forward-looking statements to reflect events or circumstances
after the date they are made.
Contact Information:
Investor Contact:
LHA
Anne Marie Fields,
afields@lhai.com
212-838-3777
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SOURCE Delcath Systems, Inc.