NEW
YORK, Feb. 14, 2023 /PRNewswire/ -- Delcath
Systems, Inc. (Nasdaq: DCTH), an interventional oncology company
focused on the treatment of primary and metastatic cancers of the
liver, announced it submitted a new drug application (NDA)
resubmission to the US Food and Drug Administration (FDA) for the
HEPZATO™ Kit (melphalan hydrochloride for Injection/Hepatic
Delivery System or Melphalan/HDS) seeking approval for the
treatment of patients with unresectable hepatic-dominant metastatic
ocular melanoma (mOM).
The resubmission is in response to a September 12, 2013 Complete Response Letter (CRL)
from the FDA. The NDA resubmission contains comprehensive data and
information to address all issues identified in the CRL. The FDA is
expected to determine whether the resubmission constitutes a
complete response and is eligible for review within 30 days. Once
accepted for review by the FDA, a new PDUFA action date will be
established for the HEPZATO Kit application.
"The submission is a critical milestone for Delcath and
represents the culmination of years of work by investigators and
employees," said Gerard Michel,
CEO of Delcath. "We look forward to receiving feedback from the FDA
regarding a PDUFA date within 30 days."
About the FOCUS Trial
The FOCUS trial evaluated the safety and efficacy of treatment
with HEPZATO Kit for patients with mOM. The primary endpoint of
Overall Response Rate (ORR) was assessed by an Independent Review
Committee per RECIST v1.1. Per protocol, patients were treated
every 6 to 8 weeks for a maximum of 6 cycles. Tumor responses were
assessed every 12 weeks (+/- 2 weeks) until disease
progression.
Delcath powered the single arm trial to demonstrate a superior
ORR versus checkpoint inhibitors, one of the few mOM treatment
categories with a significant amount of peer reviewed publications.
The checkpoint inhibitor ORR was calculated based on a
meta-analysis covering 16 different publications and 476 patients.
Based on those assumptions, a 21.0% ORR was required to demonstrate
superiority over the checkpoint inhibitors at a 95% confidence
interval. The FOCUS study's intent-to-treat (ITT) population was
comprised of a total of 102 mOM subjects. In the ITT population, 91
patients were administered at least one study treatment. Of the 91
patients in the treated population, 51 (56.0%) had no prior therapy
for liver metastases and 40 (44.0%) had at least one line of prior
therapy. As previously reported, treatment with HEPZATO Kit in the
treated population resulted in an ORR of 36.3% [95% CI: 26.44,
47.01] including 7.7% of patients with a complete response (CR).
The median duration of response was 14.00 months [95% CI: 8.31,
17.74] and the disease control rate (DCR) was 73.6% [95% CI: 63.35,
82.31].
In addition, the NDA resubmission includes updated estimated
median overall survival (OS) of 20.53 months [95% CI: 16.79, 25.26]
and updated estimated OS at 1 year of 0.80 [95% CI: 0.70, 0.87].
Delcath will continue to follow patients until May 2023 (24 months after the last patient's last
treatment).
In the FOCUS trial safety population (95 patients), 39 patients
(41.1%) experienced a treatment-related serious adverse event. The
most commonly reported treatment-related serious adverse events
were thrombocytopenia, neutropenia and febrile neutropenia which
were well-manageable. Five percent of patients experienced
treatment-related serious cardiac adverse events; in all cases the
events resolved with no ongoing complications. There were no
treatment-related deaths in the trial. This is consistent with
CHEMOSAT, the HDS device component of HEPZATO Kit, approved in
Europe under a CE mark to deliver
melphalan to the liver. The safety data submitted in the NDA is
consistent with the CHEMOSAT safety data documented in numerous
European single-center and multi-center publications.
About the HEPZATO Kit
The HEPZATO Kit is a drug-device combination product comprised
of the drug (melphalan) and device (HDS) constituent parts.
Melphalan is a well-established, broadly effective anticancer
chemotherapeutic agent belonging to the alkylating class and is
responsible for the combination product's primary mode of action.
The procedure of surgical isolation and simultaneous filtration of
hepatic venous blood during drug infusion and washout, known as
percutaneous hepatic perfusion, or PHP, results in loco-regional
delivery of a relatively high melphalan dose, which can potentially
induce a clinically meaningful tumor response with minimal
hepatotoxicity and reduce systemic exposure relative to the
comparable intravenous (IV) dose.
In the US, the efficacy and safety of HEPZATO Kit have not been
established for any indication and it is not presently approved by
the FDA.
About Delcath Systems, Inc.
Delcath Systems, Inc. is an interventional oncology company
focused on the treatment of primary and metastatic liver cancers.
The company's proprietary products, HEPZATO Kit (melphalan
hydrochloride for Injection/Hepatic Delivery System) and
CHEMOSAT® Hepatic Delivery System for Melphalan
percutaneous hepatic perfusion (PHP) are designed to administer
high-dose chemotherapy to the liver while controlling systemic
exposure and associated side effects during a PHP procedure.
In the United States, HEPZATO
Kit is considered an investigational drug/device combination
product regulated as a drug by the United States Food and Drug
Administration (FDA). HEPZATO Kit is comprised of the
chemotherapeutic drug melphalan and Delcath's proprietary Hepatic
Delivery System (HDS). The HDS is used to surgically isolate the
liver while simultaneously filtrating hepatic venous blood during
melphalan infusion and washout. The use of the HDS results in
loco-regional delivery of a relatively high melphalan dose, which
can potentially induce a clinically meaningful tumor response with
minimal hepatotoxicity and reduce systemic exposure. In the US,
HEPZATO Kit was the subject of a February
14, 2023 new drug application resubmission to FDA for the
treatment of patients with unresectable hepatic-dominant metastatic
ocular melanoma (mOM), also known as metastatic uveal melanoma
(mUM). In Europe, the device-only
configuration of the HDS is regulated as a Class lll medical device
and is approved for sale under the trade name CHEMOSAT Hepatic
Delivery System for Melphalan, or CHEMOSAT, where it has been used
in the conduct of percutaneous hepatic perfusion procedures at
major medical centers to treat a wide range of cancers of the
liver.
Safe Harbor/Forward-Looking Statements
The 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: actions by the FDA
relating to the application; the ability of the Company to respond
to FDA queries related to the application; the Company's successful
inspections by the FDA or foreign regulatory agencies; the timing
and results of the Company's clinical trials, our determination
whether to continue a clinical trial program or to focus on other
alternative indications, and the impact of the COVID-19 pandemic on
the completion of our clinical trials; the impact of the
presentations at major medical conferences and future clinical
results consistent with the data presented; the Company's ability
to successfully commercialize the HEPZATO Kit and the potential of
the HEPZATO Kit/CHEMOSAT system as a treatment for patients with
primary and metastatic disease in the liver; our ability to obtain
reimbursement for commercialized product in various markets; the
Company's ability to successfully enter into strategic partnership
and distribution arrangements and the timing and revenue, if any,
of the same; uncertainties relating to the timing and results of
research and development projects; 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:
Investor Relations Contact:
Ben Shamsian
Lytham Partners
646-829-9701
shamsian@lythampartners.com
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SOURCE Delcath Systems, Inc.