Recommends Special Arrangement Designation Which Increases Reimbursement Options
NEW YORK, April 21, 2021 (GLOBE NEWSWIRE) -- Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, announced that the United Kingdom’s (UK) National Institute for Health and Care Excellence (NICE), through the Interventional Procedures Advisory Committee, has updated its guidance for the Delcath CHEMOSAT® Hepatic Delivery System for Melphalan (CHEMOSAT) in the treatment of patients with metastases in the liver from Ocular Melanoma.
Previously, the NICE guidance recommended CHEMOSAT only be used in the context of formal research studies. Due to that guidance, both private insurance and regional funding were generally not available for treatments with CHEMOSAT, nor was it possible to apply for national coverage. Under the revised NICE guidance, CHEMOSAT has been categorized under a Special Arrangement designation. Under this designation, private insurance may be more likely to fund treatment with CHEMOSAT, some regional funding may be more accessible, and a process is now available to seek national reimbursement.
The revised guidance which formed the basis in the designation change can be found at https://www.nice.org.uk/guidance/ipg691.
“We welcome this Special Arrangements designation from NICE for the treatment of patients with metastases in the liver from ocular melanoma and the recommendations within the final guidance. We look forward to working with specialist treatment centers to increase the availability of this treatment to patients with metastases in the liver from ocular melanoma in the UK” said Gerard Michel, CEO of Delcath. “This change in designation, combined with the recently announced positive preliminary results from the FOCUS trial, is further evidence of the potential value of the HEPZATO and CHEMOSAT platform to patients worldwide suffering from metastatic ocular melanoma.”
About HEPZATO™ KIT and CHEMOSAT®
The HEPZATO™ KIT (melphalan hydrochloride for injection/hepatic delivery system), or HEPZATO, is a drug/device combination product. HEPZATO is designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects. In Europe, the commercial product is a stand-alone medical device and is approved for sale under the trade name CHEMOSAT Hepatic Delivery System for Melphalan, or CHEMOSAT, where it has been used at major medical centers to treat a wide range of cancers of the liver. In the United States, HEPZATO is considered a combination drug and device product regulated by the United States Food and Drug Administration (FDA). Primary jurisdiction for regulation of HEPZATO has been assigned to the FDA’s Center for Drug Evaluation and Research. The FDA has granted Delcath six orphan drug designations (five for melphalan in ocular melanoma, cutaneous melanoma, cholangiocarcinoma, hepatocellular carcinoma, and neuroendocrine tumor indications and one for doxorubicin in the hepatocellular carcinoma indication). HEPZATO has not been approved for sale in the United States.
In Europe, CHEMOSAT is regulated as a Class IIb medical device and received its CE Mark in 2012. Delcath is commercializing CHEMOSAT in select markets in the European Union (EU), where the prospect of securing reimbursement coverage for the use of CHEMOSAT is strongest.
HEPZATO is being studied in the FOCUS trial which is a single-arm, multi-center, open-label trial to treat patients with hepatic-dominant metastatic ocular melanoma (mOM). The FOCUS Trial is being conducted at approximately 30 sites in the United States and Europe. The primary endpoint of the FOCUS Trial is Objective Response Rate (ORR) as measured by RECISTv1.1, in the Intent to Treat (ITT) population. The single arm trial was powered 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 which included 476 patients. The pooled overall response rate was 5.5% [95% CI: 3.6, 8.3]. To achieve statistical significance at a 95% Confidence Interval the lower bound of the ORR for HEPZATO is required to exceed the 8.3% upper bound of the meta-analysis.
Secondary endpoints include Duration of Response (DOR), Disease Control Rate (DCR), Overall Survival (OS), and Progression-Free Survival (PFS). Additional exploratory outcome measures include time to objective response, hepatic progression-free survival, hepatic objective response, and quality of life, safety, and other pharmacokinetic measures. Initially, the trial was a randomized controlled trial which was amended to a single arm trial given slow enrollment due to the rarity of ocular melanoma, absence of crossover to the experimental trial arm, competing clinical trials and the commercial availability of CHEMOSAT in Europe. Included in the prespecified analyses are comparisons against the Best Alternative Care (BAC) arm which enrolled 32 patients prior to the amendment to a single-arm trial.
On March 31, 2021 Delcath released a preliminary analysis of the FOCUS trial data based on 87% of enrolled patients using prespecified analyses. An Independent Review Committee assessed an ORR of 29.2% [95% CI: 20.1, 39.8] in the ITT population, the lower bound of which exceeded the upper bound of the predefined success criteria (8.3%) for the primary ORR endpoint. In the per protocol populations, evaluable patients in the HEPZATO arm had a statistically significant improvement over BAC in prespecified endpoints including: ORR of 32.9% [95% CI: 22.8, 44.4] versus 13.8% [CI: 3.9, 31.7] for the BAC arm (Chi-square P