FDA Grants Orphan Drug Designation to RenovoCath’s Delivery of Intra-Arterial Gemcitabine for the Treatment of Cholangiocarcinoma

August 2020, Vol 1, No 1

On June 2, 2020, the US Food and Drug Administration (FDA) granted an orphan drug designation to RenovoCath, an intra-arterial delivery device of gemcitabine (Gemzar) for the treatment of patients with cholangiocarcinoma (CCA). RenovoCath is developed by RenovoRx, a medical devices developer based in Los Altos, CA.

RenovoCath uses a dual-balloon infusion catheter and the Transarterial Micro-Perfusion (TAMP) technique for the targeted delivery of gemcitabine to the tumor site. The device’s 2 balloons (proximal and distal occlusion balloons) enable the effective isolation of the arterial site to allow for the delivery of gemcitabine in a controlled environment, thereby avoiding the delivery of chemotherapy to nontargeted areas.

This technique has had good application in the treatment of hepatocellular carcinoma and is now an option for the treatment of patients with CCA.

The safety of the targeted intra-arterial delivery of gemcitabine was tested in 20 patients with locally advanced pancreatic adenocarcinoma at doses of up to 1000 mg/m2.1 The patients’ symptoms and laboratory values were monitored for safety and tolerability. The secondary end points included the effect on tumor size, tumor markers, and survival. The approach was determined to be safe, and the efficacy results suggest a survival benefit compared with the historic comparison group. The results demonstrated that the localized and selective delivery of intra-arterial gemcitabine to the pancreas was feasible using RenovoCath.

RenovoCath’s TAMP delivery method is also being investigated in the TIGeR-PaC clinical study.2 TIGeR-PaC is a phase 3, multicenter, unblinded, randomized control study of patients with locally advanced unresectable pancreatic adenocarcinoma. All patients in the trial will receive induction therapy of intravenous gemcitabine plus nab-paclitaxel, as well as radiation therapy for approximately 4 months. Patients who remain eligible will then be randomized to receive intra-arterial chemotherapy with gemcitabine or to continue gemcitabine plus nab-paclitaxel. The goal of the trial is to prove extended median survival and improved quality of life through the targeted delivery of therapy.

“Receiving a second orphan drug designation from the FDA is a significant milestone as we build the TAMP platform for solid tumor treatment. This new orphan drug indication builds on the momentum of the TIGeR-PaC phase 3 clinical trial currently treating pancreatic cancer patients in the United States and Europe,” said RenovoRx’s CEO Shaun Bagai in a press release.3

“To help bile duct cancer patients, we are designing a phase 1/2 clinical trial that will launch by early next year. This expansion of the RenovoRx platform, beyond pancreatic cancer, could improve outcomes for more cancer patients,” Mr Bagai added. “Our team is evaluating market opportunities in Asia since it is estimated that more than 100,000 patients are diagnosed annually with bile duct cancer that could be treated with RenovoRx’s TAMP delivery of intra-arterial gemcitabine.”3


  1. Rosemurgy AS, Ross SB, Vitulli PL, et al. Safety study of targeted and localized intra-arterial delivery of gemcitabine in patients with locally advanced pancreatic adenocarcinoma. J Pancreat Cancer. 2017;3:58-65.
  2. Clinicaltrials.gov. Intra-arterial gemcitabine vs. IV gemcitabine and nab-paclitaxel following radiotherapy for LAPC (TIGeR-PaC). https://clinicaltrials.gov/ct2/show/NCT03257033. Accessed July 24, 2020.
  3. RenovoRx announces orphan drug designation granted for treating bile duct cancer. Press release. June 2, 2020. www.prnewswire.com/news-releases/renovorx-announces-orphan-drug-designation-granted-for-treating-bile-duct-cancer-301068993.html. Accessed July 27, 2020.

Related Items

FDA Grants TT-00420, a Multi-Kinase Inhibitor, Fast-Track Review for Cholangiocarcinoma
Web Exclusives
On November 3, 2021, TransThera Sciences announced that the FDA granted its investigational drug, TT-00420, a spectrum-selective multi-kinase inhibitor, a fast-track designation for the treatment of patients with cholangiocarcinoma (CCA) who have no available standard treatment options. In preclinical studies, TT-00420 has shown high activity in a variety of FGFR mutations.
Tibsovo First Targeted Therapy FDA Approved for Advanced or Metastatic CCA with IDH1 Mutation
September/October 2021, Vol 2, No 3
On August 25, 2021, the FDA approved ivosidenib (Tibsovo; Servier Pharmaceuticals), an oral IDH1 inhibitor, for the treatment of adults with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) and an IDH1 mutation, as detected by an FDA-approved test. The FDA granted ivosidenib a priority review for this indication and an orphan drug designation.
Ivosidenib Approved for Advanced or Metastatic Cholangiocarcinoma
Web Exclusives
Ivosidenib has been approved by the FDA for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 mutation as detected by an FDA-approved test.
FDA Grants Accelerated Approval to Infigratinib for Metastatic Cholangiocarcinoma
June/July 2021, Vol 2, No 2
The FDA granted accelerated approval to the kinase inhibitor infigratinib (Truseltiq) for the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) that harbors an FGFR2 fusion or other rearrangement.

Subscribe Today!

To sign up for our newsletter or print publications, please enter your contact information below.

I'd like to receive: