FDA Grants Orphan Drug Designation to First Liver-Targeted Drug for HCC

A novel liver-targeted drug, MIV-818, has been granted an Orphan Drug Designation by the FDA for the treatment of patients with hepatocellular carcinoma (HCC), according to a press release from Medivir AB, developer of the drug.¹

This designation follows an Orphan Medicinal Drug Designation in Europe for the same indication, which was granted in April by the Committee for Orphan Medicinal Products, a division of the European Medicines Agency.²

“It is very satisfying that MIV-818 has received a positive opinion on orphan medicinal drug designation by EMA and has been granted orphan drug designation by the FDA for the treatment of HCC”, says Uli Hacksell, CEO at Medivir.¹

MIV-818 is currently under investigation in 2-part phase I/IIa study (NCT03781934). In early March, Medivir announced that the first patient with liver cancer was dosed with MIV-818 in the study. It was also reported that in the phase Ia study, 5 out of 9 patients had stable disease following treatment with MIV-818.³

"Patients with advanced liver cancer have limited treatment options and the unmet medical need is large" said Uli Hacksell, PhD, Medivir, in a statement. "Having obtained an encouraging proof-of-concept for the liver-cancer directed effect of MIV-818 in phase Ia, we hope to get additional supportive data from the phase Ib study. We believe that MIV-818 has the potential to provide liver cancer patients with major therapeutic benefits."

The goal of the current phase Ib study is to determine the safety and preliminary efficacy of MIV-818 in patients with liver cancer which consists of patients with HCC and intrahepatic cholangiocarcinoma (iCCA). The primary end point is the number of participants with treatment-related adverse events, which is assessed per the Common Terminology Criteria for Adverse Events and will consist of an evaluation of the clinically significant changes in echocardiograms, and vital signs like heart rate, weight, and height.

The secondary end point is preliminary efficacy per RECISIT evaluation, which will be determined by the objective response rate observed with the drug.

Patients with HCC or iCCA are eligible to enroll in the study if they are 18 years of age or older with measurable disease per RECIST v1.1, have child-Pugh A or B status, and a life expectancy of at least 12 weeks, according to investigator opinion.

At the time of screening, patients are also required to have alanine aminotransferase and aspartate aminotransferase ≤ 5.0 × upper limit of normal, a total bilirubin ≤ 3.0 mg/dL, adequate renal function, a platelet count, ≥ 75,000/mL, and an International Normalized Ratio ≤ 1.7.

MIV-818 is designed to selectively treat liver cancer and lessen the side effects associated with liver cancer. Medivir states that the drug has the potential to become the first liver-targeted, oral drug available to patients with HCC.¹

_References:

_{FDA grants MIV-818 Orphan Drug Designation for the treatment of hepatocellular carcinoma [news release]. Stockholm, Sweden: Medivir AB; May 06, 2020. https://bit.ly/2SGK5c9. Accessed May 06, 2020.}

_{Medivir receives positive opinion on Orphan Medicinal Drug Designation by the European Medicines Agency for MIV-818 [news release]. Stockholm, Sweden: Medivir AB; April 30, 2020. https://bit.ly/2L2Sd2o. Accessed May 6, 2020.}

_{First liver cancer patient dosed in the MIV-818 phase Ib study [news release]. Stockholm, Sweden: Medivir AB; March 10, 2020. https://bit.ly/2W8UI9F. Accessed May 6, 2020.}