FDA Grants Orphan Drug Designation to Pvsripo for Treatment of Advanced Melanoma


Pvsripo is a novel immunotherapy that activates patients’ innate and adaptive immune system to enable anti-tumor response and establish immunologic memory to prevent the recurrence of cancer.

Istari Oncology, Inc, has announced that the FDA has granted orphan drug designation to Pvsripo for the treatment of advanced melanoma in stages IIB through IV. The designation offers incentives for the development of drugs for rare diseases, including eligibility for federal grants, a potential 7-year marketing exclusivity period following FDA approval, and more.

Pvsripo is a novel immunotherapy based on the Sabin type 1 polio vaccine that activates patients’ innate and adaptive immune system to enable anti-tumor response and establish immunologic memory to prevent the recurrence of cancer. The drug could be groundbreaking in the treatment of melanoma, which has approximately 7000 deaths in the United States each year and is the deadliest type of skin cancer.

Patients with treatment-resistant advanced melanoma have poor survival rates, with fewer than 28% of patients with metastatic melanoma surviving 5 years, according to a press release. Pvsripo could address the unmet need in melanoma treatment, since most patients are treated with checkpoint inhibitors and many are treatment-resistant with limited alternative treatment options.

“We are happy to kick off the new year with the announcement that our request for an orphan drug designation has been granted to Pvsripo for the treatment of advanced melanoma,” said Matt Stober, president and CEO of Istari Oncology, in a press release.

Recruitment is ongoing for LUMINOS-102, a phase 2 open-label, randomized trial in patients with advanced, unresectable melanoma who previously failed anti-programmed cell death protein 1 (PD-1) therapy. The study will examine the safety, tolerability, and initial efficacy of Pvsripo intratumoral injection alone and in combination with a PD-1 inhibitor, and the first patient is expected to be dosed in the first quarter of 2021.

LUMINOS-102 follows the successful phase 1 monotherapy study of Pvsripo in anti-PD1 refractory advanced melanoma in which patients who received 3 injections had an overall response rate of 67%, according to the press release from Istari.

“We are encouraged by a data from our phase 1 trial presented at last year’s Society for Immunotherapy of Cancer (SITC) 2020 annual meeting,” said Garrett Nichols, MD, MS, chief medical officer at Istari Oncology. “We plan to build upon that success with LUMINOS-102. As those data and the data from our other solid tumor trials emerge, we will continue to work closely with the FDA toward the goal of bringing Pvsripo to market.”

Orphan drug designation is granted to drugs or biological products for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the United States. Incentives that come with the designation include eligibility for federal grants, research and development tax credits, waiver of filing fees, and the potential for a 7-year marketing exclusivity period. The designation does not alter the standard regulatory requirements and process for obtaining marketing approval.


Istari Oncology Announces FDA Grants Orphan Drug Designation for Pvsripo for the Treatment of Advanced Melanoma [news release]. BusinessWire; January 11, 2021. https://www.businesswire.com/news/home/20210111005373/en/Istari-Oncology-Announces-FDA-Grants-Orphan-Drug-Designation-for-PVSRIPO-for-the-Treatment-of-Advanced-Melanoma. Accessed January 11, 2021.

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