Ascentage Pharma has been granted orphan drug designation (ODD) for its Bcl-2 inhibitor APG-2575 from the US Food and Drug Administration (FDA) for the treatment of Waldenström macroglobulinemia (WM).

The orally administered Bcl-2 selective inhibitor that has been designed for the treatment of a range of hematologic malignancies by selectively inhibiting Bcl-2 to restore the normal apoptosis process in cancer cells, said Ascentage Pharma.

The FDA orphan drug designation enables APG-2575 to qualify for certain development incentives such as a tax credit on expenses incurred in conducting clinical studies, a waiver of the new drug application (NDA) fee, a research grant from the pharma regulator, and also seven years of market exclusivity in the US following an approval.

Waldenström macroglobulinemia is a form of lymphocytic lymphoma in which the bone marrow is infiltrated by lymphoplasmacytic cells with increased levels of monoclonal immunoglobulin M (IgM) antibodies.

According to Ascentage Pharma, treatment recommendations for Waldenström macroglobulinemia from present guidelines of the US National Comprehensive Cancer Network (NCCN) suggest an objective response rate (ORR) of around 80% with currently available therapies. However, these current therapies provide a very low rate of very good partial response (VGPR) or deeper responses with most patients facing disease progression after initial response, said the clinical-stage biotechnology company.

Also, patients are diagnosed with Waldenström macroglobulinemia at a median age of 70, when several of them are not tolerant existing therapies due to poor health conditions, thereby representing a major unmet medical need for more effective and safer therapies.

Dr. Dajun Yang – Chairman and CEO of Ascentage Pharma said: “APG-2575 is a key drug candidate in Ascentage Pharma’s pipeline targeting apoptosis. This ODD by the US FDA for the treatment of WM marks an important milestone in the global development and commercialization of APG-2575.

“All the policy support and incentives as a result of this ODD will help us accelerate the global clinical development of APG-2575, which we hope will soon transform to benefit more patients.”

APG-2575 is the second drug candidate of Ascentage Pharma to have been given the FDA orphan drug designation after the BCR-ABL inhibitor HQP1351, which was granted the status by the US pharma regulator in May 2020 for chronic myeloid leukemia treatment.

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