FDA Tells Hutchmed It Won’t Approve Treatment of Advanced Neuroendocrine Tumors

The U.S. Food and Drug Administration notified Hutchmed that it would not approve its application to market surufatinib for the treatment of pancreatic and extra-pancreatic neuroendocrine tumors because the current data package does not support an approval in the United States at this time.

The data package was based on two positive phase 3 trials in China and one bridging study in the United States. In a letter to the company, the agency indicated that a multi-regional clinical trial is required for U.S. approval.

Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptors and fibroblast growth factor receptor, which both inhibit angiogenesis, and colony stimulating factor-1 receptor, which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.

On December 29, 2020, surufatinib was granted drug registration approval in China for the treatment of extra-pancreatic (non-pancreatic) neuroendocrine tumors, epNETs. Surufatinib is marketed in China under the brand name Sulanda. The approval was based on results from the SANET-ep study, a phase 3 trial in patients with advanced epNETs conducted in China. The study met the pre-defined primary endpoint of PFS at a preplanned interim analysis and was published in The Lancet.

On June 16, 2021, surufatinib was granted drug registration approval in China for the treatment of pancreatic neuroendocrine tumors (pNET). The approval was based on results from the SANET-p study, a phase 3 trial in patients with advanced pNET in China. The pre-defined primary endpoint of PFS was met at a preplanned interim analysis and was published in The Lancet Oncology.

Surufatinib received U.S. FDA Fast Track designations in April 2020 for the treatment of pNETs and epNETs. Orphan Drug Designation for pNETs was granted in November 2019. In a May 2020 pre-NDA meeting, Hutchmed reached an agreement with the FDA that the two positive phase 3 studies of surufatinib in patients with pNETs and epNETs in China, along with the bridging trial in the United States could form the basis to support a U.S. NDA submission. The FDA accepted the filing of the NDA on June 30, 2021.

“Although this decision from the FDA is disappointing, we remain confident about the clinical value of surufatinib for NET patients and committed to making surufatinib available to patients globally,” said Weiguo Su, CEO and chief scientific officer of Hutchmed. “There are very few treatments approved and used in these rare diseases, and patients and physicians would benefit from more options to address the unmet medical need.”

Author: Rare Daily Staff