On December 18, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that US WorldMeds1 (USWM), a Kentucky-based specialty pharmaceutical company to whom it divested certain assets in its eflornithine pediatric neuroblastoma program, received FDA approval of its New Drug Application (NDA) for the use of eflornithine as a maintenance therapy for high-risk neuroblastoma patients who have achieved at least a partial response to certain prior therapies (Press release, Panbela Therapeutics, DEC 18, 2023, View Source;utm_medium=rss&utm_campaign=panbela-therapeutics-announces-us-worldmeds-nda-approval-for-eflornithine-dfmo-in-pediatric-neuroblastoma [SID1234638649]).
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The approval of USWM’s NDA for the use of eflornithine for the treatment of patients with high-risk neuroblastoma marks the first FDA approval of an NDA for any polyamine targeted therapy in a cancer indication. "This approval highlights the role polyamines can play in cancer therapy as we look forward to data from our ongoing programs in metastatic pancreatic cancer, colorectal cancer, non-small cell lung cancer, and prostate cancer and the advancement of preclinical programs in ovarian and multiple myeloma," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.
In July 2023, Panbela divested its pediatric neuroblastoma program to USWM in an arrangement entitling Panbela to up to approximately $9.5 million of non-dilutive funding, including payments upon USWM’s successful completion of milestones related to eflornithine’s clinical development, regulatory approval, and commercial sales. "The FDA’s approval of USWM’s eflornithine NDA for high-risk neuroblastoma is an exciting milestone in our partnership. This demonstrates the potential for polyamine targeted therapies in cancer," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.
"This approval is a prerequisite for considerable development milestone payments for Panbela as US WorldMeds continues its efforts to bring eflornithine to the market." "After many years investigating the role of polyamines and the therapeutic potential of eflornithine in neuroblastoma, it is rewarding to see USWM’s success with the FDA’s positive review of its NDA for eflornithine," said Elizabeth Bruckheimer, Ph.D., Vice President & Chief Scientific Officer of Panbela, "We are excited about how the USWM NDA approval of eflornithine should help address this high unmet need in patients with neuroblastoma."
Neuroblastoma, a rare cancer originating from immature nerve cells, contributes to nearly 15% of pediatric cancer deaths.[1] Panbela’s subsidiary, Cancer Prevention Pharmaceuticals, extensively collaborated with leading neuroblastoma research groups such as the Neuroblastoma Medulloblastoma Translational Research Consortium (NMTRC) (now Beat Childhood Cancer), New Advances in Neuroblastoma Therapy (NANT), the Children’s Oncology Group (COG), and the National Cancer Institute (NCI) in the clinical development of eflornithine as a treatment for neuroblastoma before the program was divested to USWM.