The Food and Drug Administration’s Oncologic Drugs Advisory Committee voted that US WorldMed’s eflornithine (difluoromethylornithine; DFMO) shows evidence of reducing the risk of relapse for pediatric patients with high-risk neuroblastoma.
The adcomm voted 14 to six on Wednesday afternoon that the Kentucky-based specialty pharma company’s results demonstrated evidence that eflornithine increased event-free survival for patients who received the oral treatment for two years, despite the fact that the data was based on a single externally controlled trial.
The reasoning among those who voted against the indication was due to the lack of a randomized clinical trial and the potential for the recommendation to become precedent-setting for other treatments down the line.
The FDA previously recommended a randomized clinical trial as the preferred path for registration for eflornithine during conversations with the drugmaker in 2015 and 2016. However, US WorldMeds opted for an externally controlled, single-arm Phase 2 study, calling it a “viable alternative” for the rare disease patient population.
The company said in its briefing document to the adcomm that its DFMO development program was conducted in accordance with agency guidance through the drug’s Orphan Drug Designation, Breakthrough Therapy Designation, Real Time Oncology Review Program and Project Orbis initiatives.
Ultimately, the majority of the adcomm members still voted ‘yes’ based on the study as well as other data and pieces of evidence presented by US WorldMeds.
Considered the most common cancer in infants, there are an estimated 700 to 800 new cases of neuroblastoma in the U.S. each year, according to the American Cancer Society. The rare disease is responsible for nearly 15% of pediatric cancer deaths.
Of note, Panbela Therapeutics divested assets within the eflornithine program, including the license to the ornithine decarboxylase inhibitor, to US WorldMeds over the summer for up to $9.5 million in non-dilutive funding.
Per terms of the deal, the Minneapolis-based company will receive payments from US WorldMeds upon the completion of milestones related to the drug’s clinical development, regulatory approval and commercial sales.
“This transaction strengthens and expands our neuroblastoma program data currently under
FDA review and builds upon our established partnerships to fully unleash the potential of
DFMO as a breakthrough treatment for neuroblastoma,” US WorldMeds CEO Paul Breckinridge Jones said in a statement at the time.
