Medexus resubmits NDA as it still looks to repurpose a decades-old drug
The German drugmaker medac’s US subsidiary is hoping the second time is the charm for the company, as it is officially asking the FDA once again to approve its cancer drug.
Medexus Pharmaceuticals resubmitted its request for approval of treosulfan to be taken in combination with fludarabine, the company said Monday. The treatment is designed as a regimen for allogeneic hematopoietic stem cell transplantation.
Medexus’ submission includes updated data and a reexamination of its Phase III trial as per the FDA’s request. The resubmission has been in the works by Medexus and medac since April.
Its Phase III data published in The Lancet showed that treosulfan was no worse than busulfan. The trial even showed a small, albeit not statistically significant benefit for treosulfan: 64% of patients who received medac’s drug went two years without relapse or death, compared to 54% of patients who received busulfan.
“We are encouraged by the recent publication of the final study results and analysis of the pivotal phase 3 clinical trial of treosulfan conducted by Medac, which met its primary endpoint and key secondary endpoints. An FDA approval within a two- to six-month period from the acceptance date would then pave the way for a commercial launch of treosulfan in the United States in the first half of calendar year 2023,” said Medexus CEO Ken d’Entremont in a statement.
While the FDA did not specifically disclose its original objections, only that more data and analysis were needed, the hit was a surprise to the company. Treosulfan was approved by Health Canada last year, and commercially launched later the same year. The EMA also approved the drug in 2019.
However, the news did see its share price drop by 37% in 2021. The shares now hover above the penny stock zone at $1.46 — a 30% drop since January.
Treosulfan has been around in some form for several decades at this point. Research into the compound’s potential to thwart cancer began almost 60 years ago and testing in humans began in 1968. With trial fits and starts over the decades, the drug never became used widely but did win approval to treat ovarian cancer.
In the 2000s, medac began developing the drug as a conditioning agent, stating that it could prove less toxic to patients than the widely-used conditioning agent busulfan.