Rare Daily Staff

The U.S. Food and Drug Administration issued a Complete Response Letter in response to the New Drug Application for treosulfan submitted by Medac GmbH, according to licensor Medexus Pharmaceuticals, a company focused on commercialization of rare disease treatment solutions in North America.

The NDA was submitted for use of treosulfan in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT), and both Medexus and medac had been optimistic that a positive decision from the FDA in connection with a planned Prescription Drug User Fee Act (PDUFA) date on August 11th would allow for a commercial launch of treosulfan in United States later this year.

However, the FDA has determined that it cannot approve the NDA in its present form and has provided recommendations specific to additional clinical/statistical data and analyses pertaining to the primary and secondary endpoints of the completed pivotal phase 3 study. Medexus and medac are reviewing the letter to determine the appropriate course of action and will work closely with the FDA to understand and address their comments.

“Given the recent Health Canada approval, European Medicines Agency approval in 2019, as well as supporting data from more than 100 publications, we were all surprised by the FDA’s response,” said Ken d’Entremont, CEO of Medexus. “That being said, Medexus and medac look forward to continuing to work with the FDA to address their requests in a timely manner, and we remain optimistic for a future, albeit delayed, approval of treosulfan in the United States, complete with Orphan Drug designation. The current standard of care is not suitable for numerous at-risk groups, due to the high toxicity effects, and treosulfan has demonstrated excellent survival data among those groups. We are hopeful that our future communications with the FDA will result in a positive outcome, and we look forward to providing further updates in due course.”

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