FDA exercises rare disease flexibility

Further evidence of the FDA's willingness to exercise a degree of flexibility with respect to rare disease drugs….

US-based rare disease specialist biopharma, Ultragenyx, has announced that the FDA has agreed to the company’s proposal to submit an NDA submission for its experimental drug, UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), based on a variety of data. Following a pre-NDA meeting to be held in the next couple of months, it is anticipated that Ultragenyx may be able to submit an NDA for UX007 in H1, 2019.

Ultragenyx proposed that the FDA allow an NDA submission based on data from a Phase 2 trial completed in 2016 - a 78 week, single-arm, open-label study in 29 patients.  Supporting this data was further data from an earlier 4 month, randomized, controlled investigator study in 32 patients, a retrospective medical record review of 20 patients treated with UX007 as part of a compassionate use protocol and numerous emergency IND cardiomyopathy cases.

This was not Ultragenyx’s first proposal for early filing submitted to the FDA. The company has had ongoing discussions since last year with both the FDA and EMA, aimed at achieving early filing. Ultragenyx argued that its Phase 2 data showed a significant reduction in major clinical events whereas, up until this month, the FDA was reported to believe that the Phase 2 data were confounded and weren’t sufficient to support an NDA.

The news of the FDA’s change in position came as a welcome surprise for Ultragenyx, which said it had difficulty  articulating exactly what had changed for the regulators.


https://globenewswire.com/news-rele...ong-Chain-Fatty-Acid-Oxidation-Disorders.html

https://www.biocentury.com/printpdf/297635

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https://clinicaltrials.gov/ct2/show/NCT01886378?term=triheptanoin&draw=3&rank=29

http://ir.ultragenyx.com/news-relea...ounces-positive-data-phase-2-study-ux007-long

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https://www.cnbc.com/2013/09/16/glo...n-in-patients-with-long-chain-fatty-acid.html

https://www.fda.gov/downloads/About...fMedicalProductsandTobacco/CDER/UCM542141.pdf