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Neuren Pharmaceuticals seeks “orphan drug” status for Rett Syndrome treatment

Last updated: 16:30 30 Dec 2014 AEDT, First published: 15:30 30 Dec 2014 AEDT

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Neuren Pharmaceuticals (ASX:NEU) has submitted applications to the US Food and Drug Administration (FDA) for Orphan Drug designation and Breakthrough Therapy designation for its NNZ-2566 treatment for Rett Syndrome.

Orphan Drug designation is a special status that the FDA may grant to a drug to treat a rare disease or condition.

It qualifies the sponsor of the drug for seven years of marketing exclusivity and various development incentives including waiver of the prescription drug user fee for a marketing application.

The FDA has previously granted Orphan Drug designation to Neuren for NNZ-2566 in Fragile X syndrome.

The company is well funded with cash reserves of about $21 million.


Rett syndrome


In November 2014, Neuren announced top-line results from its Phase 2 clinical trial in Rett syndrome, which successfully demonstrated clinical benefit from treatment with NNZ-2566.

The benefit observed in the trial encompassed many of the core symptoms of Rett syndrome and was observed in both clinician and caregiver assessments.

It expects to meet with the FDA in the first half of 2015 to discuss the remaining requirements for the development of NNZ-2566 in Rett syndrome.


Fragile X syndrome

To date, 22 subjects have commenced treatment in Neuren’s Phase 2 trial of NNZ-2566 in Fragile X syndrome, 19 of which have already completed.

The trial represents a significant commitment for families, with only 2 subjects entering the trial during the U.S. holiday period in November and December.

Neuren is taking a number of steps to accelerate enrolment into the trial, including working closely with advocacy organisations to ensure that the recent results of the trial of NNZ-2566 in Rett syndrome are widely communicated.

The number of trial sites enrolling will increase from 6 to 9 in January 2015 and further sites will be added to expand the geographical coverage in the United States.

Neuren is also currently investigating the feasibility of extending the trial to include sites in Australia

Top-line results from the trial that were targeted for the second quarter of 2015, are now expected in the second half of 2015.



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