Shareholders in Neuren Pharmaceuticals Ltd (ASX: NEU) are rejoicing after one of the company's drugs, which shows promise in treating a rare genetic disorder, received fast-track designation from the US Food & Drug Administration (FDA).
Neuren told the ASX in a statement on Monday that its compound, NNZ-2591, had been granted the tick by the FDA, which would allow it to speed up the process of testing its compound and hopefully bringing it to market.
As the company explained:
Fast Track is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Currently there are no FDA-approved treatments for PMS (Phelan-McDermid Syndrome).
The biotechnology company said it had recently started a randomised, Phase 3 trial of the compound in children aged 3-12.
Alignment was reached with the FDA on the single Phase 3 trial design and endpoints to support a New Drug Application.
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Rare disorder targeted
Phelan-McDermid Syndrome is a genetic disorder that affects how a person's brain develops and functions, and is triggered by a defect on the 22nd chromosome.
The syndrome results in intellectual and physical disabilities, and according to the US-based Phelan-McDermid Syndrome Foundation, most sufferers require long-term care and medical attention.
Neuren Chief Executive Officer John Pilcher said the company was very pleased to have been granted fast-track designation.
Neuren's Koala trial is the first ever Phase 3 clinical trial for PMS, which we hope may lead to a much-needed treatment for this community. We encourage all initiatives to increase awareness and diagnosis of PMS and applaud the leadership of both the Phelan-McDermid Syndrome Foundation and CureSHANK.
The NNZ-2591 compound has also been granted fast-track designation for its use in Angelman Syndrome.
The company said the benefits of the fast-track designation included more frequent meetings with the FDA to discuss the drug's development plan, more frequent communication about such matters as the design of clinical trials, and eligibility for accelerated approvals.
It also meant the compound was subject to rolling review, "which means that a drug company can submit completed sections of its New Drug Application for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed''.
Neuren shares hit a 12-month high of $22.86 on the news on Monday, before settling back to be 9.3% higher at $22.57.
The company's share price has more than doubled from lows of $8.61 per share over the course of the past year.
