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FDA grants orphan drug status to Treeway's ALS treatment

Amyotrophic Lateral Sclerosis drug TW001 gainsregulatory benefits

FDA HQ 

Dutch biotech company Treeway has won orphan drug status for its investigational treatment for ALS (Amyotrophic Lateral Sclerosis).

The Rotterdam-based firm, which as founded by entrepreneurs Bernard Muller and Robbert Jan Stuit, both diagnosed with ALS, gains a range of financial incentives and marketing exclusivity benefits for its lead pipeline compound TW001.

Ronald van der Geest, chief development office at Treeway, said: “Obtaining orphan designation for TW001 from the FDA is an important milestone for Treeway.

"The orphan drug designation will help us further to align with FDA on the acceptability of a US clinical study programme, and to profit from possible financial incentives related to the orphan designation status."

These include reduced clinical trial costs, a waiver of around $2m in user fees for the product's future NDA submission and seven years of marketing exclusivity for the drug.

The FDA's decision follows that of the European Medicines Agency, which in November 2014 granted TW001 an orphan designation.

ALS, which is commonly known as Lou Gehrig's disease in the US and as motor neurone disease in certain other countries, causes muscle weakness, disability and eventually death.

The disease received a major boost in awareness last year due to the ALS ice bucket challenge, but to date there is no cure for it.

Moreover, the only one approved treatment for ALS - Sanofi's Rilutek (riluzole) - was launched in the 1990s and provides only a modest benefit on survival in the disease, and research into further treatments has so far been unsuccessful - with Biogen's dexpramipexole one of the most recent disappointments.

Article by
Dominic Tyer

24th March 2015

From: Research

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