Is the Future Outlook of ALS Treatments Grim?

Many companies, including Biogen Idec and now Novartis, are investing in drug development for rare and orphan diseases. This looks like a safe bet. Based on the recently published IMS Institute for Health Informatics report, “The Global Use of Medicines: Outlook Through 2016,” the number of new molecular entities (NMEs) for the treatment of rare and orphan diseases is projected to account for about 33% of all NMEs by 2016. However, despite this good news, if nothing is done to resuscitate the ALS development pipeline, the projected outlook for ALS therapies is grim. In GlobalData’s recent report “Amyotrophic Lateral Sclerosis (ALS) – Analysis and Market Forecasts to 2019,” the projected market for ALS is expected to decrease from $112 million in 2011 to $70 million by 2019. The report cites the lack of late stage therapeutics in the current pipeline, the recent failure of Biogen’s Phase III study of dexpramipexole, and Sanofi’s Rilutek patent expiration this upcoming year as key contributors to the market decline. Prize4Life’s $1M ALS Treatment Prize and Mouse Colony initiatives were launched to change this gloomy picture by providing some life support to the ALS drug development pipeline.

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