Regulatory Focus™ > News Articles > Meeting to Focus on Regulatory Paradigm of Developing, Evaluating ALS Therapies

Meeting to Focus on Regulatory Paradigm of Developing, Evaluating ALS Therapies

Posted 30 January 2013 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) will soon hold a public meeting to obtain input on ways to enhance the development of new therapies to treat amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease.

The condition affects patients by degenerating their nerve cells, causing a loss of muscle control over some of the body's key functions, including breathing and swallowing, ultimately causing death. At present, there is no known cure for the disease, and only one medication-Rilutek (riluzole)-is FDA approved to mitigate the progression of the symptoms, according to the US National Library of Medicine. Even that drug only succeeds in prolonging survival by several months.

An estimated 20,000 patients in the US have the condition at any one time, with 5,000 diagnosed each year. FDA noted that only 10% of patients-the lucky few-survive longer than nine years, while the rest typically only survive 3-5 years.

FDA's Federal Register notice explains that ALS patients face "profound unmet medical needs," and the agency intends to facilitate the development of new therapies to the extent that it is able.

It is particularly interested in hearing from stakeholders on the agency's regulatory paradigms-" the way FDA regulates the scientific evaluation of, marketing authorization for, and postmarketing surveillance of, drugs for treatment of ALS."

The agency said it would accept presentations detailing nonclinical testing, clinical trials, decisions regarding marketing authorization, postmarketing surveillance and the diagnosis or treatment of the disease.

The meeting will be held on 25 February at FDA's White Oak campus in Silver Spring, MD. FDA added that due to the "debilitating effects" of ALS, it will be employing "all available measures to enable participation of people who are mobility-limited."


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Tags: ALS, Meeting

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