Amyotrophic Lateral Sclerosis Lacks New and Effective Treatment Options

Wednesday 6 March 2013, Amsterdam

Amyotrophic Lateral Sclerosis (ALS) is the most common type of Motor Neuron Disease (MND), yet the therapeutics market is woefully inadequate, states a new report by healthcare experts GlobalData.

ALS, also known as Lou Gehrig’s or Charcot disease, accounts for around 80% of all motor neuron cases worldwide. The progressive neurodegenerative disease causes the impairment and eventual death of the motor neurons in the spinal cord, brainstem, and motor cortex, which are responsible for communication between the nervous system and voluntary muscles. As motor neurons die, the brain loses the ability to control muscle action, progressively hindering voluntary movement, with patients in late stages of the disease sometimes becoming totally paralyzed. Disease progression and patient survival varies, but only 10% of those diagnosed with ALS live beyond a decade.

Patient numbers are set to rise, due to a growing elderly population and increasing disease awareness. Current treatment options are limited to off-label medications that offer symptomatic relief, and Rilutek (riluzole), the only approved disease-modifying treatment for ALS. Rilutek has been shown to slow disease progress and improve survival in ALS patients by three to four months, exhibiting disease-modifying characteristics. However, while it allows patients more time in higher-functioning states of the disease, it cannot cure the condition, and its low efficacy and numerous side-effects have restricted its use. Common side effects include nausea, dizziness, decreased lung function, diarrhoea, pneumonia and vertigo. Around 14% of ALS patients who received Rilutek in pre-marketing clinical trials discontinued treatment due to adverse reactions. A high unmet needtherefore remains for ALS treatments with a novel mechanism of action which can alter disease progression.

The ALS pipeline has a few late-stage therapies. Pipeline drugs that could potentially launch in the forecast period include Avicena’s ALS-02 (2015) and Mitsubishi Tanabe’s Radicut (edaravone) (Japan only, 2013). These products will expand the market, but will not transform the treatment paradigm, as they will be mainly used as add-on treatments to Rilutek. The most promising drug, Biogen’s dexpramipexole, was discontinued last month due to a lack of efficacy and improved survival in phase III trial results. The anticipated patent expiry of Rilutek in 2013 will therefore act as a major barrier to market growth, due to the lack of any upcoming therapeutics.

In 2011, the global ALS market was estimated at US$112m, but this value is expected to decline at a negative Compound Annual Growth Rate (CAGR) of 5.7% to hit an estimated US$70m by 2019.

Amyotrophic Lateral Sclerosis – Analysis and Market Forecasts to 2019

This report is an essential source of information and analysis on the global Amyotrophic Lateral Sclerosis Therapeutics market. It identifies the key trends shaping and driving the market, and provides insights on the prevalent competitive landscape and the emerging players expected to significantly alter the market positioning of the current market leaders.

This report was built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis conducted by GlobalData’s team of industry experts.