The European Medicines Agency (EMA) has recommended granting a marketing authorisation for Kanuma (sebelipase alfa), for the treatment of lysosomal acid lipase (LAL) deficiency, a rare life-threatening inherited condition.

Patients with this condition have a wide range of symptoms such as growth failure, enlarged liver, diarrhoea and malabsorption (when nutrients from food are not easily absorbed during digestion). There are different types of LAL deficiency with the disease in infants, also called Wolman disease, being the most severe. In its most severe form, the disease is usually fatal in the first year of life.

There is currently no approved therapy for this condition. Due to the lack of treatment and the high death rate in infants under the age of one, EMA’s Committee for Medicinal Products for Human Use (CHMP) decided to speed up the evaluation of this medicine and recommended marketing authorisation following an accelerated assessment. This mechanism is one of the Agency’s tools to speed up patient access to new medicines where there is an unmet medical need.

LAL deficiency occurs when the body does not produce enough active lysosomal acid lipase (LAL), an enzyme which breaks down fatty material. The lack of the LAL enzyme can lead to a build-up of fatty material in a number of vital body organs including the liver and blood vessels.

For more details, go to: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2015/06/news_detail_002352.jsp&mid=WC0b01ac058004d5c1