http://www.raredr.com/news/kanuma-infants-naspghan
Kanuma Improves Survival in Infants with LAL Deficiency

James Radke

In December 2015, Alexion’s Kanuma (sebelipase alfa) was approved for treating patients with lysosomal acid lipase (LAL) deficiency. The approval was based largely on a phase 3 study involving patients ranging from 4-54 years of age who were found to benefit from Kanuma.

But is the drug effective in infants who have the most severe form of the rare disease? The answer seems to be yes – at least according to new data presented at the North American Society of Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) Annual Meeting being held in Las Vegas this week.

LAL deficiency is a rare, life-threatening lysosomal disease due to mutations in the  LIPA gene that is responsible for the synthesis of the enzyme LAL. In the absence of LAL, there is an accumulation of lipids, especially in the liver, blood vessel walls, the intestinal tract, as well as other organs. As with many lysosomal diseases, the severity of the symptoms will depend on the amount of LAL that is diminished. In the most severe cases, babies with little or no LAL normally do not survive past the first few years. However, with the approval of Kanuma, the data presented at NASPGHAN shows that even the youngest patients with this rare condition can thrive.
 
The data showed that 8 in 10 infants given Kanuma prior to the age of 8 months are still alive. One infant died at 5 months and another at died at the age of 13.8 months. The causes of both deaths were considered to be unrelated to treatment with Kanuma. 

Each of the 8 surviving infants is over 1 year of age (as of August 2017, median age was 29.8 months [range, 16.5-39.4]). The oldest patient is now into his 4th year of being treated with Kanuma.

Other parameters noted in the assessment of these children were that they have gained weight and that the patients´ growth is significantly improving.

Treatment has also improved their dyslipidemia. While data is limited to 2 patients, their median low-density lipoprotein cholesterol (LDL-C) level at baseline was 118.8 mg/dL and by week 48, that had dropped by 47.5%. And in 3 patients that have had their high-density lipoprotein cholesterol (HDL-C) rose 33% during the first 49 weeks of treatment.

Serious adverse events related to sebelipase alfa were reported in 6 infants. All were resolved.

Per Alexion: “Prior to the availability of Kanuma the vast majority of infants with rapidly progressive LAL-D did not survive to their first birthday,” said John Orloff, M.D., Executive Vice President and Global Head of R&D at Alexion. “We are humbled to see that many of these young children are surviving when treated with Kanuma and also experiencing meaningful improvements in their disease symptoms.”

Image courtesy of wikimedia commons.

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