Thanks to Alexion Pharmaceuticals, two ultra-rare diseases finally got the first treatment in the European Union, namely Lysosomal acid lipase deficiency (LAL-D) and Pediatric-onset hypophosphatasia.
Alexion ramps up its product-pipeline. Until now, the Connecticut-based company, which comprises a market cap of about $38.5Bn (€34,2Bn), has lived mostly on sales of Soliris, the most expensive drug on the planet (up to $700,000 a year). In just the second quarter of 2015, Soliris cashed in $636M ($566M).
However, the biotech just proved it is not a one-hit-wonder. After they got positive feedback from the EMA in July, two therapies treating respective Pediatric-onset hypophosphatasia and Lysosomal acid lipase deficiency become the first approved treatment in the European Union. Fewer than 20 out of 1 million people are affected by the diseases, but those who are unlucky suffer devastating consequences.
LAL-D is caused by a genetic mutation that leads to the loss of LAL enzyme activity across multiple tissues. Without it, cholesteryl esters and triglycerides are not removed and can lead to multi-organ damage and premature death.