Alexion launches a duo of new therapies for ultra-rare diseases in the EU

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.

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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. Alexion’s innovative treatment, Kanuma, replaces the insufficient levels of enzyme, which has a great effect on the patients. Amazingly, 6 out of the 9 infant patients survived beyond 12 months, compared to none of the 21 untreated patients.

The second approved therapy, Strensiq, is also an enzyme replacement therapy. Strensiq treats the bone deformities caused by pediatric-onset hypophosphatasia. These are a result of genetic mutations in a certain phosphatase that is normally expressed in osteoblasts and chondrocytes. As a result, bones are deformed, muscles become profoundly weak and respiratory failure can lead to premature death in infants. Strensiq demonstrated rapid and sustained improvements in bone mineralization as well as in skeletal structure.

The approvals add another strong bond to Alexion’s relation with the EU, in which the US company is currently expanding – for example, a new Parisian R&D center was opened in June. Plus, Alexion is currently building a manufacturing facility in Ireland, which is the first of its kind outside the United States. Just another indication that Europe is attractive for drug makers!

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