Soliris Grabs A Sixth Orphan Drug Designation

In Regulation & Government by Cameron


Alexion Pharmaceuticals’ blockbuster orphan drug, Soliris, scooped a sixth Orphan Drug Designation last Friday for Myasthenia Gravis.

This sixth designation for Soliris (eculizumab) sees Alexion’s blockbuster treat the debilitating  neurological disorder Myasthenia Gravis (MG). MG is a neurological disorder caused by uncontrolled complement activation caused tissue damage, and interference between muscle fibres and the nerve-muscle junction. This interference is the result of auto-antibodies that targets specific parts of the nerve-muscle junction.

Patients with MG initially experience weakness in their eye muscles, which typically progress towards a more sever and general form affecting places such as the head, spine, limbs, and respiratory muscles.

Soliris, across its six designations, is by far and away the biggest earner for Alexion. But, not wishing to put its eggs all in one basket, the developer is developing two seperate treatments for orphan indications: Asfotase alfa for Hypophosphatasia; and Cyclic Pyranopterin Monophosphate (cPMP) for Molybdenum Cofactor Deficiency (MoCD) Type A.

Alexion have also recently found themselves issuing three separate recalls for batches of Soliris in less than a year. The recalls, all voluntary, were issued after visible particles were seen in vials during testing by the company.

Soliris has received designation for 5 other indication including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome  aHUS, Shiga-Toxin producing Escherichia Coli Hemolytic Uremic   Syndrome, NeuroMyelitis Optica, and the prevention of delayed graft function after renal transplantation.