Soliris granted orphan drug designation for new indication

In Clinical Development, Regulation & Government by Tim Peplow

alexion soliris indication fda orphan drug (eutrophication&hypoxia

Alexion Pharmaceutical’s Soliris is widely touted as the world’s most expensive drug, and now the Connecticut-based biopharmaceutical company has announced that the drug has been granted an orphan drug designation by the US FDA for the treatment of a new indication, neuromyelitis optica (NMO).

Soliris is a terminal complement inhibitor that is approved in several regions, including the US and EU, for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). The drug is currently not approved for the treatment of NMO in any country – indeed, there are currently no approved therapies for patients with NMO, a life-threatening, ultra-rare neurological disorder which can lead to paralysis, blindness and death.

Martin Mackay, executive vice president and global head of R&D at Alexion, said: “This orphan drug designation is a positive step toward understanding and meeting the needs of this underserved patient population. In clinical trials to date, terminal complement inhibition with Soliris appeared to significantly reduce the attack rate in patients with severe, refractory relapsing NMO.”

While Soliris is currently Alexion’s only marketed drug, the company also recently received orphan drug designation for another product, asfotase alfa, for the treatment for hypophosphatasia. The company have several other drugs in early stage trials, alongside working on expanding the indications for Soliris in the neurological and nephrological spaces.

Martin Mackay, executive vice president and global head of R&D at Alexion, will be speaking at the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.

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Read the press release from Alexion >