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Alexion Pharmaceuticals: Soliris Gains Another FDA Orphan Drug Designation

June 27, 2013

Alexion Pharmaceuticals’ orphan drug Soliris (Eculizumab) receives on June 24, 2013, another FDA Orphan Drug Designation (ODD), for the treatment of NeuroMyelitis Optica (NMO). According to, after the close of the markets on June 26, 2013, Alexion Pharmaceuticals ends the day up almost 5%.

NMO, also known as Devic’s Disease, involves an attack of the spinal cord and optic nerves by the body’s own immune system. It is similar to Multiple Sclerosis “in that it can leave patients with weakness or paralysis in the legs and arms, decreased vision, and loss of bladder control.” NMO is where immune system cells and antibodies attach and destroy myelin, a fatty substance that surrounds nerve fibers and helps nerve signals move from cell to cell. As a result, patients develop optic neuritis, which causes eye pain and vision loss. Soliris is currently being evaluated in an investigator-initiated Phase 2 trial in patients with NMO.

Soliris is Alexion Pharmaceuticals’ only marketed product and generates blockbuster profits from multiple indications for ultra-rare diseases. Soliris is one of the world’s most expensive drugs, costing approximately $409,500/year/patient. Soliris treats ultra-rare diseases such as :

•   Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare genetic blood disorder (approved in 40 countries)
•   Atypical Hemolytic Uremic Syndrome (aHUS), an ultra-rare genetic disorder (approved in US and EU).

Alexion Pharmaceuticals is planning on expanding the Soliris franchise with the following 5 additional indications :

  2. Neuromyelitis Optica (NMO)
  3. MG Kidney transplant
  4. Delayed transplant graft function
  5. Myasthenia Gravis.


Alexion Pharmaceuticals Pipeline.

Please Note: FDA Official Logo from FDA website.

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