A lifeline for rare disease patients

For more than 25 years Alexion has been blazing a trail in rare diseases, bringing a series of life-changing medicines to market. pharmaphorum’s Richard Staines spoke with Sean Richardson, general manager, UK & Ireland and Charles de Wet, medical lead, UK, Ireland, Nordics & Netherlands, to hear about the challenges developing rare disease drugs, getting them to market, and the life-changing value they have for patients.

Alexion has been developing life-transforming drugs for patients with rare and ultra-rare diseases for more than 25 years. The company has a wealth of experience and is most famous for the rare disease drug eculizumab (Soliris), which has been approved in paroxysmal nocturnal haemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS) and refractory generalised myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive.

But it has also had other rare disease drugs and indications for existing drugs approved in recent years, and has a pipeline stocked with several other potential therapies.

Alexion’s pipeline includes potential new treatments for copper metabolism disorder Wilson disease, and the neurological disorder neuromyelitis optica spectrum disorder (NMOSD).

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