PNH

Rare Blood Disease Drug Trialled for Use with Other Conditions

A pharmaceutical company, Alexion Pharmaceuticals that makes a single product Soliris® that is used as a therapy for the very rare chronic blood disease Paroxysmal Nocturnal Hemoglobinuria (PNH) have stated that they are testing the use of the drug as a therapy for up to a further 8 rare diseases, these include:

• Atypical Hemolytic Uremic Syndrome (aHUS)
• Membranoproliferative glomerulonephritis (MPGN)
• Cryopyrin Associated Periodic Syndromes (CAPS)

Leonard Bell, M.D., CEO of Alexion said,

“Research presented this week in Innsbruck suggests that Soliris®  has the potential to benefit patients with a variety of ultra-rare genetic diseases characterized by the presence of TMA,” and “Our primary focus is to diligently bring Soliris®  to patients with PNH in a growing number of countries worldwide. We recognize the often devastating outcomes for patients who have very few and limited therapeutic options, and we are committed to evaluating the safety and efficacy of Soliris®  in these diseases where we believe complement inhibition could have a dramatic impact on patients’ lives.”

This is good news for many of the rare conditions that share a common pathology. Trials continue, so far the research indicates that the results are promising and bring hope to patients that at the moment have very few alternative therapies at their disposal.

The benefits of Soliris® as a therapy far seem to far outweigh any side effects that may be caused. What is more significant is that Soliris® does not interfere with many of the other drugs used for conditions like PNH such as the anticoagulant Warfarin.  Soliris® inhibits the complement system. As an inhibitor if treatment is halted the blood cells will again start to be destroyed.

For this reason, the fact that warfarin is being taken and because the condition is, so rare it is suggested that a medical alert bracelet be worn to alert first responders in the case of a medical emergency.

Resources:

Announcement on Trading Markets

Alexion

PNH Source

CAPS Community

National Organization for Rare Diseases – aHus

Blogs:

Strong Quarter for Alexion – Analyst Blog | Stock Market News …