Rare Disease Report

Alexion Begins 2 Phase 3 trials for 2 Rare Diseases

OCTOBER 27, 2016
Andrew Black
Alexion Pharmaceuticals has gone to work. The company has initiated 2 Phase 3 trials for their drug ALXN1210, for 2 rare diseases, paroxysmal nocturnal hemoglobinuria (PNH), and aHUs. 

Their orphan drug ALXN1210, is a longer-acting anti-C5 antibody that inhibits terminal complement.

PNH Trial

The first trial is a is a Phase 3 open-label, multinational, active-controlled study evaluating ALXN1210 compared to eculizumab (Soliris) in complement inhibitor treatment-naïve patients with PNH 
 
Patients will receive a single loading dose of ALXN1210, followed by regular maintenance dosing every 8 weeks based on 3 weight cohorts. Patients in the eculizumab arm will receive 4 weekly induction doses, followed by regular maintenance dosing every 2 weeks.

 aHUS Trial

The second trial is a Phase 3, open-label, single arm, multinational study to evaluate the safety and effectiveness of ALXN1210 in complement inhibitor treatment-naïve adolescent and adult patients with aHUS. 
 
Like the PNH study, patients will receive a single loading dose of ALXN1210, followed by regular maintenance dosing every 8 weeks based on 3 weight cohorts.
 
Alexion expects to begin enrolling patients into these trials later this year, and plans to initiate a Phase 3 trial of ALXN1210 in pediatric patients with aHUS in 2017.

About paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder in which red blood cells break apart prematurely. It is an acquired hematopoietic stem cell disorder.
 
Some hematopoietic stem cells in individuals with PNH are defective and consequently produce defective blood cells. These defective red blood cells of PNH are extremely susceptible to premature destruction by a particular part of a person’s own immune system called the complement system.

About aHUS

Atypical hemolytic uremic syndrome (aHUS) is an extremely rare disease characterized by low levels of circulating red blood cells due to their destruction (hemolytic anemia), low platelet count (thrombocytopenia) due to their consumption and inability of the kidneys to process waste products from the blood and excrete them into the urine (acute kidney failure), a condition known as uremia.
 


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