Other Therapeutic Candidates

Metabolic Disorders

Alexion is developing a highly innovative, first-in-class therapy for the treatment of hypophosphatasia (HPP), an ultra-rare, life-threatening, genetic metabolic disorder that leads to progressive damage to multiple vital organs. Alexion is also developing a highly innovative therapy for the treatment of molybdenum cofactor deficiency (MoCD) type A, a severe and life threatening, ultra-rare, genetic metabolic disorder that causes catastrophic and irreversible neurologic damage within first weeks of life. There are no approved treatment options for HPP or MoCD.


Hypophosphatasia (HPP) is a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death. It is caused by a genetic deficiency of an enzyme known as tissue non-specific alkaline phosphatase (TNSALP), which causes life-long abnormalities in metabolism of the two vital minerals calcium and phosphate, leading directly to the debilitating morbidities and premature mortality of the disease.

Alexion is developing an investigational drug known as asfotase alfa, a first-in-class recombinant protein that addresses the underlying cause of HPP by targeting replacement of the missing enzyme to the necessary body tissues. Alexion recently received a Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for asfotase alfa, which is designed to normalize the genetically defective metabolic process and prevent or reverse the severe and life-threatening complications of life-long dysregulated mineral metabolism in patients with HPP.

Learn more about HPP

Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is a severe, ultra-rare and genetic metabolic disease affecting newborns in which a genetic deficiency of cyclic pyranopterin monophosphate (cPMP) results in the inability of the body to form an essential cofactor called molybdenum cofactor, resulting in its absence. This cofactor is essential for the appropriate functioning of several critical metabolic enzymes. A deficiency or absence of this cofactor leads to accumulation of toxic molecules, including the neurotoxin sulfite. Clinically, the absence of this cofactor and the resulting build-up of sulfite in the brain leads to damage and destruction of nerve cells, brain swelling, uncontrollable seizures, catastrophic and irreversible brain damage, and ultimately, death. There are currently no treatment options for patients with MoCD Type A.

Alexion is working with research collaborators to accelerate the development of an investigational therapy that would replace the missing cPMP in infants with MoCD type A so that their bodies can eliminate sulfite normally. Encouraging early clinical experience with this cPMP replacement therapy has been reported by independent investigators in Germany and Australia.

Inflammatory Disorders

Alexion is also investigating new therapeutic agents designed to regulate the complement inflammatory pathway. These include ALXN1007, a novel anti-inflammatory antibody designed to target severe and ultra-rare inflammatory disorders. Based on the outcome of early-stage clinical trials of ALXN1007 in healthy volunteers, we expect to continue development of ALXN1007 in multiple severe, life-threatening and ultra-rare conditions.