Alexion Pharma International SÃ rl has received a breakthrough therapy designation from the US Food and Drug Administration (FDA) for its cyclic pyranopterin monophosphate (cPMP, ALXN1101), an enzyme co-factor replacement therapy to treat patients with molybdenum cofactor deficiency (MoCD) type A.
The breakthrough therapy designation is part of the FDA Safety and Innovation Act of 2012 and was aimed at expediting the development of a drug to treat a serious or life-threatening disease when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.
The company seeks to work closely with the FDA and obtain its guidance in the development of ALXN1101 for the treatment of patients with MoCD Type A, as well as receive advice on generating evidence needed to support approval of the drug.
In addition, the company has also started a natural history study in MoCD Type A patients and completed dosing with the synthetic cPMP in a study in healthy volunteers.
ALXN-1101 is designed to replace the deficient cPMPs thereby resolving the metabolic abnormalities and resulting in dramatic clinical improvement.
Alexion executive vice-president and global head of R&D Martin Mackay said in designating cPMP as a breakthrough therapy, the FDA recognises the life-threatening nature of MoCD Type A and the lack of any effective treatment options for infants born with this disease.
"ALXN1101 is an innovative approach to the treatment of MoCD Type A, as it targets an essential step in the pathophysiology of the disease and the underlying cause of the disease by replacing the naturally occurring cPMP molecule, which is lacking in patients with MoCD Type A," Mackay said.
The company believes that the cPMP replacement therapy could be an effective treatment option for MoCD type A, as the therapy induced MoCD production in the infant’s body can help eliminate the toxic sulfite and result in the efficient uptake of cPMP and restoration of molybdenum cofactor-dependent enzyme activities.
MoCD type A is a severe, ultra-rare, genetic metabolic disorder that causes catastrophic and irreversible neurologic damage within the first weeks of life.
It affects newborns in which a genetic deficiency of cPMP results in the inability of the body to form an essential cofactor called molybdenum cofactor, resulting in its absence.
The cofactor is essential for suitable functioning of several critical metabolic enzymes and its absence resulting in build-up of sulfite in the brain may lead to damage and destruction of nerve cells, brain swelling, uncontrollable seizures, severe brain damage, and death.
