Ultra-rare specialists Alexion have announced that the FDA has granted a Breakthrough Therapy designation to cyclic pyranopterin monophosphate (cPMP), also known as ALXN1101. The drug is an enzyme co-factor replacement therapy for molybdenum cofactor deficiency (MoCD) type A.
The FDA says the Breakthrough Therapy designation is “intended to expedite the development and review of drugs for serious or life-threatening conditions”. The new regulatory pathway could slash the time taken for a new drug to be approved. The process is moved on as quickly as possible, with communications with the FDA taking minutes instead of weeks or months.
MoCD type A is a severe and life-threatening, ultra-rare, genetic metabolic disorder that can cause irreversible neurologic damage within the first weeks of life.
Martin Mackay, Global Head of R&D at Alexion, who is also speaking at this year’s World Orphan Drug Congress Europe 2013, said “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.”
The World Orphan Drug Congress Europe brings together pioneers from the orphan drug community, including key opinion leaders from the payer, HTA, public health and patient advocacy bodies. Download the brochure for the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.
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