Market Overview

Alexion Announces FDA Breakthrough Therapy Designation for cPMP Replacement Therapy for Patients with MoCD Type A


Alexion Pharma International Sàrl, a subsidiary of Alexion
Pharmaceuticals, Inc. (NASDAQ: ALXN), today announced that the U.S. Food
and Drug Administration (FDA) has granted a Breakthrough Therapy
designation to cyclic pyranopterin monophosphate (cPMP, or ALXN1101), an
enzyme co-factor replacement therapy for the treatment of patients with
molybdenum cofactor deficiency (MoCD) type A, a severe and
life-threatening, ultra-rare, genetic metabolic disorder that causes
catastrophic and irreversible neurologic damage within the first weeks
of life.

According to the FDA, a Breakthrough Therapy designation is designed to
expedite 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

See full press release

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