Netherlands-based AM-Pharma has received orphan drug designation from the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for its recombinant Alkaline Phosphatase (recAP) to treat hypophosphatasia (HPP).
HPP is a mineralisation disorder, which results in soft bones (rickets or osteomalacia) and in defects of teeth and periodontal tissues.
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The recAP is a proprietary recombinant human AP built from two naturally occurring human isoforms of the AP enzyme that is highly stable and active.
AM-Pharma CEO Erik van den Berg said: "We are delighted with the FDA’s and EMA’s decisions to grant recAP orphan drug status for hypophosphatasia, a devastating and potentially fatal disease.
"Whilst pursuing the primary indication for recAP in acute kidney injury, we are exploring the product’s potential in other indications. These new orphan designations help provide a strong grounding for recAP in hypophosphatasia."
recAP is currently in Phase II development for testing the potential treatment of acute kidney injury, with the potential to develop for hypophosphatasia.
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By GlobalDataAccording to the firm, an oral formulation for recAP is expected to be developed for the treatment of ulcerative colitis.
In April this year, Pfizer purchased a minority equity position in AM-Pharma and it has an exclusive option to acquire the remaining equity in the company.
Pfizer can exercise its option after the completion of a Phase II trial of recAP in sepsis-associated acute kidney injury.
