Quick Facts
According to DelveInsight’s analysis, in 2023, the total diagnosed prevalent cases of hypophosphatasia, an ultra-rare disease, was approximately 6,200 in the 7MM. While almost 85% have less severe forms of the disease, severity may vary based on genetic and environmental factors.
Due to the low number of diagnosed cases, few companies have shown interest in developing treatments for this condition. To date, only one drug, STRENSIQ, has been approved. However, a few others are in clinical trials, including Alexion’s, a subsidiary of AstraZeneca, ALXN1850 (efzimfotase alfa), AM-Pharma’s Ilofotase alfa, and PuREC’s REC-01.
Historically, developing d...
