FDA批准阿福他酶α(asfotase alfa)用于围产期、婴儿和青少年发病的低磷酸酯酶症

Asfotase alfa is a tissue non-specific alkaline phosphatase replacement therapy. It was developed by Alexion Pharmaceuticals in the United States. It was approved by the US Food and Drug Administration (FDA) for marketing on October 23, 2015. Strensiq received the title of breakthrough therapy, and the FDA also granted aftase alfa the orphan drug designation.

Asfotase alfa approved indications

1. Hypophosphatasia

On October 23, 2015, the US Food and Drug Administration approved asfotase alfa as the first approved drug for the treatment of perinatal, infant and adolescent-onset hypophosphatasia (HPP). Asfotase alfa is the first and only treatment for perinatal, infant and adolescent-onset hypophosphatasia.

2. About hypophosphatasia

Hypophosphatasia is a rare hereditary progressive metabolic disease that can have devastating effects on multiple systems of the body, leading to severe disability and life-threatening complications. It is characterized by defects in bone mineralization that may lead to rickets and bone softening, leading to skeletal abnormalities. It can also cause complications such as severe muscle weakness, loss of mobility, seizures, pain, respiratory failure, and premature death.

Important information about asfotase alfa

1. Frequency of asfotase alfa injection

Asfotase alfa needs to be injected 3-6 times a week. It works by replacing the enzyme (tissue non-specific alkaline phosphatase) responsible for the formation of essential minerals in normal bones. It has been clinically proven to improve patient prognosis.

2. Side effects of asfotase alfa

The most common side effects in patients receiving asfotase alfa include injection site reactions, hypersensitivity reactions (such as dyspnea, nausea, dizziness, and fever), lipodystrophy (loss of fat tissue leading to skin indentations or thickening of fat tissue leading to subcutaneous masses) at the injection site, and ectopic calcification of the eyes and kidneys.