Asfotase alfa
- Generic Name
- Asfotase alfa
- Brand Names
- Strensiq
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1174277-80-5
- Unique Ingredient Identifier
- Z633861EIM
- Background
Asfotase alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzy...
- Indication
Indicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
- Associated Conditions
- Infantile-onset Hypophosphatasia, Juvenile-onset Hypophosphatasia, Perinatal-onset Hypophosphatasia
- Associated Therapies
- -
How Alexion markets a rare disease drug by 'starting with the end in mind'
Alexion, now part of AstraZeneca, faces challenges in commercializing rare disease drugs due to small patient populations and regulatory hurdles. Scott Weintraub emphasizes the importance of early integration of R&D and commercial strategies. Alexion's mission includes patient-centric development, exemplified by drugs like Strensiq and Ultomiris, which aim to reduce injection frequency and improve patient quality of life. With AstraZeneca, Alexion gains access to advanced diagnostics and global markets, enhancing its ability to address rare diseases and contribute to AstraZeneca's ambitious growth goals.
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