AstraZeneca (NASDAQ: AZN, LON: AZN) , a leading UK pharmaceutical company, has received approval for its rare disease therapy Strensiq (asfotase alfa) from the Guangdong Medical Products Administration (MPA) bureau. This clearance allows Strensiq to be introduced to medical institutions under the “Hong Kong-Macau Drug and Device Access” policy, a component of the Greater Bay Area initiative that facilitates patient access to medications across Hong Kong, Macau, and several cities within Guangdong. The therapy serves as a long-term enzyme replacement for patients diagnosed with hypophosphatasia (HPP), specifically addressing the skeletal symptoms of the condition.
HPP is an exceedingly rare inherited metabolic disorder characterized by a deficiency in alkaline phosphatase (ALP), which is pivotal for bone mineralization and osteopontin synthesis. This deficiency can result in impaired bone growth and reduced bone strength, alongside a range of complications such as fractures, bone pain, mobility and respiratory issues, epilepsy, and a significantly diminished quality of life.
Asfotase alfa made history as the first and only enzyme replacement therapy for HPP when it was initially approved in Japan in July 2015. Since then, it has garnered marketing approvals from regulatory bodies in the European Union and the United States, solidifying its position as a groundbreaking treatment for HPP patients.- Flcube.com
