Hypophosphatasia drug Strensiq is first introduced in the Greater Bay Area under the “Hong Kong and Macao Drug and Device Access” policy – Sina Hong Kong

The hypophosphatasia drug Strensiq is first introduced in the Greater Bay Area under the “Hong Kong and Macao Drug and Device Access” policy.

Make new drugs more accessible to patients suffering from this rare disease

AstraZeneca China, relying on the “Hong Kong and Macao Use of Drugs and Devices” policy, announced the innovative drug Strensiq (generic name: asforzyme) in the field of rare diseasesaInjection / asfotase alfa) has been approved by the Guangdong Provincial Food and Drug Administration and has been officially introduced to designated medical centers in the Greater Bay Area for use as a long-term enzyme replacement therapy. for patients with infantile onset hypophosphatasia (HPP). , to treat skeletal manifestations of the disease. This first approval in the Greater Bay Area recognizes that the advanced clinical use of the drug enables mainland children to use the drug at designated medical centers in the Greater Bay Area without leaving the country. , which will reduce the problem of Chinese patients in crisis. of medicine.

Hypophosphatasia is a progressive hereditary metabolic disorder very rare breathing, epilepsy, etc., which may even cause lifelong burdens and greatly affect the quality of life.[1].

As the first and only enzyme therapy* in the world approved for the treatment of hypophosphatasia[2]sourceaThe active substances in the injection can replace the alkaline phosphatase that is missing in the patient’s body and serve as a long-term enzyme replacement therapy to control the patient’s symptoms1. Four prospective, open-label clinical trials evaluated and confirmed the efficacy of asphorase in patients with hypophosphatasia of infancy, childhood, or early childhood.aClinical efficacy and safety of injections. A Kaplan-Meier analysis of overall survival in patients with perinatal/infancy-onset hypophosphatasia treated with the drug (37 patients) compared to historical control patients with hypophosphatasia (48 patients) found that SulfaseaInjections can significantly increase patient survival rates. Studies have shown that through asferaseaThe survival rate of patients treated with injections was 95% at the age of 1 year, while the survival rate of patients in the historical control group was 42%;aThe survival rate of patients with injection treatment at 3.5 years of age was 86%, while the survival rate of patients in the historical control group was 27%;aThe survival rate of patients injected at age 5 was 82%, compared to 27% of patients in the historical control group[3]. For babies and young children, asphoraseaInjections help reverse the symptoms of hypomineralization[4],[5],[6]. In pediatric patients, asphoraseaInjections may help patients achieve a complete or substantial cure for rickets associated with hypophosphatasia[7].

Enzyme replacement therapy is an internationally recognized effective treatment method for the cause of hypophosphatasia, especially for young patients and patients with obvious symptoms, and can be used as the first treatment method of choice[8]however, access to related drugs has always been a major problem for patients. This time swallowaThe injection was the first to be approved in the Greater Bay Area, which is a major breakthrough in the treatment of hypophosphatasia in China and gives new treatment hope to domestic children with hereditary hypophosphatasia.

Lin Xiao, General Manager of AstraZeneca’s China Biopharmaceutical Business and Head of Hong Kong and MacauSaid: “With over 30 years of experience in China, AstraZeneca has a deep understanding of the unmet needs of Chinese patients. This time, AstraZenecaaBenefiting from support from the ‘Hong Kong and Macao Drug and Device Connect’ policy, the injection was first approved for introduction in the Greater Bay Area, shortening the treatment time for Chinese patients with hypophosphatasia to use global innovative drugs. In the future, we will continue to work with all partners to bring more innovative drugs globally that are urgently needed by patients into the country through the Bay Area Great, benefiting more Chinese patients. “

Hu Yiqing, Vice President of AstraZeneca China and Head of Rare Disease DivisionSaid: “‘Patient-century’, AstraZeneca not only actively participates in the field of rare diseases and continues to research the field of rare diseases, but is also committed to joining to various parties to solve the drug-free dilemma for Chinese rare disease patients, and to improve access to innovative drugsaThe injection was first approved for introduction in the Greater Bay Area, allowing Chinese patients with hypophosphatasia to quickly access the world’s leading drug. In the future, AstraZeneca will continue to place great emphasis on the needs of patients with hypophosphatasia and other rare diseases, will take a number of steps to accelerate the introduction and availability of innovative drugs, and will integrate all party to support the construction and development of rare disease diagnosis and treatment in China’s ecosystem, let’s walk hand in hand and light the light of life. “

* As of August 2024, retrieved from www.fda.gov, www.ema.europa.eu, https://strensiq-hcp.com/

Disclaimer: The drugs and indications contained in this information have not been approved in mainland China.

About hypophosphatasia

Hypophosphatasia is an inherited, chronic, progressive and life-threatening metabolic disease that has devastating effects on multiple body systems, leading to debilitating or life-threatening complications.[9]. Hypophosphatasia is characterized by low activity of alkaline phosphatase (ALP) and defective bone mineralization, which can cause bone destruction and deformity and other skeletal abnormalities, as well as systemic problems such as myasthenia and respiratory failure, leading to death premature in babies 4,8 ,[10].

About AstraZeneca

AstraZeneca (LSE / STO / Nasdaq: AZN) is a global science biopharmaceutical company focused on the research, development, production and marketing of prescription drugs, with a focus on oncology, rare diseases and cardiovascular, renal and metabolic, respiratory and immunological. diseases AstraZeneca’s global headquarters are located in Cambridge, UK, with operations in more than 100 countries worldwide.

About AstraZeneca China

Since AstraZeneca entered China in 1993, it has focused on the treatment areas with the most urgent needs for Chinese patients, including oncology, cardiovascular, kidney, metabolism, respiratory, digestive, diseases rare, vaccine antibodies and autoimmunity, etc., and has developed more than 40 innovative substances have been brought to China. AstraZeneca’s China headquarters and global R&D center in China are located in Shanghai. In recent years, the company has established regional headquarters in Beijing, Guangzhou, Hangzhou, Chengdu, and Qingdao. AstraZeneca is also working with partners to build a diversified innovation troika including the China Health Innovation Center (CCiC), the International Life Sciences Innovation Park (iCampus), and the Medical Industry Fund AstraZeneca CICC The innovative international health ecosystem promotes the joint rapid development of the regional economy and the health industry in general. Today, China has become AstraZeneca’s second largest market in the world.

Strensiq Hong Kong (40mg) prescribing information, version April 2023 (Chinese) ↑

Scott LJ. Asfotase Alfa: A Review in Pediatric-Onset Hypophosphatasia. Drugs. 2016 February; 76(2):255-62. doi: 10.1007/s40265-015-0535-2. ↑

Kaplan-Meier analysis of survival of patients with perinatal and infantile hypophosphatasia. Patients treated with Asfotase alfa (studies ENB-002-08 / ENB-003-08 and ENB-010-10) had a significantly better survival rate, calculated using the Kaplan-Meier outcome limit estimate, compared to historical controls (study ENB -011-10; ↑

Whyte BP, Greenberg CR, Salman NJ, et al. Enzyme replacement therapy in life-threatening hypophosphatasia. N English J Med. 2012; 366(10):904-913. ↑

Whyte MP, Simmons JH, Moseley S, et al. Asfotase alfa for infants and young children with hypophosphatasia: 7-year results of a single-arm, open-label, phase 2 extension trial. Lancet Diabetes Endocrinol. 2019; 7(2):93-105. ↑

Hofmann CE, Harmatz P, Vockley J, et al. Efficacy and safety of asfotase alfa in infants and young children with hypophosphatasia: a phase 2 open-label study. J Clin Endocrinol Metab. ↑

Whyte BP, Madson KL, Phillips D, Reeves AL, McAlister WH, Yakimoski A, Mack KE, Hamilton K, Kagan K, Fujita KP, Thompson DD, Moseley S, Odrljin T, Rockman-Greenberg C. Asfotase alfa therapy for children with hypophosphatasia. JCI perspective. 2016 Jun 16;1(9):e85971. doi: 10.1172/jci.insight.85971 ↑

Liu Min, Zhao Yun, Gong Chunxiu New progress in research on hypophosphataseemia[J]. ↑

Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013; 10 (suppl. 2): 380-388. ↑

Fraser D. Hypophosphatasia. Am J Med. 1957; 22(5):730-746. ↑