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Real-World Data and Real-World Evidence
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Asfotase alfa (Strensiq), was approved in 2015 by FDA, for the treatment of patients with perinatal/infantile- and juvenile-onset Hypophosphatasia (HPP). HPP is a genetic, chronic, progressive, and life-threatening rare metabolic bone disease, characterized by low Alkaline Phosphatase (ALP) activity and defective bone mineralization that could lead to destruction and deformity of bones and other skeletal abnormalities. Asfotase alfa is an enzyme-replacement therapy used to reverse the skeletal mineralization defects in HPP.
External Control Using RWE and Historical Data in Clinical Development
Published in Harry Yang, Binbing Yu, Real-World Evidence in Drug Development and Evaluation, 2021
Qing Li, Guang Chen, Jianchang Lin, Andy Chi, Simon Davies
The efficacy and safety evidence contained within the Strensiq® NDA submission was not based on a traditional randomized, controlled phase 3 trial. Instead, data came from four clinical trials comprised of patients with perinatal-, infantile-, and juvenile-onset HPP. Table 4.1 summarizes the relevant trial information. Table 4.1 shows that the ENB-002-08/ENB-003-08 and ENB-010-10 studies were single-arm studies. The objectives of these studies were to determine the efficacy (along with safety, long-term tolerability, and pharmacokinetics [PK]) of asfotase alfa in treating the skeletal manifestations of patients with perinatal/infantile-onset HPP. Of note, there were no concurrent control arms in these studies; they were single-arm studies due to the lack of standard of care for patients with HPP.
Biocatalytic Nanoreactors for Medical Purposes
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2019
Oscar González-Davis, Chauhan Kanchan, Rafael Vazquez-Duhalt
Hypophosphatasia is osteomalacia caused by mutations in the gene that encodes the alkaline phosphatase (ALP) that affects the development of bones and teeth (Whyte, 2010; Mornet, 2018). The ALP deficiency disrupts a process called mineralization, in which minerals such as calcium and phosphorus are deposited in developing bones and teeth. The ERT is now available for hypophosphatasia. Asfotase alfa is a recombinant glycoprotein that contains the catalytic domain (the active site) of tissue-nonspecific alkaline phosphatase. Asfotase alfa therapy improved skeletal, respiratory, and physical symptoms with only a few severe side effects in patients with hypophosphatasia, supporting the safety and efficacy of ERT (Kitaoka et al., 2017; Whyte, 2017).
Prenatal diagnosis facilitated prompt enzyme replacement therapy for prenatal benign hypophosphatasia
Published in Journal of Obstetrics and Gynaecology, 2020
Yuka Ishijima, Takashi Iizuka, Kyosuke Kagami, Sakiko Masumoto, Kyohei Nakade, Yusuke Mitani, Yo Niida, Atsushi Watanabe, Rena Yamazaki, Masanori Ono, Hiroshi Fujiwara
Enzyme replacement therapy (ERT) consisting of asfotase alfa, a recombinant bone-targeted alkaline phosphatase for HPP became available in 2015 (Millan and Plotkin 2012; Whyte et al. 2016). We herein present a case of prenatal HPP diagnosed prenatally for which ERT was initiated immediately after birth.