Date: 2015-09-16
Type of
information: Presentation of results at a congress
phase:
Announcement:
Company: Alexion Pharmaceuticals (USA - CT)
Product: Strensiq™ (asfotase alfa)
Action
mechanism:
- protein/enzyme replacement therapy (ERT). Asfotase alfa is an investigational, highly innovative, first-in-class targeted alkaline phosphatase enzyme replacement therapy. Asfotase alfa is designed to address the underlying cause of hypophosphatasia by normalizing the genetically defective metabolic process, and preventing or reversing the severe and potentially life-threatening complications of life-long dysregulated mineral metabolism.
- Strensiq™ is approved in the European Union , Japan , and Canada as a treatment for patients with HPP. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Strensiq and accepted Alexion\'s Biologics License Application (BLA) for Priority Review.
Disease: hypophosphatasia
Therapeutic
area: Rare diseases - Genetic diseases - Metabolic diseases
Country:
Trial
details:
Latest
news:
- • On September 16, 2015, Alexion Pharmaceuticals announced that researchers will present new data on the long-term efficacy and tolerability of Strensiq™ (asfotase alfa) in children with hypophosphatasia (HPP) who were treated for up to five years, as well as new data comparing the functional mobility of children with HPP treated with Strensiq compared to untreated historical control patients. Researchers will also present new data on the dosing for Strensiq in patients with HPP and the use of the Radiographic Global Impression of Change (RGI-C) scale for assessing skeletal manifestations of HPP in infants and children, along with a case literature review of HPP manifestations in adults with pediatric-onset HPP. The data will be presented at the 2015 American Society for Bone and Mineral Research (ASBMR) Annual Meeting being held October 9-12, 2015 , in Seattle.
- The following abstract will be presented in an oral session on Saturday, October 10, 2015 , from 5:00 to 5:15 p.m., Pacific Daylight Time (PDT):
Abstract 1071: \"Asfotase Alfa: Sustained Efficacy and Tolerability in Children with Hypophosphatasia Treated for 5 Years,\" Rockman-Greenberg, et al.
The following abstract will be presented in a poster session on Saturday, October 10, 2015 , from 12:30 to 2:30 p.m., Pacific Daylight Time (PDT):
Abstract SA0376: \"A Longitudinal, Prospective, Long-Term Registry of Patients with Hypophosphatasia,\" Seefried, et al.
The following abstracts will be presented in a poster session on Sunday, October 11, 2015 , from 12:30 to 2:30 p.m., Pacific Daylight Time (PDT):
Abstract SU0380: \"Exposure-Response Modeling and Simulation to Support Evaluation of Efficacious and Safe Exposure and Dose Range for Asfotase Alfa in Patients with Hypophosphatasia,\" Pradhan, et al.
Abstract SU0381: \"Manifestations of Hypophosphatasia in Adults with Pediatric Onset of Symptoms: A Review of the Case Literature,\" Sawyer, et al.
The following abstracts will be presented in a poster session on Monday, October 12, 2015 , from 12:30 to 2:30 p.m., Pacific Daylight Time (PDT):
Abstract MO0382: \"Improved Functional Mobility with Asfotase Alfa Treatment in Childhood Hypophosphatasia,\" Madson, et al.
Abstract MO0059: \"Validation of a Novel Scoring System, the Radiographic Global Impression of Change (RGI-C) Scale, for Assessing Skeletal Manifestations of Hypophosphatasia in Infants and Children,\" Whyte, et al.
Is
general: Yes
