Date: 2015-01-20
Type of
information: Presentation of results at a congress
phase:
Announcement: presentation of results at the Endocrine Society’s 97th Annual Meeting and Expo (ENDO)
Company: Alexion Pharmaceuticals (USA - CT)
Product: asfotase alfa
Action
mechanism:
- enzyme replacement therapy. Asfotase alfa is an investigational, highly innovative, first-in-class enzyme replacement therapy. Asfotase alfa is designed to address the underlying cause of HPP by aiming to restore the genetically defective metabolic process, thereby preventing or reversing the severe and potentially life-threatening complications of life-long dysregulated mineral metabolism.
- In 2013, the FDA granted Breakthrough Therapy designation for asfotase alfa. In 2014, Alexion completed regulatory filings in the U.S., Europe and Japan for asfotase alfa for the treatment of hypophosphatasia.
Disease: hypophosphatasia
Therapeutic
area: Rare diseases - Genetic diseases - Metabolic diseases
Country:
Trial
details:
Latest
news:
- • On January 20, 2015, Alexion Pharmaceuticals announced that researchers will present new functional and quality-of-life data from clinical studies in children with hypophosphatasia (HPP) who were treated with asfotase alfa for at least three years at the Endocrine Society’s 97th Annual Meeting and Expo (ENDO). Researchers will also present data from two patient-reported surveys characterizing the burden of HPP in adults. Abstracts summarizing these studies were published on the ENDO website. The ENDO annual meeting will be held March 5-8, 2015, in San Diego.
- The following abstracts will be presented in a poster session on Friday, March 6, 2015, from 1:00 to 3:00 p.m., Pacific Standard Time (PST):
- Abstract FRI-224: “Improved Activities of Daily Living and Physical Function, with Decreased Pain, in Children with Hypophosphatasia Treated for Three Years with Asfotase Alfa: Results from the Childhood Health Assessment Questionnaire and the Pediatric Outcomes Data Collection Instrument,” Phillips, et al. (https://endo.confex.com/endo/2015endo/webprogram/Paper21021.html)
- Abstract FRI-240: “Burden of Disease in Adult Patients with Hypophosphatasia: Results from Patient-Reported Outcome Surveys,” Weber, et al. (https://endo.confex.com/endo/2015endo/webprogram/Paper20871.html)
- The following abstract will be presented in an oral session on Saturday, March 7, 2015, from 11:30 a.m. to 1:00 p.m., Pacific Standard Time (PST):
- Abstract OR29-4: “Significantly Improved Muscle Strength, Running Speed, and Agility in Children with Hypophosphatasia Treated with Asfotase Alfa,” Phillips, et al. (https://endo.confex.com/endo/2015endo/webprogram/Paper20906.html)
Is
general: Yes
