Health Canada approves Alexion’s Strensiq to treat patients with hypophosphatasia

Strensiq is the first approved treatment for HPP, an ultra-rare, genetic, metabolic disease in which patients experience devastating, progressive effects on multiple organs of the body, leading to debilitating morbidities or pre-mature mortality.

"Strensiq is critical and pivotal for the treatment of patients with HPP who to date have not had an effective therapy. HPP is a rare and extremely debilitating disease. It has a very high mortality rate in the most severe paediatric cases," said Dr. Cheryl Rockman-Greenberg, Distinguished Professor, Department of Pediatrics and Child Health, University Of Manitoba and Clinician Scientist in the Children’s Hospital Research Institute of Manitoba, and lead Canadian investigator in the HPP trials.

"In clinical studies to date, Strensiq has greatly reduced mortality in affected infants with HPP with 89 per cent overall survival after three years of treatment compared with almost 100 per cent mortality documented in the past. As well, in clinical studies to date, Strensiq has greatly reduced pain associated with HPP and improved physical functioning of patients, including healing of the ‘rickets’ and improvement in mobility such as increased speed of walking, jumping, endurance and balance. It is very exciting to now have an approved therapy for patients who previously only faced early death, or had to live with severe disability and pain."

The approval of Strensiq for patients with paediatric-onset HPP results from a series of medical advances in Canada.

"HPP was first correctly diagnosed in Canada, the asfotase alfa molecule was invented here, and the first patient to ever receive asfotase alfa was treated at the University of Winnipeg. In addition, although HPP is very rare, we recognize that the incidence of the severe form of the disease in the Mennonite population in Manitoba is higher than in other studied populations," said Dr. Philippe Crine, Ph.D. and inventor of asfotase alfa.

"It is very gratifying that this important medical breakthrough can now benefit patients and families suffering with HPP in Canada."

The marketing of Strensiq was authorized under Health Canada’s Notice of Compliance with Conditions (NOC/c) policy. Approvals under the NOC/c policy are granted to products that, among other factors, are intended for the treatment of a life-threatening or severely debilitating illness, have promising evidence of clinical effectiveness and an acceptable safety profile, and respond to a serious unmet medical need in Canada.

Under this NOC/c approval, Alexion will complete and submit additional data from existing trials and one additional study with Strensiq.

"Health Canada’s approval of Strensiq represents a very important milestone in Alexion’s mission to provide transformative therapies for patients with severe and rare diseases," said John Haslam, General Manager of Alexion Pharma Canada.

"However, the approval is not the final step required in ensuring that patients can receive Strensiq. We look forward to the Provinces and private insurers in Canada providing access for the very few patients with HPP as rapidly as possible."

Strensiq recently received marketing approval for patients with HPP in Japan from the Ministry of Health, Labour and Welfare (MHLW).

Alexion has submitted a Biologics License Application for Strensiq with the U.S. Food and Drug Administration (FDA), which was accepted for priority review, and received a positive CHMP opinion recommending marketing authorization by the European Commission for Strensiq for patients with paediatric-onset HPP.