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news.Alexion Pharmaceuticals has received approval from the Japan’s Ministry of Health, Labour and Welfare (MHLW) for its new drug application (NDA) of Strensiq to treat patients with hypophosphatasia (HPP), a life-threatening, ultra-rare metabolic disorder.
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Strensiq (asfotase alfa), a bone-targeted enzyme replacement therapy, is the first such treatment to be approved in Japan for HPP patients.
The company intends to start commercial treatment with Strensiq in Japan in the third quarter of this year.
Alexion chief executive officer David Hallal said: "The rapid approval of the Strensiq NDA in Japan underscores the devastating nature of HPP and the life-transforming impact that Strensiq can provide to Japanese patients living with HPP.
"We are delighted that this regulatory approval in Japan marks the first treatment option for patients with HPP, and we look forward to urgently working with the healthcare authorities to make Strensiq available to Japanese patients who can benefit from this therapy."
The approval of Strensiq was based on data from three pivotal prospective clinical studies and their extensions, a retrospective natural history study in infants, and one investigator-sponsored study in Japan.
The trials involved 71 patients, including five Japanese patients, with infantile and juvenile-onset HPP aged one day to 65 years.
The results showed that patients with infantile-onset HPP (ages =5 years at enrollment) treated with Strensiq demonstrated rapid and sustained improvements in bone mineralization.