Alexion is focusing on medical education ahead of an anticipated approval of asfotase alfa for patients with hypophosphatasia, or HPP.
These are taking the form of diagnostic education programs to help physicians think about screening and testing patients for the ultra-orphan disease.
“We’re just creating our medical teams around the world to help physicians understand what HPP is,” said Leonard Bell, CEO, during Alexion’s fourth-quarter 2013 earnings call.
The drug has received the FDA’s Breakthrough Therapy designation, and Alexion said it’s on track to file for an initial marketing authorization in HPP in the US and Europe by mid-2014, as well as in Japan by year’s end.
With the launch expected by late this year, med ed has focused on HPP as a multi-organ disease, not just bone, and in all ages, not just pediatric, according to a recent investor note from Jeferries analyst Eun Yang.
A “basic element” of the diagnostic campaign will be to help shape physicians’ clinical presentation of patients, said David Hallal, Alexion chief commercial officer, who was also on the call.
Hallal added that the biotech is initially focusing its effort on those patients with perinatal, infantile and juvenile onset—those who “very much needed a transformative treatment”—but that market research shows that many adults living with HPP have onset earlier than age 18, adding, “We’re going to continue to work with all segments of the population and develop our launch strategies appropriately.”
As with any ultra-rare disease, Hallal continued, “there’s typically a misunderstanding as to which patients have the disease.
“As we continue to establish our metabolic therapeutic area for our anticipated [asfotase alfa] launch near late 2014, we have now added our initial field-based medical teams in the US and Western Europe,” he said. “At launch, we will be prepared to serve all patients with pediatric onset HPP, including adults.”
From the March 01, 2014 Issue of MM+M - Medical Marketing and Media
