- The FDA has approved Strensiq (asfotase alfa), which is the first approved treatment for hypophosphatasia (HPP).
- HPP is a rare, genetic, progressive disease. The main feature of this disease is defective bone mineralization, leading to severe muscle weakness, seizures, loss of mobility, pain, respiratory failure and premature death.
- During the development process, Alexion received Breakthrough Therapy Designation, priority review and orphan drug designation for Strensiq.
This is a solid win for Alexion. First, it took a bet on asfotase alfa when it purchased the drug for $1.8 billion from Enoba. In addition, the company received a much sought-after rare disease priority review voucher—the last time a voucher changed hands it was worth $350 million. And, most important, patients with HPP can benefit from treatment, resulting in substantial bone healing and improvements in growth and mobility in patients.
The shocker was the price, which is going to be $285,000 per year, according to Alexion. The price was perceived of as low by some analysts, who were expecting a price tag in the $400,000 range, and some have speculated that this is in response to the glaring spotlight on pharmace's pricing practices at the moment. Nonetheless, analysts are still bullish on Strensiq and estimate peak sales of $350 million per year.