Prime Highlights
- BioMarin will acquire Alesta Therapeutics for $275 million upfront, with up to $215 million in additional milestone payments.
- The deal gives BioMarin access to ALE1, an experimental oral treatment that could become the first oral therapy for hypophosphatasia.
Key Facts
- BioMarin Pharmaceutical is a biotechnology company focused on developing medicines for rare and serious diseases.
- Hypophosphatasia is a rare genetic bone disorder, with more than 9,000 diagnosed cases reported in the US.
Background
BioMarin Pharmaceutical will acquire Alesta Therapeutics for $275 million upfront, gaining access to an experimental oral treatment for hypophosphatasia (HPP), a rare genetic bone disorder.
The deal will give BioMarin rights to ALE1, which is currently being tested in early-stage clinical trials. The company said the treatment could become the first oral therapy for HPP. BioMarin will also pay up to $215 million in additional milestone payments if the drug achieves certain development targets.
The acquisition strengthens BioMarin’s rare skeletal disease portfolio, which includes Voxzogo. The company has made several moves in the rare disease sector over the past year, including its planned $4.8 billion acquisition of Amicus Therapeutics.
BioMarin said ALE1 fits its strategy of investing in clinical-stage medicines that can address major unmet medical needs and support long-term growth.
More than 9,000 people in the US have been diagnosed with HPP, although the actual number of people affected may be higher, according to BioMarin.
Alesta’s management said BioMarin’s global reach and experience in developing rare disease medicines will help expand ALE1’s potential reach among patients.
The acquisition is expected to close during the current quarter, subject to customary closing conditions.
The transaction adds another potential treatment to BioMarin’s rare disease pipeline as pharmaceutical companies increasingly invest in medicines for conditions with limited treatment options.



