Two years after AstraZeneca PLC completed the acquisition of the rare disease specialist Alexion Pharmaceuticals Inc., the companies have started executing on a combined growth strategy, with Alexion left to lead the charge in rare diseases.
Alexion On Its AstraZeneca Integration And Growth Plan
An Interview With Senior VP-US Commercial Operations Scott Weintraub
Two years after being acquired by AstraZeneca, the company remains relatively autonomous in rare disease, but with “bridges” to its big pharma parent.

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RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
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The highlights of recent comments and insights from industry executives on the key issues covered in Scrip.
RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.
Private Company Edition: The latest group of drug developers to announce venture capital financings is remarkable for its geographic diversity, from Character Biosciences’ $93m series B round in the US to Augustine’s $85m series B in Belgium to a $29.2m series C for Aculys in Japan.