Amgen, Inc. previously specialized in large therapeutic areas like cardiovascular diseases and inflammation, but added a big focus on medicines for rare diseases through its $27.8bn acquisition of Horizon Therapeutics plc last year. Now that the Horizon products and its research and development programs are in Amgen’s hands, the big biotech is going public with its plans for driving growth in rare diseases now and in the future.
Amgen Lays Out Plans For Rare Disease Portfolio Growth
Ex-US Push, Subcutaneous Dosing For Tepezza
Amgen detailed plans for increasing sales of drugs acquired in its Horizon transaction and outlined growth opportunities in its R&D pipeline during a rare disease showcase.
More from Rare Diseases
• By
The small interference RNA therapeutic can be used by patients regardless of inhibitor status.
• By
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.
• By
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.
• By
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.
More from Scrip
• By
Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.
• By
The four-year-old firm said it plans to advance programs toward the clinic from the funding round, which comes just over a year after signing two major pharma partnerships.
• By
A Phase III trial testing the cortisol modulator showed a benefit on PFS and OS in patients with platinum-resistant ovarian cancer.