Alexion, best known for its flagship product Soliris (eculizumab), received a positive opinion from CHMP of EMA for a new 100mg/ml formulation of Ultomiris (ravulizumab) with a significantly reduced infusion time, approximately 60% less than its original 10mg/ml formulation. Ultomiris is indicated for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) – two ultra-rare disorders. It was developed as a follow-up product of Soliris requiring less frequent dosing, which is eight weeks apart compared to the two-week regimen of the latter. Currently, Ultomiris is the only long-acting C5 inhibitor and the new formulation, which requires administration 8 weeks apart, will dramatically reduce the infusion time for the drug and lesser time spent by the patients at the hospital, thereby reducing the overall burden on the healthcare system especially in hospitals already strained with COVID-19.
This year, Amgen settled its disputes with Alexion and received the license to begin manufacturing and distributing of its eculizumab biosimilar 2025 onwards based on USFDA approval. Three other companies – Samsung Bioepis, Generium and Epirus Biopharmaceuticals are next in line with eculizumab biosimilars. With such competition looming around Soliris, Alexion is strategically positioning Ultomiris as an improved version of Soliris and hoping to switch patients to Ultomiris by 2025 to wade-off some of the competitive pressure from Soliris biosimilars. Ultomiris is also priced slightly lesser than Soliris at USD 6404 per vial vs USD 6543, although the discount from Soliris needs to be substantial enough to trigger patients to switch to Ultomiris. Alexion also has a subcutaneous Ultomiris in the pipeline and anticipates regulatory filing in USA and EU in Q3 2021.
Price-based competition dynamics in rare diseases are complex, given the substantially low patient volumes for the same. Orphan drugs are usually high-priced to recoup the research and development and manufacturing costs from a handful of patients. The competition due to generics or biosimilar leading to price reduction lacks business merit since such indications would not yield a volume advantage. Given the evolving competitive landscape in orphan drugs, the growing participation of big-pharma in rare disease indications and the use of several off-label biosimilars for rare indications, creating a long-term value strategy for orphan drugs is essential for value realization. Instead of relying on price-based competition, companies are now trying to leverage other value-added metrics such as ease of administration, frequency of dosing, etc. to get an edge over other competing products in the market. Alexion’s Ultomiris strategy is an illustrative example of the same, innovating in formulation and administration route to wade-off some of the competition.