Rare Disease

In a research letter appearing this week in JAMA Internal Medicine, authors from Oregon Health and Science University raised the question of whether the “large and increasing revenue from eculizumab” could be tied to off-label use for non-approved indications.
As innovator biologics that have achieved blockbuster sales status reach or near the end of their exclusivities, biosimilar competition is close behind. This week, stakeholders got a closer look at how innovator drug companies are positioning themselves to maintain their sales in the face of such competition.
Genentech has announced that a phase 3 study of its brand-name rituximab, Rituxan, met its primary end point and demonstrated that treatment with rituximab is superior to treatment with mycophenolate mofetil in patients with moderate to severe pemphigus vulgaris (PV), a rate autoimmune disease that affects the skin and mucous membranes.  
While eculizumab is being targeted by multiple biosimilar developers who are in phase 3 clinical trials with their competitive products, spending on eculizumab is reaching concerning levels; as such, it is increasingly important to clarify eculizumab’s place in the treatment paradigm for myasthenia gravis (MG).
A small, yet rapidly growing segment of commercially insured US patients have drug costs of more than $250,000 each year, and this group could account for 15% of all drug expenditures in the next 5 years.
This week, Russian drug maker Generium announced that it has received approval for—and has launched—its biosimilar dornase alfa in the Russian marketplace. Generium’s product is the first biosimilar dornase alfa biosimilar to be approved in any market worldwide.
During this week’s meeting of the European Committee for Treatment and Research in Multiple Sclerosis, held September 11-13 in Stockholm, Sweden, researchers are presenting new data for eculizumab in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disorder that typically affects the optic nerves and spinal cord and that can cause significant, irreversible disability.
Last week, Alexion disclosed in a filing to the US Securities and Exchange Commission that the European Patent Office did not grant Alexion its request for 2 patents on its brand-name eculizumab product, Soliris, a C5 complement inhibitor that treats rare and ultrarare diseases.
Samsung Bioepis has begun recruiting patients with paroxysmal nocturnal hemoglobinuria (PNH) in India for a phase 3 clinical study of its proposed eculizumab biosimilar, SB12, referencing Soliris.
Drug maker Alexion, developer of the rare disease drug eculizumab (Soliris), announced Friday that the FDA has accepted for priority review its long-acting C5 complement inhibitor, ravulizumab (Ultomiris), which offers less frequent administration than eculizumab. 

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