Drug maker Alexion says that it is preparing for regulatory submissions in the United States, the European Union, and Japan for eculizumab (Soliris) for the treatment of anti-aquaporin-4 auto antibody-positive neuromyelitis optica spectrum disorder.
Drug maker Alexion says that it is preparing for regulatory submissions in the United States, the European Union, and Japan for eculizumab (Soliris) for the treatment of anti-aquaporin-4 auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD).
NMOSD is a rare, complement-mediated demyelinating disorder of the central nervous system that primarily affects the optic nerves and spinal cord, leading to blindness and paralysis. The disorder was once thought to be related to multiple sclerosis, but with the identification of a unique biomarker antibody, NMOSD was understood to be a distinct disease state.1
While typical treatment for NMOSD includes the use of corticosteroids, plasma exchange, and immunosuppressive medications (including anti-CD20 monoclonal antibodies such as rituximab), there is currently no approved treatment for NMOSD, and Alexion hopes that its eculizumab could become the first authorized therapy.
Alexion studied eculizumab in patients with antibody-positive NMOSD in the phase 3 PREVENT trial, a multinational, double-blind, parallel-group, time-to-event study in 143 patients. The patients were randomized 2 to 1 to receive either eculizumab or placebo, and the primary endpoint was the time to the first on-trial relapse.
The study met its primary endpoint, having demonstrated that treatment with the study drug reduced the risk of relapse by 94.2% versus placebo (P <.0001). At 48 weeks of treatment, 97.9% of patients receiving eculizumab were free of relapse versus 63.2% of patients receiving the placebo.
Eculizumab was generally well tolerated, and its safety profile was consistent with that seen in previous clinical studies and real-world use in other indications. No cases of meningococcal infection—an adverse event of particular concern with eculizumab—were observed.
“These results far exceeded our expectations,” said John Orloff, MD, executive vice president and head of research and development at Alexion, in a statement. “The remarkable reduction in relapse risk demonstrates the unique ability of Soliris to inhibit complement, and suggests a promising new treatment for NMOSD.” Orloff added that the company is “moving quickly” to file for approval of the new indication.
Eculizumab is already approved to treat 3 other rare diseases: paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome, and generalized myasthenia gravis. Given the high cost of the drug, adding a fourth indication for the drug could bring in an additional $500 million to $700 million to Alexion, say some analysts. At a list price of approximately $500,000 per patient per year, eculizumab is one of the most expensive therapies in the world.
Yet Alexion’s profitability in this area could be challenged in the near future; the molecule is targeted by at least 1 biosimilar developer; Amgen is currently engaged in a phase 3 study in Spain that evaluates the efficacy and safety of its proposed biosimilar, ABP 959, versus the reference eculizumab in patients with PNH. However, Alexion is hoping to stave off biosimilar competition with a set of 3 patents, awarded in August 2017, that will extend protection for the brand-name Soliris until 2027.
Reference
1. Kessler RA, Mealy MA, Levy M. Treatment of neuromyelitis optica spectrum disorder: acute, preventative, and symptomatic. Curr Treat Options Neurol. 2016;18(1): 2. doi: 10.1007/s11940-015-0387-9.
The Role of Coverage Strategies in Biosimilar Market Impact and Cost Savings
September 4th 2024A recent study highlights that although biosimilars have led to significant price reductions, originator products with sole preferred coverage strategies have maintained market share, suggesting that increased biosimilar uptake alone may not fully leverage the market's competitive and cost-saving potential.
Biosimilars in America: Overcoming Barriers and Maximizing Impact
July 21st 2024Join us as we explore the complexities of the US biosimilars market, discussing legislative influences, payer and provider adoption factors, and strategies to overcome industry challenges with expert insights from Kyle Noonan, PharmD, MS, value & access strategy manager at Cencora.
Exploring the Biosimilar Horizon: Julie Reed's Predictions for 2024
February 18th 2024On this episode of Not So Different, Julie Reed, executive director of the Biosimilars Forum, returns to discuss her predictions for the biosimilar industry for 2024 and beyond as well as the impact that the Forum's 4 new members will have on the organization's mission.
Pure-Play Biosimilar Company Reports 10-Fold Revenue Increase for First Half of 2024
August 22nd 2024Alvotech reported $236 million in revenue for the first 6 months of 2024, a 10-fold increase from the same period the year prior. The new figures come after the company received its first 2 FDA approvals in 2024.