Enspryng Recommended for EU Approval for Patients, 12 and Older
A branch of the European Medicines Agency has recommended that Enspryng (satralizumab) be approved to treat patients, 12 and older, with neuromyelitis optica spectrum disorder (NMOSD), who carry antibodies against the aquaporin-4 protein (AQP4-IgG).
The recommendation, made by the agency’s Committee for Medicinal Products for Human Use (CHMP), covers Enspryng’s use alone or in combination with immunosuppressive therapy. Immunosuppressants are commonly used to manage NMOSD symptoms associated with relapses.
If approved by the European Commission — which will now review the application and make a final decision in the near future — the treatment will be the first available to adults and adolescents with AQP4-IgG-positive NMOSD in Europe.
It could also offer a treatment option for currently unserved patient populations, such as people who have experienced only one NMOSD attack, adolescents, and those with more advanced disease.
Furthermore, Enspryng can be self-administered at home by patients or caregivers, via an under-the-skin (subcutaneous) injection every four weeks.
Enspryng, developed by Roche’s subsidiary Chugai Pharmaceutical, is approved in the U.S. for adults with AQP4-IgG-positive NMOSD. Several other countries, such as Canada, Switzerland, Japan, and Taiwan, have approved it for adults and adolescents.
This “positive CHMP opinion is an important step toward bringing Enspryng to people in the EU living with NMOSD who have limited treatment options,” Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, said in a press release.
“If approved, Enspryng would be the first and only treatment that can be self-administered subcutaneously at home following appropriate training,” he said.
NMOSD is an autoimmune disorder characterized by inflammatory damage to the optic nerves and spinal cord. In up to 80% of cases, the disease is caused by elevated levels of AQP4-IgG antibodies, which activate a part of the immune system called the complement pathway, and cause inflammation that damages the central nervous system.
Enspryng is a lab-made, humanized antibody that targets the receptors for the immune signaling protein interleukin-6, which plays a role in inflammation and is emerging as an important factor in NMOSD development.
The treatment was designed using a proprietary recycling antibody technology, which allows for longer circulation time and dosing every four weeks after an initial injection.
The recommendation for EU approval was based on results from two worldwide Phase 3 clinical studies, SAkuraStar (NCT02073279), evaluating Enspryng alone in adults with NMOSD, and SAkuraSky (NCT02028884), testing Enspryng in combination with immunosuppressive therapy in people, 12 and older, with NMOSD.
The SAkuraSky trial is currently in its open-label phase, with completion estimated for March 2022.
In both studies, Enspryng was shown to be safe and well-tolerated, and significantly reduced the risk of relapse compared with a placebo. The benefits were seen when the therapy was used alone and as an add-on to immunosuppressants, and were sustained for 96 weeks (nearly two years).
The most common side effects of Enspryng were headache, joint pain, a decrease in white blood cells, high levels of circulating fats, and injection-related reactions.
“Enspryng has been shown to reduce the risk of relapse significantly, while also offering a favourable safety profile,” Garraway said.