EU regulators recommend approval of infusion therapy for pediatric MS
Final decision from European Commission expected in the near future
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Regulators in the EU have recommended that the infusion therapy Ocrevus be approved to treat children and teens, ages 10 and older, who have relapsing forms of multiple sclerosis. Photo by iStock)
- European regulators recommended approving Ocrevus for children and teens, ages 10 and older, with relapsing MS.
- A final decision from the European Comission is expected in the near future.
- Clinical trials demonstrated that Ocrevus effectively lowered relapse rates compared to an older oral treatment.
Regulators in the European Union (EU) have recommended that the infusion therapy Ocrevus (ocrelizumab) be approved to treat children and teens, ages 10 and older, who have relapsing forms of multiple sclerosis (MS).
The recommendation comes from the Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency. The CHMP’s opinion will now be reviewed by the European Commission, which has final say over medication approvals in the EU. The Commission isn’t technically required to follow the CHMP’s recommendations, but it almost always does.
A final decision from the commission is expected in the near future, according to a press release from Roche, which markets Ocrevus globally. The company sells the therapy in the U.S. through its subsidiary Genentech.
“The positive CHMP opinion brings us closer to bridging a longstanding gap in Europe, offering young people with MS a high-efficacy therapy backed by a decade of adult experience,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development.
Treatment options for children, teens ‘have lagged behind’
MS is a chronic disorder marked by inflammation that damages healthy nerve cells. In most people with MS, the disease is marked by relapses or flares — attacks where symptoms suddenly worsen due to new inflammation.
The neurodegenerative disease usually develops during adulthood, but in a small number of patients, it manifests in childhood or adolescence. This is known as pediatric-onset MS or POMS. Virtually all young people with POMS experience relapses.
Although more than a dozen therapies are widely approved to treat adults with relapsing MS, most of these treatments have not been formally tested in children with POMS.
“Children and teens living with multiple sclerosis experience more frequent and severe relapses than adults, yet their treatment options have lagged behind,” Garraway said.
Ocrevus is an antibody-based therapy that works by depleting B-cells, a type of immune cell that plays a key role in MS-driving inflammation. The therapy is given by infusion into the bloodstream; the first two infusions are given two weeks apart, and subsequent infusions are given every six months.
In the EU, Ocrevus is currently approved to treat certain adults with relapsing forms of MS or primary progressive MS (PPMS). It’s also approved for adults with relapsing MS or PPMS in the U.S., and earlier this year, the U.S. Food and Drug Administration (FDA) authorized the therapy for pediatric patients with relapsing-remitting MS, ages 10 and older.
Extending Ocrevus, a proven treatment for adults, to younger patients is an important step forward to suppress disease activity early on, with the goal of preserving their physical and cognitive health.
FDA approval of Ocrevus for pediatric MS, as well as the new CHMP opinion for the same indication, were based on data from a Phase 3 clinical trial called OPERETTA 2 (NCT05123703).
The study enrolled nearly 200 people with POMS, who were randomly assigned to take Ocrevus or Gilenya (fingolimod), an oral therapy that was previously approved for pediatric MS. Results showed that relapse rates were lower with Ocrevus than Gilenya over the course of the two-year trial, and safety data for Ocrevus were consistent with its profile in adults.
“Living with a pediatric MS diagnosis means that children and adolescents must navigate a complex condition with unpredictable relapses that require hospital admission and acute therapies, lead to missed days of school and social activities, and face the potential for future disability,” said Brenda Banwell, MD, of Johns Hopkins Children’s Center. “Extending Ocrevus, a proven treatment for adults, to younger patients is an important step forward to suppress disease activity early on, with the goal of preserving their physical and cognitive health.”
For adults, Ocrevus is also available as an under-the-skin injection (sold in the U.S. as Ocrevus Zunovo). That formulation is not approved for children in the U.S., and CHMP’s recent opinion only covers the intravenous formulation.
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