In ‘landmark’ approval, FDA OKs Ocrevus for kids 10 and older with RRMS
Decision brings safe, high-efficacy treatment option to pediatric patients
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An adult holds the bandaged hand of a child in a hospital bed receiving an infusion, while the youngster clutches a teddy bear with the other hand. (Photo from iStock)
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The FDA has approved Ocrevus for treating relapsing-remitting multiple sclerosis among children 10 and older in the U.S.
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In a clinical trial, the infusion therapy was shown to significantly reduce relapses and new or enlarging lesions in pediatric RRMS patients.
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As the second treatment approved for children and adolescents with RRMS, the therapy offers a new option for patients, according to developer Genentech.
The U.S. Food and Drug Administration (FDA) has approved the infusion therapy Ocrevus (ocrelizumab) for treating children with relapsing-remitting multiple sclerosis (RRMS) who are ages 10 and older and weigh at least 55 pounds (25 kg).
The approval makes Ocrevus the second-ever therapy to win formal FDA approval for this indication, according to its developer Genentech, a member of the Roche Group. Previously, Gilenya (fingolimod) was the only MS therapy approved for pediatric patients in the U.S.
“This approval represents a landmark for children living with MS in the U.S. and their families, which can help close the longstanding gap in high-efficacy treatment options for children aged 10 and older,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Genentech, said in a company press release announcing the FDA’s decision.
“By bringing a decade of efficacy and safety data to this younger population, Ocrevus may reduce relapses and potentially redefine what’s possible for their future,” Garraway said.
MS is a chronic disorder marked by inflammation that damages nerve cells in the brain and spinal cord, leading to symptoms that can include difficulty moving, mental health challenges, and fatigue. The disorder primarily affects adults, but in some rare cases, it can manifest during childhood or adolescence. When MS onset happens prior to age 18, patients are said to have pediatric-onset MS or POMS.
Virtually everyone with pediatric MS initially develops the relapsing-remitting form of the disease, which is marked by relapses or flares in which symptoms suddenly worsen, followed by periods of remission where symptoms ease.
Ocrevus already FDA approved in US for treating adults
Ocrevus works by depleting B-cells, a type of immune cell with a central role in MS-driving inflammation. The therapy is administered by intravenous, or into-the-vein, infusion, with the first two doses administered two weeks apart and subsequent doses given every six months thereafter.
The therapy was already approved in the U.S. for adults with relapsing forms of MS, specifically clinically isolated syndrome, RRMS, and active secondary progressive MS, as well as primary progressive MS.
The new FDA approval for the treatment of pediatric MS was based mainly on data from a Phase 3 clinical trial called OPERETTA 2 (NCT05123703), which enrolled 187 children and adolescents with MS. The participants were randomly assigned to receive either Ocrevus or Gilenya, a daily oral medication, for two years.
The OPERETTA 2 trial met its main goal of showing that Ocrevus was at least as effective as Gilenya at reducing relapse rates. In fact, patients treated with Ocrevus had a 48% lower risk of relapse compared with those receiving the oral medication, the data showed.
Additional results showed Ocrevus significantly reduced the number of patients with new or enlarging MS lesions by 48% and also lowered the number of participants with actively inflamed lesions by 87%.
Growing up with MS, I know the frustration … and the fear. … Having an FDA-approved, high-efficacy treatment option like Ocrevus available for age-appropriate children and adolescents is a game changer.
The safety profile seen with Ocrevus in children with MS was consistent with its known profile in adults. Also, no side effects led to treatment discontinuation in the Ocrevus group, whereas three participants withdrew in the Gilenya group.
The FDA’s approval of Ocrevus for pediatric MS was welcomed by advocates within the community.
“Growing up with MS, I know the frustration of being dismissed and the fear of what comes next,” said Emily Blosberg, who was diagnosed with MS as a teenager. “Having an FDA-approved, high-efficacy treatment option like Ocrevus available for age-appropriate children and adolescents is a game changer.”
After her diagnosis, Blosberg went on to found the POMS-focused nonprofit Mr. Oscar Monkey, whose goal is “Supporting kids and families on their journey with MS.”
According to Blosberg, this approval “means the next generation of patients won’t have to wait for answers — they have an opportunity to take control of their disease early and potentially stop relapses and brain lesions before they have a chance to take a toll.”
Genentech noted that POMS affects approximately 5,000 to 10,000 children and adolescents in the U.S.
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