Gail Rodich, MD: Rituximab Approval and Its Impact

For clinicians treating pediatric patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA), the US Food and Drug Administration’s approval of rituximab (Rituxan) was a monumental step.

Rituximab’s approval, which requires it to be used in combination with glucocorticoids, in patients 2 and older marks the first treatment for GPA and MPA to receive approval for pediatric.

The benefit of rituximab in both GPA and MPA was observed in an international, multicenter, open-label, single-arm, clinical trial involving 25 patients between the ages of 6 and 17 years old with an active form of the disease. At 6 months, 14 patients had reached remission with rituximab plus glucocorticoid treatment and at 18 months all 25 had achieved remissions.

The Genentech therapy was previously approved to treat adult patients with GPA and MPA in 2011, and has been approved for the treatment of 9 conditions—including rheumatoid arthritis in 2006.

For more on what this approval means for clinicians and patients, MD Magazine® sat down with Gail Rodich, MD, a pediatrician and principal medical science liaison of immunology at Genentech, between sessions at the Clinical Congress of Rheumatology (CCR) West 2019 annual meeting in San Diego, CA.

MD Mag: What does the new approval for rituximab mean for clinicians and patients?

Rodich: So, granulomatosis with polyangiitis (GPA) and micro polyangiitis—also GPA and MPA— are 2 potentially life-threatening diseases that affect blood vessels. So, these primarily effect small blood vessels, so this involves kidneys, lungs, sinuses and a variety of other organs. It can effect patients in a variety of ways.

These are rare diseases. So the estimated prevalence in the US is about 3 cases per 100,000 people and it's even more rare in the pediatric patient population. Until yesterday there were no FDA approved medicines to treat pediatric patients with GPA or MPA.

So, the approval of Rituxin with glucocorticoids is very important in providing them a treatment option. This approval is really a result of Genentech's ongoing commitment and working with the FDA to develop medicines for pediatric patients with rare diseases where there is unmet need.