Updates on FDA drug approvals

At the American College of Rheumatology (ACR) Convergence 2022, Amit Golding, MD, PhD, medical officer for the US Food and Drug Administration (FDA), provided updates on recent FDA approvals for drugs and discussed how biosimilars are approved.

Golding outlined the 4 categories of similarity between adult and pediatric conditions. Different conditions show no overlap, dissimilar conditions contain slight overlap with significant differences, similar conditions show significant overlap with slight differences, and same conditions show significant overlap with no clear significant differences.

The first approval discussed by Golding was the use of belimumab IV (BENLYSTA; GlaxoSmithKline plc) for pediatric lupus nephritis in patients aged 5 to 17 years. Approval was based on established efficacy in adults and a successful study in pediatric patients. The pediatric dosage is 10 mg/kg in 2-week intervals for the first 3 doses, followed by 4-week intervals, matching the adult dosage.

Another approval was the use of ustekinumab SC (Stelara; Janssen Pharmaceuticals) for psoriatic arthritis in pediatric patients aged 6 to 17 years. The approval followed efficacy in adults and completed studies in pediatric patients with plaque psoriasis. A dosage based on weight is given 4 weeks after the initial dose, then every following 12 weeks.

Golding also discussed a study for secukinumab (COSENTYX; Novartis) for pediatric psoriatic arthritis and enthesitis-related arthritis. The study evaluated the treatment in pediatric patients aged 2 to under 18 years. In part 2 of the study, participants were randomized to secukinumab or placebo, with a primary endpoint of time to flare. 

Following the study, approval was given to the product. Adverse events were mild and matched the safety profile for secukinumab, and efficacy for reducing flare rate was proven.

In discussing biosimilars, Golding described them as treatments that are highly similar and with, “no clinically meaningful differences from the FDA approved reference product.” They use the same mechanisms for conditions of use, which have been previously approved for the reference product. Biosimilars should meet the standards of safety, purity, and potency.

To evaluate differences in products, Golding recommended analytical study. Golding also stated that a biosimilar should have similar efficacy and safety as the reference product, and that an assessment of immunogenicity should be conducted.

Reference

Golding, A. Pediatric rheumatology FDA update 2022 ACR Convergence November 14, 2022. Presented at: ACR Convergence 2022, Philadelphia, Pennsylvania. November 11 to November 14, 2022.