On Wednesday, the U.S. Food and Drug Administration (FDA) approved AstraZeneca Plc’s (NASDAQ:AZN) Koselugo (selumetinib) granules and capsules for pediatric patients 1 year of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN), benign, nerve sheath tumors.
Neurofibromatosis is a group of genetic conditions causing non-cancerous tumors to grow in the nervous system, skin, and bone.
In 2020, the FDA approved selumetinib capsules for pediatric patients 2 years of age and older with NF1 who have symptomatic, inoperable PN.
Wednesday’s approval was based on adequate bridging between the oral granule and approved capsule formulations in a relative bioavailability (BA) study in healthy adults (Study 89) and exposure matching between the pediatric patient populations in the SPRINT Phase 2 Stratum I study (capsule formulation, ≥ 2 years of age) and the SPRINKLE study (oral granule formulation, ≥ 1 year of age).
Also Read: AstraZeneca Lung Cancer Combo Extends Patient Survival By Years In Key Study
Similar exposure between the formulations supports extrapolation of efficacy from pediatric patients ≥ 2 years of age to ≥ 1 year of age.
Based on body surface area, the recommended selumetinib dose is 25 mg/m2 orally twice daily, until disease progression or unacceptable toxicity.
The SPRINT trial investigated the efficacy of selumetinib in pediatric patients with NF1 and a measurable target PN that could not be surgically removed without risk of substantial morbidity.
The primary efficacy outcome measure was the overall response rate (ORR), which was 66%. All patients had a partial response, and 82% of responders had sustained responses lasting at least 12 months.
An independent central review of ORR was performed using the same response criteria and demonstrated an ORR of 44%.
Price Action: AZN stock is down 0.33% to $83.96 at the last check Wednesday.
Read Next:
Photo by JHVEPhoto via Shutterstock
