FDA Expands Approval For AstraZeneca's Drug For Pediatric Patients With Rare Type Of Cancer

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:

• Metaplanet Secures $1.45 Billion Capital Raise, Nets $30 Million Investment From KindlyMD Subsidiary Nakamoto

Photo by JHVEPhoto via Shutterstock