AstraZeneca Pharma India bags DCGI nod to import and market Selumetinib capsules

in ≥20% of patients included disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction. Patients received selumetinib 25 mg/m2 orally twice a day until disease progression or unacceptable toxicity.

The primary efficacy outcome measure was overall response rate (ORR) as assessed by NCI and defined as the percentage of patients who experienced ≥20% reduction in tumor volume on Magnetic Resonance Imaging (MRI) confirmed on a subsequent MRI within 3-6 months. The ORR was 66% (n=33; 95% CI: 51,79). 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% (95% CI: 30,59).

The primary safety data were from 74 pediatric patients with NF1 and PN who received selumetinib during SPRINT. The most common adverse reactions (≥40% of patients) were vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain, fever, acne, stomatitis, headache, paronychia, and pruritus.

Selumetinib can also cause cardiomyopathy, ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment and impaired vision, and increased creatinine phosphokinase. Selumetinib should be withheld, dosage reduced, or permanently discontinued based on the severity of adverse reactions.

The recommended selumetinib dose is 25 mg/m2 orally twice a day on an empty stomach until disease progression or unacceptable toxicity.