On Thursday, the U.S. Food and Drug Administration (FDA) approved AstraZeneca Plc’s (NASDAQ:AZN)Koselugo (selumetinib) for adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

NF1 is a rare, progressive, genetic condition caused by a spontaneous or inherited mutation in the NF1 gene.

It is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and, in up to 50% of patients, tumors called plexiform neurofibromas (PN) that can develop along nerve sheaths.

These PN can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder or bowel dysfunction.

The approval was based on positive results from the KOMET Phase 3 trial.

In the primary analysis of the KOMET Phase 3 trial, Koselugo showed a statistically significant and clinically meaningful overall response rate (ORR) of 20% compared to 5% with placebo by cycle 16, with 86% of patients on Koselugo having an observed duration of response (DOR) of at least 6 months.

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After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles.

The safety of Koselugo in the KOMET Phase 3 trial was consistent with its known profile and established use in paediatric patients.

Koselugo was recently approved in the EU, Japan, and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN based on data from the KOMET Phase 3 trial, and additional regulatory reviews are ongoing.

In the U.S., Koselugo granules were recently approved for paediatric patients aged one year and older with NF1 PN.

Price Action: AZN stock is up 0.66% at $89.58 at the last check on Thursday.

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