FDA News

FDA grants orphan drug designation to larotrectinib for solid tumors with NTRK gene fusions

The FDA granted orphan drug designation to larotrectinib for the treatment of solid tumors with neurotrophic tyrosine receptor kinase–fusion proteins, according to the drug’s manufacturer.

Larotrectinib (LOXO-101, Loxo Oncology) is a potent, oral and selective drug for the treatment of patients with cancers that harbor abnormalities that involve tropomyosin receptor kinases (TRKs).

An ongoing phase 1 trial of larotrectinib has demonstrated encouraging preliminary efficacy, according to a Loxo Oncology–issued press release.

Larotrectinib is being evaluated in two other trials.

The global, multicenter phase 2 NAVIGATE trial is designed to evaluate larotrectinib in patients with solid tumors that harbor TRK gene fusions. The phase 1/phase 2 SCOUT trial is designed to evaluate the drug in pediatric patients, including those with advanced cancer, TRK gene fusions and infantile fibrosarcoma.

The FDA previously granted breakthrough therapy designation, rare pediatric disease designation and orphan drug designation to larotrectinib.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

The FDA granted orphan drug designation to larotrectinib for the treatment of solid tumors with neurotrophic tyrosine receptor kinase–fusion proteins, according to the drug’s manufacturer.

Larotrectinib (LOXO-101, Loxo Oncology) is a potent, oral and selective drug for the treatment of patients with cancers that harbor abnormalities that involve tropomyosin receptor kinases (TRKs).

An ongoing phase 1 trial of larotrectinib has demonstrated encouraging preliminary efficacy, according to a Loxo Oncology–issued press release.

Larotrectinib is being evaluated in two other trials.

The global, multicenter phase 2 NAVIGATE trial is designed to evaluate larotrectinib in patients with solid tumors that harbor TRK gene fusions. The phase 1/phase 2 SCOUT trial is designed to evaluate the drug in pediatric patients, including those with advanced cancer, TRK gene fusions and infantile fibrosarcoma.

The FDA previously granted breakthrough therapy designation, rare pediatric disease designation and orphan drug designation to larotrectinib.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.