FDA Conditionally Approves Eli Lilly's Thyroid Cancer Drug For Pediatric Patients With Certain Mutations

Wednesday, the FDA granted accelerated approval to Eli Lilly And Co’s LLY selpercatinib (Retevmo) for pediatric patients two years of age and older with the following:

• Advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, who require systemic therapy.
• Advanced or metastatic thyroid cancer with a RET gene fusion, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
• Locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options.

It is the first FDA approval of targeted therapy for pediatric patients under 12 years of age with RET alterations.

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Selpercatinib was previously granted accelerated approval for thyroid cancer indications in adults and pediatric patients 12 years of age and older.

It was also previously granted accelerated approval for solid tumor indication in adults.

Efficacy in pediatric and young adult patients was evaluated in LIBRETTO-121 study.

The major efficacy outcome measures were confirmed overall response rate (ORR) and duration of response (DOR).

The confirmed ORR was 48%. The median DOR was not reached, with 92% of responders remaining in response at 12 months.

Durable responses were observed in pediatric and young adult patients with RET-mutant MTC (n=14; ORR 43%) and RET fusion-positive thyroid cancer (n=10; ORR 60%).

Price Action: LLY shares are up 0.16% at $809.16 at the last check on Wednesday.

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