The following is a message from the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur:
On September 21, 2022, the Food and Drug Administration granted regular approval to selpercatinib (Retevmo, Eli Lilly and Company) for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a rearranged during transfection (RET) gene fusion, as detected by an FDA-approved test.
Today, FDA also approved the Oncomine Dx Target (ODxT) Test (Thermo Fisher Scientific) as a companion diagnostic for selpercatinib.
Selpercatinib was previously granted accelerated approval for the NSCLC indication on May 8, 2020, based on initial overall response rate (ORR) and duration of response (DOR) in 144 patients enrolled in the LIBRETTO-001 trial (NCT03157128), a multicenter, open-label, multi-cohort trial. The conversion to regular approval was based on data from an additional 172 patients and 18 months of additional follow-up to assess durability of response.
Efficacy was demonstrated in total of 316 patients with locally advanced or metastatic RET fusion-positive NSCLC. Patients received selpercatinib until disease progression or unacceptable toxicity.
The primary efficacy measures were ORR and DOR as determined by a Blinded Independent Review Committee (BIRC). Among 69 treatment-naïve patients, ORR was 84% (95% CI: 73, 92) with a DOR of 20.2 months (95% CI: 13, not estimable). Among 247 patients previously treated with platinum-based chemotherapy, ORR was 61% (95% CI: 55, 67) with a DOR of 28.6 months (95% CI: 20, not estimable).
The median age of patients was 61 years (range 23 to 92). Selected demographics were as follows: 58% female; 49% White, 41% Asian and 5% Black; 97% had ECOG performance status 0 or 1; 97% had metastatic disease. Previously treated patients received a median of two prior systemic therapies (range 1 to 15); 58% had received prior anti PD 1/PD-L1 therapy.
The most common adverse reactions (≥25%) in patients were edema, diarrhea, fatigue, dry mouth, hypertension, abdominal pain, constipation, rash, nausea, and headache.
The recommended selpercatinib dose based on body weight is:
- Less than 50 kg: 120 mg orally twice daily
- 50 kg or greater: 160 mg orally twice daily
View full prescribing information for Retevmo.
This application was granted breakthrough designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. The application also was granted orphan drug designation.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.
For information on the COVID-19 pandemic, see the following resources:
- FDA: Coronavirus Disease 2019 (COVID-19)
- NCI: Coronavirus: What People With Cancer Should Know
- CDC: Coronavirus (COVID-19)
Follow the Oncology Center of Excellence on Twitter @FDAOncology.
Please visit the FDA Hematology/Oncology (Cancer) Approvals & Safety Notifications webpage for a list of current and past approvals.
