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  6. FDA D.I.S.C.O. Burst Edition: FDA approval of Retevmo (selpercatinib) for advanced or metastatic RET fusion-positive thyroid cancer
  1. Resources for Information | Approved Drugs

FDA D.I.S.C.O. Burst Edition: FDA approval of Retevmo (selpercatinib) for advanced or metastatic RET fusion-positive thyroid cancer

Podcast

Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer drug approval.

On May 29, 2024, the FDA granted accelerated approval to selpercatinib (brand name Retevmo) for pediatric patients two years of age and older with the following:

  • advanced or metastatic medullary thyroid cancer with a RET mutation, as detected by an FDA-approved test, who require systemic therapy
  • advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) and
  • locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options.

This is the first FDA approval of a targeted therapy for pediatric patients less than 12 years of age with RET alterations. Selpercatinib was previously granted accelerated approval for the thyroid cancer indications in adults and pediatric patients 12 years of age and older. It also was previously granted accelerated approval for the solid tumor indication in adults.

Efficacy in pediatric and young adult patients was evaluated in LIBRETTO-121, an international, single-arm, multi-cohort trial. Patients received selpercatinib, 92 mg/m2 orally twice daily, until disease progression, unacceptable toxicity, or other reason for treatment discontinuation. The primary efficacy population included 25 patients ages 2 to 20 with locally advanced or metastatic RET-activated solid tumors non-responsive to available therapies or with no standard systemic curative therapy available.

The major efficacy outcome measures were confirmed overall response rate and duration of response. The confirmed overall response rate, as determined by blinded independent review committee was 48%. The median duration of response 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 medullary thyroid cancer and RET fusion-positive thyroid cancer.

The most common adverse reactions reported in more than 25% of patients were musculoskeletal pain, diarrhea, headache, nausea, vomiting, coronavirus infection, abdominal pain, fatigue, pyrexia, and hemorrhage. The most common Grade 3 or 4 laboratory abnormalities reported in more than 5% were decreased calcium, decreased hemoglobin, and decreased neutrophils.

LIBRETTO-121 was conducted as part of a pediatric Written Request under the Best Pharmaceuticals for Children Act.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatfda.

Health care professionals should report serious adverse events to www.fda.gov/medwatch.

Follow the Division of Drug Information on X (formally Twitter) @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov. Thanks for tuning in to the D.I.S.C.O. Burst Edition.

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