the Food and Drug Administration granted traditional approval to selpercatinib (Retevmo, Eli Lilly and Company) for adult and pediatric patients 2 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy.

Innovent Biologics, Inc...is pleased to see that the National Medical Products Administration (NMPA) of China has approved the New Drug Application (NDA) for selpercatinib (40mg & 80mg capsules) for...adult and pediatric patients 12 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation who require systemic therapy...

On March 11, the Ministry of Food and Drug Safety authorized the use of Lilly’s Retevmo in adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC), adults and pediatric patients 12 years of age or older with advanced or metastatic RET-mutated medullary thyroid cancer requiring systemic therapy...The approval for Retevmo was based on the LIBRETTO-001 trial in RET-mutated advanced or metastatic solid cancer patients.

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation for Retsevmo® (selpercatinib) as monotherapy for several RET-driven advanced lung and thyroid cancers....The treatment was approved for...adults and adolescents 12 years and older with advanced RET mutant medullary thyroid cancer (MTC) who require systemic therapy following prior treatment with cabozantinib and/or vandetanib.

Retsevmo as monotherapy is indicated for the treatment of adults and adolescents 12 years and older with advanced RET-mutant medullary thyroid cancer (MTC) who require systemic therapy following prior treatment with cabozantinib and/or vandetanib.

RETEVMO is a kinase inhibitor indicated for the treatment of...Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy

Selpercatinib is recommended for use within the Cancer Drugs Fund, as an option for treating...advanced RET-mutant medullary thyroid cancer in people 12 years and older who need systemic therapy after cabozantinib or vandetanib…

Medullary Thyroid Cancer: Preferred regimens…Selpercatinib (RET mutation-positive)

Selpercatinib treatment resulted in superior progression-free survival and treatment failure–free survival as compared with cabozantinib or vandetanib in patients with RET-mutant medullary thyroid cancer.

...Identity of RET gene fusions, mutations, and concurrently activated oncogenic pathways in tumor biopsies and cfDNA. Cohorts 1 and 2: enrollment will be restricted to patients with evidence of a RET gene alteration in tumor...MTC syndrome spectrum cancers (e.g., MTC, pheochromocytoma), cancers with neuroendocrine features/differentiation, or poorly differentiated thyroid cancers with other RET alteration/activation may be allowed with prior Sponsor approval...

With longer f/u and additional pts, selpercatinib continues to demonstrate very durable responses in MTC patients with or without prior cab/van therapy, suggesting first line use of selpercatinib appears to be highly effective in treatment of MTC. The safety profile is unchanged despite longer duration on treatment.

Of 77 enrolled patients, 29 had RET-mutant MTC and one had RET fusion-positive TC….In all enrolled MTC patients (n = 29), the ORR by IRC was 58.6% (95% CI, 38.9–76.5). One RET fusion-positive TC patient treated for 23.4 weeks achieved a partial response at week 8 that was ongoing at cutoff....Selpercatinib showed robust antitumor activity and was well tolerated in Chinese patients with advanced RET-altered TC, consistent with global data from LIBRETTO-001.

Medullary thyroid carcinoma (MTC) in the context of multiple endocrine neoplasia type 2 (MEN2) is caused by mutations in the RET proto-oncogene...The fall in serum calcitonin was evident within 2 weeks of the start of selpercatinib, and responses were ongoing at a median follow-up of 13 months (range, 11-22 months).... Selpercatinib has shown excellent therapeutic efficacy with minimal toxicity in children with MEN2 and progressive metastatic RET-mutated MTC.

The ORR on selpercatinib (69%) was markedly higher than for the last prior therapy (10%) received before enrollment. ORR improvements with selpercatinib were observed regardless of prior therapy...Prior to selpercatinib, response with previous multikinase therapy was rare. By contrast, selpercatinib demonstrated robust efficacy regardless of response to or specific prior therapy in pts with RET mutant MTC.

Patients (pts) with RET-mutant medullary thyroid cancer (MTC) and RET-fusion positive thyroid cancer (TC) were enrolled...In this updated analysis, selpercatinib continued to show marked and durable antitumor activity in pts with RET-altered thyroid cancers. Selpercatinib was well tolerated and no new safety concerns were identified.

...selpercatinib demonstrated a significant improvement in PFS and OS versus cabozatinib and versus placebo...The selpercatinib OS HR was 0.25 (95% CI:0.12,0.54; p=0.002) and 0.11 (95% CI:0.05,0.24;p<0.001) versus cabozantinib and placebo...PFS curve data were available for the RET-positive cohort but OS was only provided forthe RET M918T subgroup...

We enrolled patients with RET-mutant medullary thyroid cancer with or without previous vandetanib or cabozantinib treatment, as well as those with previously treated RET fusion-positive thyroid cancer, in a phase 1-2 trial of selpercatinib….In this phase 1-2 trial, selpercatinib showed durable efficacy...

In the first 55 consecutively enrolled patients with RET-mutant medullary thyroid cancer...In this phase 1-2 trial, selpercatinib showed durable efficacy with mainly low-grade toxic effects in patients with medullary thyroid cancer with and without previous vandetanib or cabozantinib treatment.

Selpercatinib use was associated with marked and durable antitumor activity in prior cabozantinib and/or vandetanib-treated patients and in cabozantinib/vandetanib-naïve patients with RET-mutant MTC…

...RET fusion+ NSCLC and papillary thyroid cancer (PTC), RET-mutant medullary thyroid cancer (MTC)...Tumor reduction was achieved in 79% of MTC pts…

Herein, we report a case of a patient with initially unresectable, widely metastatic, RET‐mutated medullary thyroid carcinoma treated on a single‐patient clinical protocol with neoadjuvant selpercatinib/LOXO‐292 followed by definitive surgery. The significant tumor response to neoadjuvant selpercatinib rendered his locoregional disease resectable, and he is now 21 months postinitiation of neoadjuvant treatment with stable distant disease (following partial response) on continued therapy.