RegulationBreakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Conditional marketing approval (China), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (China)

Structure/Sequence

Molecular FormulaC29H31N7O3

InChIKeyXIIOFHFUYBLOLW-UHFFFAOYSA-N

CAS Registry2152628-33-4

Clinical Trials associated with Selpercatinib

CTIS2025-522431-34-00

/ Not yet recruitingPhase 2

TAILOR-PANC: Molecularly Tailored Therapy versus Standard Care in Advanced Pancreatic Cancer (DPCG-02)

Start Date01 Oct 2026

Sponsor / Collaborator

Region Hovedstadens Apotek

NCT07146893

/ Not yet recruitingEarly Phase 1IIT

Feasibility Study for Repurposing RET Inhibitors for Treating Cancer Cachexia

The purpose of this study is to assess the effects of selpercatinib on cachexia and anorexia in patients diagnosed with NSCLC, colorectal cancer, or pancreatic cancer.

Start Date26 Feb 2026

Sponsor / Collaborator

University of Oklahoma

[+1]

NCT07010393

/ Not yet recruitingPhase 4IIT

A Multicenter Prospective-Retrospective Real-World Study Evaluating Conversion-to-Surgery and Survival After Genotype-Matched Neoadjuvant Systemic Therapy in Locally Advanced Thyroid Carcinoma

This multicenter registry tests whether genomically matched neoadjuvant therapy (1-4 cycles tailored to BRAF V600E, RET fusion/mutation, isolated TERT mutation, triple-negative BRAF/RET/TERT, or ICI ± TKI) can render locally advanced, initially unresectable-or high-morbidity-thyroid cancers operable. The primary endpoint is conversion-to-surgery; key secondaries are R0/1 margin rate and 12-month event-free survival, with propensity-score weighting correcting cohort imbalances. Findings aim to define a precision-guided neoadjuvant standard for down-staging advanced thyroid tumors.

Start Date01 Jul 2025

Sponsor / Collaborator

Fujian Medical University

100 Clinical Results associated with Selpercatinib

100 Translational Medicine associated with Selpercatinib

100 Patents (Medical) associated with Selpercatinib

497

Literatures (Medical) associated with Selpercatinib

01 Oct 2026·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Identification of RET PROTAC with excellent degradation efficiency against medullary thyroid carcinoma

Article

Author: Jeon, Eunhye ; Kim, Chanil ; Song, Jaewon ; Lim, Sugene ; Park, Seung Min ; Park, Jong Bae ; Sim, Taebo

Mutations and rearrangements of Rearranged during Transfection kinase (RET) are highly implicated with thyroid cancer. This study aimed to explore the therapeutic potential of a targeted protein degradation (TPD) strategy against RET-associated medullary thyroid carcinoma (MTC). The investigation of the structure-activity relationship (SAR) led to the identification of a novel RET-CRBN degrader, JW15 which possesses excellent degradation capability (DC₅₀ = 0.3 nM) and potent anti-proliferative activity (GI₅₀ = 1.2 nM) on TT cells (MTC). Mechanistic studies revealed that JW15 degrades RET kinase via the ubiquitin-proteasome system (UPS) and effectively recruits cereblon (CRBN) E3 ligase in target engagement assays. Notably, JW15 exhibited superior suppression of RET phosphorylation and downstream signaling compared with selpercatinib. In addition, JW15 demonstrated greater efficacy than selpercatinib in inducing apoptosis and suppressing colony-forming capacity on TT cells, particularly at a concentration of 1 nM. JW15 administered intravenously at 20 mg/kg twice weekly demonstrated significant antitumor efficacy, showing 70% tumor growth inhibition in RET Ba/F3 xenograft models over 13 days. Taken together, our study provides fundamental guidelines for structure-guided design and optimization for the development of RET degraders, offering a promising strategy for targeting RET-driven cancers.

01 Jun 2026·CANCER LETTERS

Modeling acquired TKI resistance and effective combination therapeutic strategies in murine RET+ lung adenocarcinoma

Article

Author: Heasley, Lynn E ; Navarro, Andre C ; Le, Anh T ; Haines, Samantha ; Hinz, Trista K ; Avila, Jasalyn T ; Nemenoff, Raphael A ; Doan, Tristan ; Skhisov, Daniel ; Ast, Abby ; Jaramillo, Sophia ; Patil, Tejas

Oncogenic RET gene rearrangements drive a subset of lung adenocarcinomas (LUAD) and the tyrosine kinase inhibitors (TKIs) selpercatinib and pralsetinib are approved therapeutics. However, acquired resistance remains a hurdle to durable management. Two RET+ lung cancer cell lines (TR.1, TR.2) were established from a Trim24-Ret mouse model and lung tumors resulting from their orthotopic transplantation initially responded to selpercatinib followed by prompt progression within ∼3 weeks of initiating TKI treatment. Cell lines derived from the selpercatinib-resistant TR.1 and TR.2 tumors exhibited in vitro sensitivity to MET and ERBB-targeted TKIs, indicating acquired bypass signaling through these receptor tyrosine kinases (RTKs). The TKI-resistant cell lines showed no evidence for MET gene amplification, but exhibited transcriptional induction of genes that function within MET and ERBB2:ERBB4 interaction networks including ligands (HGF, NRG1), adaptors (GAB1) and co-receptors (NRP1). Exogenous HGF, but not NRG1 reversed in vitro growth inhibition by selpercatinib in TR.1 and TR.2 cells. Mice bearing orthotopic TR.1 or TR.2 lung tumors progressing on selpercatinib underwent significant re-shrinkage upon co-treatment with the MET inhibitor, crizotinib, although similar to the clinical experience, progression again occurred. By contrast, upfront treatment with selpercatinib and crizotinib in orthotopic tumors yielded complete elimination of 7 of 9 TR.1 tumors and both deepened and prolonged the duration of response in TR.2 tumors. The findings provide new insight into mechanisms of acquired resistance through bypass signaling and highlight the therapeutic benefit of simultaneous upfront blockade of driver oncogenes and dominant resistance mechanisms in LUAD.

01 Jun 2026·EXPERIMENTAL CELL RESEARCH

RET inhibition by selpercatinib suppresses TGF-β1–induced epithelial-mesenchymal transition in retinal pigment epithelium

Article

Author: Hu, Chun ; Chen, Meilan ; Xu, Lu ; Zhao, Rui ; Chen, Xiaohui

PURPOSE:

The transforming growth factor-β (TGF-β) pathway is a master regulator of epithelial-mesenchymal transition (EMT) in retinal pigment epithelial (RPE) cells, a key process in fibrotic ocular diseases. However, key modulators fine-tuning this pathway in RPE are incompletely characterized. This study investigates the role of the receptor tyrosine kinase RET in TGF-β1-induced RPE-EMT.

METHODS:

The role of RET was assessed in vitro using the clinically approved RET inhibitor, Selpercatinib, in RPE cells stimulated with TGF-β1. Outcomes included EMT markers, migration, proliferation, and transcriptional reprogramming. Key downstream signaling effectors Smad3 and ERK were analyzed. In vivo validation was performed in mouse models of TGF-β1-induced EMT and sodium iodate (NaIO₃)-induced RPE injury.

RESULTS:

Pharmacological inhibition of RET with Selpercatinib effectively suppressed TGF-β1-induced EMT, migration, proliferation, and associated transcriptional reprogramming in RPE cells. Mechanistically, RET kinase activity was required for the TGF-β1-induced activation of Smad3 and ERK. In mouse models, Selpercatinib treatment attenuated RPE-EMT and preserved retinal structure.

CONCLUSIONS:

Our findings establish RET as a critical modulator in the TGF-β signaling pathway in RPE cells and highlight the therapeutic potential of repurposing RET inhibitors for the treatment of fibrotic retinal diseases.

239

News (Medical) associated with Selpercatinib

14 Jul 2026

·www.prnewswire.com

Innovent Biologics and Spero Therapeutics Announce Exclusive License for IBI355 (SP001), a Phase 2-Ready Third-Generation Anti-CD40L Antibody

Under the terms of the agreement, Spero will receive exclusive global rights, excluding Greater China, to develop, research, manufacture, and commercialize IBI355 (Spero R&D code: SP001) Innovent will receive an upfront payment, and is eligible to receive development, regulatory and commercial milestone payments, totaling approximately US$1.1 billion, as well as tiered royalties on net sales Spero plans to initiate a Phase 2 trial for IBI355 in Q2 2027 in IgG4-related disease (IgG4-RD), a serious, chronic fibroinflammatory condition with significant treatment burden and limited therapeutic options Innovent plans to initiate a Phase 2 trial in China by early 2027 for Sjögren's disease (SjD), a chronic autoimmune disorder with limited therapeutic options SAN FRANCISCO, CAMBRIDGE, Mass., and SUZHOU, China, July 14, 2026 /PRNewswire/ -- Innovent Biologics, Inc. (01801.HK) and Spero Therapeutics, Inc. (Nasdaq: SPRO) today announced an exclusive license agreement for IBI355 (Spero R&D code: SP001), a third-generation Fc-silent anti-CD40L antibody. Spero will receive exclusive rights to develop, research, manufacture, and commercialize IBI355 worldwide, excluding Greater China, where Innovent retains rights. Spero currently expects to advance IBI355 into a Phase 2 study in IgG4-RD patients in Q2 2027; Innovent expects to initiate a Phase 2 trial in China for SjD by early 2027. IBI355 (SP001) targets CD40L, an immune signal protein that sits upstream of multiple immune pathways and has the potential to redefine treatment paradigms across a range of immune-mediated diseases. Innovent has evaluated this antibody in two healthy volunteer Phase 1 trials: a single ascending dose (SAD) study and a multiple ascending dose (MAD) study. Innovent has also evaluated IBI355 in a Phase 1b MAD study in patients with SjD. Data from the Phase 1b SjD study were presented in a poster session at the EULAR 2026 Congress. "CD40L is an important upstream mediator of immune activation and T-cell/B-cell collaboration, both of which are relevant to IgG4-RD progression," said Dr. Arezou Khosroshahi, MD, Professor of Medicine, Emory University School of Medicine. "A non-B cell depleting approach that interrupts CD40L signaling could represent an important area for clinical evaluation in IgG4-RD, where patients need additional options that address chronic inflammation, relapse, and treatment burden." "IgG4-related disease, Sjögren's disease, and other autoimmune conditions impose significant burdens on patients worldwide, with many facing limited treatment options." said Dr. Lei Qian, Chief R&D Officer (General Biomedicine) of Innovent Biologics. "We are pleased to partner with Spero Therapeutics on IBI355 to advance the development of this CD40L-targeted approach. By combining Innovent and Spero's immunology expertise and development commitment, we aim to accelerate the delivery of differentiated therapies to patients living with these chronic conditions. Our shared goal is to bring meaningful new options to patients in need, wherever they are." "Having successfully advanced and delivered a therapy for patients with serious infectious diseases, we are now applying the same disciplined development approach to immune-mediated diseases. This in-licensing transaction establishes the foundation of our new pipeline and positions Spero to pursue potentially transformative therapies for patients living with chronic, debilitating conditions," said Esther Rajavelu, President and Chief Executive Officer of Spero Therapeutics. "We view CD40L as a clinically validated pathway with broad applicability across several immune-mediated diseases. In IgG4-RD, we believe this approach has the potential to meaningfully address the burden patients face from a chronic, relapsing condition with limited treatment options. Beyond IgG4-RD, we see opportunities to explore the role of CD40L inhibition across multiple autoimmune and inflammatory conditions. We are delighted to partner with Innovent Biologics, whose scientific leadership, development expertise, and commitment to innovation have been instrumental in advancing this program to its current stage." Under the terms of the license agreement, Spero will receive exclusive rights to develop, research, manufacture, and commercialize IBI355 worldwide, excluding Greater China (Mainland China, Hong Kong, Macau and Taiwan). Innovent will receive an upfront payment and is eligible to receive development, regulatory and commercial milestone payments, bringing total value of the deal to approximately $1.1 billion. Innovent is also eligible to receive tiered royalties on net sales generated in Spero's licensed territories. About IBI355 (SP001) IBI355 (SP001) is a third-generation, humanized, Fc-silent IgG1 monoclonal antibody targeting CD40L, an upstream immune activation signal involved in B-cell, antigen-presenting cell, and platelet biology. By blocking CD40L, IBI355 has the potential to provide a targeted non-B-cell-depleting treatment across multiple immune-mediated diseases where immune-cell interactions drive chronic inflammation, relapse, and tissue damage. IBI355 is designed to address platelet activation concerns associated with earlier anti-CD40L antibodies, while preserving key monoclonal antibody properties, including FcRn interaction that supports IgG-like half-life. About IgG4-Related Disease IgG4-related disease is a serious, chronic fibroinflammatory condition that can affect multiple organs, including the pancreas, salivary glands, lacrimal glands, kidneys, lungs, lymph nodes, and other tissues. The disease can cause immune-mediated inflammation, fibrosis, organ damage, relapse risk, and significant treatment burden. Current approaches can include steroids and B-cell-directed therapies, but additional options are needed to support durable disease control, reduce long-term treatment burden, and preserve immune function. About Sjögren's Disease (SjD) Sjögren's disease is a chronic, systemic autoimmune disorder that primarily affects the salivary and tear glands, but can also involve multiple organs in more severe cases. It is one of the most common autoantibody-driven diseases, yet no approved therapies address its underlying systemic nature, and it predominantly affects women. The disease is marked by autoantibody production, chronic inflammation, and lymphocytic infiltration, leading to symptoms such as dry eyes, dry mouth, joint pain, and fatigue. In some patients, organ involvement can include kidney or lung dysfunction, and there is an increased risk of non-Hodgkin's B-cell lymphoma. More than half of patients develop moderate-to-severe disease, with a burden comparable to other serious autoimmune conditions. About Innovent Biologics Innovent is a leading biopharmaceutical company founded in 2011 with the mission to empower patients worldwide with affordable, high-quality biopharmaceuticals. The company discovers, develops, manufactures and commercializes innovative medicines that target some of the most intractable diseases. Its pioneering therapies treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has launched 19 products in the market. It has 1 asset under NMPA review, 5 assets in phase 3 or pivotal clinical trials, and 14 more molecules in early clinical stage. Innovent partners with over 30 global healthcare companies, including Eli Lilly, Roche, Takeda, Pfizer, Sanofi, Incyte, LG Chem and MD Anderson Cancer Center. Guided by the motto, "Start with Integrity, Succeed through Action," Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit , or follow Innovent on Facebook and LinkedIn. Statement: Innovent does not recommend the use of any unapproved drug (s)/indication (s). Ramucirumab Injection (Cyramza®), Selpercatinib Capsules (Retsevmo®), Pirtobrutinib Tablets (Jaypirca®) and abemaciclib (Verzenios®) are products discovered and developed by Eli Lilly. About Spero Therapeutics Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases and major treatment gaps. The company's lead program, SP001, is a next-generation, Fc-silent anti-CD40L monoclonal antibody being advanced first in IgG4-related disease, with broader potential for additional immune-related conditions. For more information, visit . Forward-looking statement of Innovent Biologics This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent, are intended to identify certain of such forward-looking statements. Innovent does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of Innovent with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond Innovent's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, Innovent's competitive environment and political, economic, legal and social conditions. Innovent, the Directors and the employees of Innovent assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialize or turn out to be incorrect. Forward-Looking Statements of Spero Therapeutics This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements include, but are not limited to, statements regarding Spero's plans, strategy, and expectations for future development; the potential benefits of SP001; the potential of CD40L blockade in IgG4-related disease and other immune-related conditions; Spero's plans to advance SP001 into clinical development; anticipated development timelines, regulatory activities, and future milestones; and the potential benefits of Spero's agreement with Innovent. In some cases, forward-looking statements may be identified by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intent," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue," the negative of these terms or other similar expressions. Forward-looking statements are based on Spero's current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, among others, risks related to the completion and integration of the Innovent transaction; Spero's ability to successfully develop SP001; the timing and outcome of clinical trials and regulatory interactions; the ability to obtain and maintain regulatory approvals; potential safety, efficacy, manufacturing, supply, intellectual property, financing, competitive, and market risks; and other risks described in Spero's filings with the Securities and Exchange Commission. The forward-looking statements included in this press release represent Spero's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Spero undertakes no obligation to update any forward-looking statements, except as required by law. SOURCE Innovent Biologics 21% more press release views with Request a Demo

Phase 1License out/inImmunotherapy

14 Jul 2026

·firstwordpharma.com

Celcuity's PI3K therapy wins FDA OK in wild-type breast cancer

Shares in Celcuity ticked up 7% on Tuesday — then handed most of those gains back in after-hours trading — after the FDA approved Revtorpyk (gedatolisib), a comprehensive inhibitor of the PI3K/AKT/mTOR (PAM) pathway, in certain patients with breast cancer.The intravenously infused therapy was greenlit in combination with fulvestrant, with or without Pfizer's Ibrance (palbociclib), for adults with second-line or later HR-positive, HER2-negative locally advanced or metastatic breast cancer who lack a PIK3CA mutation, though the supporting Phase III VIKTORIA-1 study included both wild-type and mutant patient cohorts. HR-positive, HER2-negative disease accounts for about 70% of all breast cancers, of which around 40% have PIK3CA mutations.Both patient populations met their primary endpoint of progression-free survival (PFS), but the non-mutant cohort read out first, and its marketing submission was granted a priority review earlier this year. VIKTORIA-1 randomised a total of 701 PIK3CA-mutant and non-mutant patients to receive either Revtorpyk plus fulvestrant and Ibrance, a duo comprising Revtorpyk and fulvestrant, or fulvestrant alone.Last year, Celcuity reported data from the wild-type cohort showing a PFS benefit of 76% for the Revtorpyk triplet compared with fulvestrant monotherapy, with a median PFS of 9.3 months and 2 months, respectively. The doublet arm also achieved statistical significance with a median PFS of 7.4 months. In addition, the Revtorpyk-Ibrance-fulvestrant group achieved an objective response rate (ORR) of 32% while patients receiving the drug duo saw an ORR of 28%, and those in the fulvestrant arm had a 1% ORR (see – KOL Views Q&A: Celcuity's gedatolisib may be a bigger hit commercially than clinically in HR+ breast cancer). Celcuity has also generated positive data for Revtorpyk in the PIK3CA-mutant cohort of VIKTORIA-1 — which used fulvestrant plus Novartis' PI3Kα inhibitor Piqray (alpelisib) as the comparator, instead of fulvestrant alone — prompting the drugmaker to lay plans in May for a supplemental filing after the patient group met its PFS primary endpoint.Detailed results for the mutant cohort, presented at this year's American Society of Clinical Oncology (ASCO) meeting, showed that the triplet regimen and the Revtorpyk duo reduced the risk of disease progression or death by 50% and 49%, respectively, versus Piqray plus fulvestrant. Median PFS for the triple combination group and the experimental pair were 11.1 months and 11.3 months, respectively, compared with 5.6 months for Piqray plus fulvestrant.PI3K problems?The biotech's see-sawing stock may have been prompted by a lack of enthusiasm for Revtorpyk among physicians. Following Celcuity's ASCO presentation, FirstWord spoke with Denise Yardley, associate director of breast cancer research at Sarah Cannon Research Institute, about the drug's Piqray-beating data (see – KOL Views Q&A: Breast cancer at ASCO – first take on AZ and Roche oral SERD debates, Gilead's Trodelvy TNBC data).While Yardley acknowledged that Revtorpyk "looks better than Piqray" in terms of efficacy, she noted that Celcuity's drug requires intravenous delivery, "which is a big burden in a setting where we were asking people to take a pill that allowed them to stay out of the clinic.""It is going to be challenging for us to use something that does not necessarily improve quality of life and is more complicated, especially when the benefit is not huge," she added.Yardley also described PI3K-targeting compounds as "hard agents to use" in general because of their slate of side effects, which includes rash, diarrhoea and hyperglycaemia. More from the FDAIn other regulatory decisions, the FDA on Tuesday also converted the accelerated approval for Eli Lilly's Retevmo (selpercatinib) into a full approval for adults and paediatric patients two years and older with locally advanced or metastatic solid tumours with a RET gene fusion who have progressed on or following prior systemic treatment, or who have no alternative treatment options.The oral RET kinase inhibitor was initially cleared for adults in 2022 and for children in 2024. At ASCO, Lilly presented data in early-stage, RET fusion-positive non–small-cell lung cancer showing that Retevmo reduced the risk of recurrence or death by about 83% over placebo.

14 Jul 2026

·www.fda.gov

FDA grants traditional approval to selpercatinib for locally advanced or metastatic RET fusion-positive solid tumors

On July 14, 2026, the Food and Drug Administration granted traditional approval to selpercatinib (Retevmo, Eli Lilly and Company) for adult and pediatric patients two years of age and older with 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. Selpercatinib received accelerated approval for this indication for adult patients in 2022 and for pediatric patients two years of age and older in 2024.Full prescribing information for Retevmo will be posted on Drugs@FDA.Efficacy and SafetyEfficacy was evaluated in a multicenter, open-label, multi-cohort trial (LIBRETTO-001, NCT03157128) and supported by results in RET fusion-positive non-small cell lung cancer (NSCLC) and RET fusion-positive thyroid cancer. Efficacy was evaluated in 75 patients with RET fusion-positive tumors other than NSCLC and thyroid cancer with disease progression on or following prior systemic treatment or who had no satisfactory alternative treatment options.The major efficacy outcome measures were overall response rate (ORR) and duration of response (DOR). ORR was 47% (95% CI: 35, 59), with a median DOR of 24.5 months (95% CI: 11.2, 49.1). Tumor types with responses included colorectal, pancreatic adenocarcinoma, salivary, soft tissue sarcoma, cholangiocarcinoma, carcinoma of the skin, unknown primary, breast, bronchial carcinoid, ovarian, small intestine, and neuroendocrine (including pancreatic).Efficacy in pediatric and young adult patients was evaluated in LIBRETTO-121 (NCT03899792), a multicenter, open-label, multi-cohort trial. Efficacy was evaluated in patients with locally advanced refractory RET fusion-positive solid tumors who were unresponsive to available therapies or had no standard systemic curative therapy available. Responses were observed in one patient with congenital infantile fibrosarcoma, one patient with a spindle cell sarcoma, and in patients with RET fusion-positive thyroid cancer.The prescribing information includes warnings and precautions for hepatotoxicity, interstitial lung disease/pneumonitis, hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, tumor lysis syndrome, risk of impaired wound healing, hypothyroidism, embryo-fetal toxicity, and slipped capital femoral epiphysis/slipped upper femoral epiphysis in pediatric patients.Recommended DosageThe recommended selpercatinib dose for adult and adolescent patients 12 years of age or older based on body weight is:Less than 50 kg: 120 mg orally twice daily50 kg or greater: 160 mg orally twice dailySee the prescribing information for the recommended dose for pediatric patients two to less than 12 years of age.This review used the Assessment Aid, a voluntary submission from the applicant to facilitate FDA’s assessment. The FDA approved this application two months ahead of the FDA goal date.Selpercatinib received orphan drug designation for the tissue agnostic indication. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.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.Follow the Oncology Center of Excellence on X @FDAOncology.

Orphan DrugDrug ApprovalClinical ResultAccelerated ApprovalClinical Study

100 Deals associated with Selpercatinib

External Link

KEGG	Wiki	ATC	Drug Bank
D11713	Selpercatinib	L01XE	DB15685

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
RET fusion-positive Solid Tumors	United States	Eli Lilly & Co.	10 Apr 2024
metastatic non-small cell lung cancer	United States	Eli Lilly & Co.	21 Sep 2022
Metastatic Solid Tumor	United States	Eli Lilly & Co.	21 Sep 2022
Thyroid Cancer, Medullary	United States	Eli Lilly & Co.	21 Sep 2022
RET fusion-positive Non-Small Cell Lung Cancer	European Union	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Non-Small Cell Lung Cancer	Iceland	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Non-Small Cell Lung Cancer	Liechtenstein	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Non-Small Cell Lung Cancer	Norway	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Thyroid Cancer	European Union	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Thyroid Cancer	Iceland	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Thyroid Cancer	Liechtenstein	Eli Lilly Nederland BV	11 Feb 2021
RET fusion-positive Thyroid Cancer	Norway	Eli Lilly Nederland BV	11 Feb 2021
Non-Small Cell Lung Cancer	United States	Eli Lilly & Co.	08 May 2020
RET Mutation-Positive Medullary Thyroid Cancer	United States	Eli Lilly & Co.	08 May 2020
Thyroid Cancer	United States	Eli Lilly & Co.	08 May 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Differentiated Thyroid Gland Carcinoma	Phase 2	United States	Eli Lilly & Co.	29 Jul 2024
Thyroid Cancer, Follicular	Phase 2	United States	Eli Lilly & Co.	01 Mar 2023
Locally Advanced Malignant Solid Neoplasm	Phase 2	China	Eli Lilly & Co.	16 Mar 2020
Central Nervous System Neoplasms	Phase 2	United States	Eli Lilly & Co.	13 Jun 2019
Central Nervous System Neoplasms	Phase 2	Japan	Eli Lilly & Co.	13 Jun 2019
Central Nervous System Neoplasms	Phase 2	Australia	Eli Lilly & Co.	13 Jun 2019
Central Nervous System Neoplasms	Phase 2	Canada	Eli Lilly & Co.	13 Jun 2019
Central Nervous System Neoplasms	Phase 2	Denmark	Eli Lilly & Co.	13 Jun 2019
Central Nervous System Neoplasms	Phase 2	France	Eli Lilly & Co.	13 Jun 2019
Central Nervous System Neoplasms	Phase 2	Germany	Eli Lilly & Co.	13 Jun 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04819100 (LIBRETTO-432, Pubmed \| N Engl J Med)	Phase 3	RET fusion-positive Non-Small Cell Lung Cancer Adjuvant RET fusion-positive	151	Selpercatinib	jzgelhyapj(rpzsyrmrns) = tkvgenaytz cwvwyohaxt (kyzuaqegxg ) View more	Positive	31 May 2026
				Placebo	jzgelhyapj(rpzsyrmrns) = uplbhousmi cwvwyohaxt (kyzuaqegxg ) View more
NCT04819100 (LIBRETTO-432, ASCO2026)	Phase 3	RET fusion-positive Non-Small Cell Lung Cancer Adjuvant RET fusion-positive	151	Selpercatinib 160mg BID	scduoqhqjr(xemuycboiz) = bnhymrnwir dxgabdqcgs (spdowceblt )	Positive	29 May 2026
				Placebo	scduoqhqjr(xemuycboiz) = aaeufbpepp dxgabdqcgs (spdowceblt ) View more
SPRINT-RET (ESMO_ELCC2026)	Not Applicable	RET fusion-positive Non-Small Cell Lung Cancer RET fusion-positive	52	Selpercatinib	zhsjvfxtxr(fjhwsqcjsg) = ybjsmogwyh fskjtnfonh (cfmddlozyy ) View more	Positive	25 Mar 2026
NCT03899792 (LIBRETTO-121, CTgov)	Phase 1/2	RET mutation Solid Tumors \| Papillary thyroid cancer \| Central Nervous System Neoplasms ... View more	36	Selpercatinib	ihibymymgj(jfwhmkyufc) = mdmbbougea rqmfnlqqfv (wipswecdpk, lazqukuaxx - opypnlcbwi) View more	-	06 Jan 2026
NCT05469113 (CTgov)	Phase 1	-	16	Repaglinide (Period 1: Repaglinide)	tlzchoigqx(kqjekvcigz) = hqqzzbgnsv huilzcmeil (jelphndyds, 57.1) View more	-	11 Dec 2025
				Selpercatinib+Repaglinide (Period 2: Selpercatinib + Repaglinide)	tlzchoigqx(kqjekvcigz) = xvujsmuitg huilzcmeil (jelphndyds, 53.2) View more
NCT04320888 (CTgov)	Phase 2	Refractory Soft Tissue Sarcoma \| Ependymoma \| Relapsed Solid Neoplasm ... View more	1	Computed Tomography+Selpercatinib	ciibteimuk = zfmrpukrke ybcwuxvssr (csesnqclqy, nxogbzshtm - zgnupmvdlc) View more	-	06 Nov 2025
NCT05338476 (CTgov)	Phase 1	-	16	Midazolam	zbdkqkfcjh(xflpqkpcjt) = imffdrmdqp xmzauxzncd (mduqylgnce, 38.4) View more	-	20 Oct 2025
NCT05468164 (CTgov)	Phase 1	-	20	Selpercatinib (Treatment A: 160 mg Selpercatinib (Fasted))	rwkrylazhy(fkhvrtexlj) = pwuufdonqo ddduasrxqw (zoztrbcsun, 38.9) View more	-	19 Sep 2025
				Selpercatinib (Treatment B: 160 mg Selpercatinib (Fed))	rwkrylazhy(fkhvrtexlj) = bmzdcvbsnz ddduasrxqw (zoztrbcsun, 20.4) View more
NCT05338489 (CTgov)	Phase 1	-	24	Selpercatinib (Part 1 Period 1: 160 mg Selpercatinib)	wbznbygtyy(uempzoufpd) = kvaqpdyxlb fkoxxsjkxf (hxshskbngn, 31.8) View more	-	19 Sep 2025
				Itraconazole+Selpercatinib (Part 1 Period 2: 160 mg Selpercatinib + 200 mg Itraconazole)	wbznbygtyy(uempzoufpd) = wytsuvaodj fkoxxsjkxf (hxshskbngn, 41.9) View more
NCT05469100 (CTgov)	Phase 1	Renal Insufficiency	37	Selpercatinib (Selpercatinib (Control; Normal Renal Function))	aorafpcgmj(yeucuqkxpl) = tniphnsfxl zyqbnvbyhj (zsfgraquuy, 52.1) View more	-	18 Sep 2025
				Selpercatinib (Selpercatinib (Mild Renal Impairment))	aorafpcgmj(yeucuqkxpl) = ltzhtfnvuv zyqbnvbyhj (zsfgraquuy, 36.0) View more

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