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Last update 14 Sep 2026

Lomitapide Mesylate

Last update 14 Sep 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Lojuxta, lomitapide, Lomitapide Mesilate

+ [7]

Target

MTTP

Action

inhibitors

Mechanism

MTTP inhibitors(Microsomal triglyceride transfer protein inhibitors)

Therapeutic Areas

Congenital DisordersEndocrinology and Metabolic DiseaseOther Diseases+ [2]

Active Indication

Hyperlipidemia Type IIaHomozygous familial hypercholesterolemiaColorectal Cancer

Inactive Indication

Familial chylomicronaemia syndrome

Originator Organization

Bristol Myers Squibb Co.

Active Organization

Chiesi Ltd.Recordati Rare Diseases Japan KK

Ersteq Co., Ltd.

+ [8]

Inactive Organization

Aegerion Pharmaceuticals, Inc.

License Organization

GryNumber Health

Amryt Pharma Holdings Ltd.

Medison Pharma Ltd.

+ [9]

Drug Highest PhaseApproved

First Approval Date

United States (21 Dec 2012),

Homozygous familial hypercholesterolemia

RegulationOrphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Overseas New Drugs Urgently Needed in Clinical Settings (China)

Structure/Sequence

Molecular FormulaC40H41F6N3O5S

InChIKeyQKVKOFVWUHNEBX-UHFFFAOYSA-N

CAS Registry202914-84-9

Clinical Trials associated with Lomitapide Mesylate

NCT04681170

/ CompletedPhase 3

Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid Lowering Therapy

This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) comprising of the following phases:
* Screening Period (starting at Week 12, i.e. ≤12 weeks prior to Baseline for up to 6 weeks)
* Stratified Enrolment and Start of Run in Period (starting at minimum at Week 6, i.e., 6 weeks prior to Baseline for a minimum of 6 weeks):
* Efficacy Phase (starting at Baseline, i.e. Day [D] 0 for 24 weeks±3 days
* Safety Phase (starting at Week 24±3 days for 80±1 weeks)

Start Date14 Dec 2020

Sponsor / Collaborator

Amryt Pharma Holdings Ltd.

EUCTR2018-002911-80-IT

/ CompletedPhase 3

Open-label study to evaluate the safety, tolerability, and efficacy of LOmitapide for the treatment of patients with Familial CHylomicroNEmia Syndrome

Start Date18 Dec 2018

Sponsor / Collaborator

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

NCT02765841

/ WithdrawnPhase 3

A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy

This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL apheresis. The study is comprised of a 12-week Run-in Period, a primary 24-week Efficacy Phase, followed by an 80-week Safety Phase.

Start Date01 May 2016

Sponsor / Collaborator

Aegerion Pharmaceuticals, Inc.

100 Clinical Results associated with Lomitapide Mesylate

100 Translational Medicine associated with Lomitapide Mesylate

100 Patents (Medical) associated with Lomitapide Mesylate

261

Literatures (Medical) associated with Lomitapide Mesylate

20 Jul 2026·BRITISH JOURNAL OF CANCER

Inhibition of the SerpinB3/protease-activated receptor 2 axis reduces liver cancer development and affects lipid metabolism

Article

Author: Martini, Andrea ; Peña-SanFelix, Patricia ; Oliviero, Barbara ; Quarta, Santina ; Parola, Maurizio ; Guerra, Pietro ; Rejano-Gordillo, Claudia M ; Gambella, Alessandro ; De Siervi, Silvia ; Sanz-Parra, Arantza ; Barrenechea-Barrenechea, Jon Ander ; Villano, Gianmarco ; Cannito, Stefania ; Mantovani, Stefania ; Guido, Maria ; González-Recio, Irene ; Ruvoletto, Mariagrazia ; Cagnin, Silvia ; Zapata-Pavas, L Estefanía ; Pontisso, Patrizia ; Nurcis, Jessica ; Turato, Cristian ; Goikoetxea-Usandizaga, Naroa ; Biasiolo, Alessandra ; Gil-Pitarch, Clàudia ; Martínez-Chantar, María Luz

BACKGROUND:

MASLD and HCC are increasing challenges in hepatology. 1-Piperidinepropionic acid (1-PPA), a protease-activated receptor 2 (PAR2) inhibitor, downregulates SerpinB3, a molecule involved in fibrosis and HCC. This study investigates the effects of 1-PPA on lipid accumulation and HCC development.

METHODS:

1-PPA was employed in primary hepatocytes and human liver organoids cultured with steatogenic compounds and lomitapide, a VLDL-formation inhibitor. Antitumoral effects of 1-PPA were evaluated by proliferation and invasion assays in HepG2 cells and in human liver organoids treated with a tumorigenic compound. MASH-related carcinogenesis was studied in vivo using C57BL/6J mice overexpressing SerpinB3 (C57/TG) and BALB/c mice deficient in the reactive site loop of Serpinb3a (BC/KO).

RESULTS:

1-PPA reduced tumour development and steatosis in vivo. Proteomic analysis showed decreased lipid synthesis and deposition post-treatment. Suppression of lipid accumulation was favoured by an increase in VLDL export, supported by an enhanced microsomal triglyceride transfer protein activity in vivo and by a competitive effect between 1-PPA and lomitapide in vitro. The compound 1-PPA also exhibited direct antitumoral effects, reducing proliferation, survival and invasion in liver organoids and HepG2 cells.

CONCLUSIONS:

1-PPA administration prevents lipid accumulation and HCC development in MASH-related liver carcinogenesis.

11 Jun 2026·JOURNAL OF PHYSICAL CHEMISTRY B

Computational Drug Repurposing Predicts FDA-Approved Drugs as Potential Inhibitors of Chikungunya Virus nsP2 Protease

Article

Author: Ngo, Son Tung ; Phung, Huong Thi Thu ; Thai, Quynh Mai

Chikungunya virus (CHIKV) infection remains a significant global health threat with no approved specific antiviral therapy. The nonstructural protein 2 protease (nsP2pro) is essential for viral replication and represents an attractive target for structure-based drug design. In this study, we employed a computational drug repurposing strategy to predict potential nsP2pro inhibitors from FDA-approved compound libraries. We performed molecular docking screens of 1820 unique compounds against the nsP2pro active site. The eight selected top-scoring compounds had docking scores better than that of the reference inhibitor pantinin-1. Subsequent molecular dynamics (MD) simulations revealed that, except for lomitapide, which dissociated from the pocket, the remaining leads formed dynamically stable complexes and modulated the catalytic environment in a chemotype-dependent manner. Small molecules expanded both the Cys478-His548 and Ser482-His548 distances, consistent with dyad perturbation, whereas GnRH-like peptides preserved a more compact geometry in the modeled noncovalent complexes, pointing to possible nonproductive occupation of the active-site region. Free energy perturbation calculations further provided a relative energetic ranking that supported the MD-derived mechanistic trends and placed six compounds ahead of pantinin-1 within the present data set. Among the small-molecule candidates, venetoclax and lapatinib emerge as computationally prioritized candidates for future biochemical and antiviral evaluation. Overall, this study provides mechanistic insight into noncovalent recognition of CHIKV nsP2pro and a framework for future experimental validation.

01 Jun 2026·CARDIOVASCULAR DRUGS AND THERAPY

Lomitapide, a Microsomal Triglyceride Transfer Protein Inhibitor, in Homozygous Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis of Efficacy and Safety

Review

Author: Abdelrahman Shata ; George Hanen ; Hazem E. Mohammed ; Mohamed Khalafalla Darwish ; Mohamed E. Haseeb ; Mohamed Nasser ; Nada A. Abdelaziz ; Anas Hussien Heiba ; Mohamed Salem Abdelkader

Abstract:

Purpose:

Homozygous Familial Hypercholesterolemia (HoFH) is a rare and life-threatening genetic disorder characterized by elevated low-density lipoprotein cholesterol (LDL-C) and early-onset atherosclerotic cardiovascular disease. Lomitapide, a microsomal triglyceride transfer protein (MTP) inhibitor, decreases LDL-C independent of LDL receptor function, providing an alternative treatment in this population. We aimed to evaluate the efficacy and safety of lomitapide in patients with HoFH through a systematic review and meta-analysis of available clinical evidence.

Methods:

A comprehensive search was conducted in PubMed, Scopus, and Web of Science through March 2025. Observational studies and clinical trials reporting on lipid profile changes and safety outcomes in HoFH patients receiving lomitapide were included. Outcomes were pooled using random-effects models, and heterogeneity was assessed using the I 2 statistic.

Results:

Eight studies comprising both adult and pediatric patients (n = 209) were included. Lomitapide significantly reduced LDL-C levels by 49.27%, total cholesterol by 46.05%, and apolipoprotein B by 51.01%. Reductions were also observed in triglycerides, VLDL-C, and non–HDL-C. HDL-C remained relatively unchanged. Adverse events were mostly gastrointestinal, with a 14% discontinuation rate. The overall quality of studies ranged from fair to good.

Conclusions:

Lomitapide demonstrates substantial efficacy in reducing LDL-C and other atherogenic lipids in HoFH patients, with an acceptable safety profile. These findings support its role as an adjunctive therapy in this population, though further randomized controlled trials are warranted to validate long-term safety and effectiveness.

News (Medical) associated with Lomitapide Mesylate

10 Sep 2026

·www.biospace.com

Retension Pharmaceuticals Appoints LeAnne Bloedon, M.S., RD, as EVP of Clinical Development and Operations, and Kevin Schutz, PharmD, as VP Regulatory Affairs

Appointments strengthen clinical and regulatory leadership as the Company advances RTN-001 through its Phase 2b trial FALLS CHURCH, Va.--(BUSINESS WIRE)--Retension Pharmaceuticals, Inc. (“Retension” or the “Company”), a clinical-stage biopharmaceutical company focused on developing medicines for the treatment of hypertension and other cardiovascular diseases, today announced that LeAnne Bloedon, M.S., RD, and Kevin Schutz, PharmD, have joined the Company as Executive Vice President, Clinical Development and Operations, and as Vice President, Regulatory Affairs, respectively. “LeAnne and Kevin are accomplished leaders who have taken novel cardiovascular and metabolic therapies from clinical development through global approval,” said Eric Keller, Chief Executive Officer of Retension Pharmaceuticals. “LeAnne’s decades of clinical development and operational leadership, together with Kevin’s regulatory strategy across the U.S. Food and Drug Administration (“FDA”), the European Medicines Agency (“EMA”), and other regulatory authorities, will strengthen our operational capabilities. Their experience will be instrumental as we advance RTN-001 through its ongoing Phase 2b trial and prepare for the next stages of development.” LeAnne Bloedon, M.S., RD — Executive Vice President, Clinical Development and Operations Ms. Bloedon brings more than 25 years of clinical development leadership across cardiovascular disease, metabolic disorders, and rare disease, and has been instrumental in the development of multiple first-in-class medications approved in the U.S., Europe, and beyond. Prior to joining Retension, she served as Vice President, Head of Development at Esperion Therapeutics, where she spent 10 years in roles of increasing responsibility, including leading or co-leading the clinical strategy behind bempedoic acid’s approval in 42 countries, and overseeing all clinical trials, medical affairs activities, and pharmacovigilance for Nexletol®, Nexlizet®, and Enbumyst™. Prior to her role at Esperion Therapeutics, Ms. Bloedon was at Aegerion Pharmaceuticals, where she was responsible for the clinical trials supporting the approval of lomitapide in 38 countries. Earlier in her career, she led discovery efforts at the University of Pennsylvania to address key challenges in administering lomitapide. Ms. Bloedon holds a Master of Science in Nutrition from Case Western Reserve University and a Bachelor of Science in Public Health from the University of North Carolina at Chapel Hill. Kevin Schutz, PharmD — Vice President, Regulatory Affairs Dr. Schutz is a regulatory affairs leader with more than 20 years of experience with particular expertise in cardiology and cardiometabolic disease, guiding small and mid-sized companies through interactions with global health authorities and drug development strategy. Prior to joining Retension, Dr. Schutz served as Vice President, Global Regulatory Affairs at Corxel Pharmaceuticals, where he led the regulatory function for three development programs in hypertension, endocrinology and neurology. Prior to joining Corxel Pharmaceuticals, Dr. Schutz led regulatory efforts at CinCor Pharma, a clinical-stage company focused on treatments for resistant and uncontrolled hypertension, which was acquired by AstraZeneca in 2023. There, he managed key FDA interactions, including the End-of-Phase 2 meeting that supported Phase 3 protocol development. He has extensive experience working with global health authorities, including the FDA, EMA, and Japan’s Pharmaceuticals and Medical Devices Agency. Earlier in his career, Dr. Schultz was in roles of increasing responsibility at Insmed, Ipsen, Amarin, and EKR Therapeutics. Dr. Schutz earned a Doctor of Pharmacy (PharmD) and a Bachelor in Science in Pharmacy from the University of the Sciences in Philadelphia. About Retension Pharmaceuticals Retension Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing medicines for the treatment of hypertension and other cardiovascular conditions. The company’s lead clinical candidate, RTN-001, is in development for patients with uncontrolled and resistant hypertension. About RTN-001 Retension Pharmaceuticals is developing RTN-001, a cardiovascular-targeted, next-generation PDE5 inhibitor designed to directly enhance nitric oxide signaling in central arteries not adequately reached by first-generation PDE5 inhibitors, thereby addressing a core driver of hypertension. RTN-001 has been studied in multiple clinical trials totaling 265 subjects. RTN-001 has a demonstrated efficacy and safety pro patients and healthy volunteers with clinically meaningful reductions in blood pressure. A Phase 2b trial of RTN-001 in hypertension is ongoing. RTN-001 is being developed under an exclusive world-wide license from Sanofi S.A. For more information, please see Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements, which statements involve substantial risks and uncertainties. All statements other than statements of historical fact, including statements regarding Retension’s future results of operations or financial condition, business strategy and plans, and objectives of management for future operations, are forward-looking statements. These forward-looking statements include, among others, statements relating to Retension’s ability to obtain regulatory approval of its current and future product candidates, the timing, progress and results of its clinical trials, its market opportunity and the potential growth of that market, and other similar matters. These forward-looking statements are based on Retension’s current expectations and assumptions about future events, which are inherently subject to uncertainties, risks and changes in circumstances that are difficult to predict. Retension’s actual results may differ materially from those expressed in, or implied by, such forward-looking statements. Retension undertakes no obligation to update any forward-looking statements after the date of this press release or to conform such statements to actual results or revised expectations, except as required by law. Contacts Press and Investor Contact Alex Schwartz Chief Financial Officer investors@retensionpharmaceuticals.com

28 Jul 2026

·www.businesswire.com

Shionogi Announces U.S. Availability of XOCOVA ® (ensitrelvir), the First and Only Oral Option to Help Prevent COVID-19 Following Exposure*

FLORHAM PARK, N.J.--(BUSINESS WIRE)--Shionogi Inc., a New Jersey-based subsidiary of Shionogi & Co., Ltd., (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, PhD; hereinafter “Shionogi”) announced U.S. availability of XOCOVA® (ensitrelvir), an oral antiviral, approved for post-exposure prophylaxis (PEP) of COVID‑19 in adults and adolescents 12 years of age and older following contact with an individual who has COVID-19.2 XOCOVA offers a new approach to help prevent COVID-19 by blocking viral replication during the critical window between exposure and COVID-19 symptom onset.4,5 XOCOVA is taken once daily, with three 125 mg tablets (375 mg) taken at the same time on the first day, followed by one 125 mg tablet on days two through five. XOCOVA should be taken as soon as possible and within 72 hours following contact with an individual who has COVID‑19.1 “With XOCOVA, we have a novel oral option to help prevent COVID-19 after exposure, and it is available just in time for the annual late summer spike,” said Charles Vega, MD, FAAFP, Health Sciences Clinical Professor, University of California, Irvine, School of Medicine. “Taking XOCOVA to help prevent COVID-19 in the critical window between exposure and symptom onset makes sense for anyone who wants to try to avoid illness, and it could be especially beneficial for older adults and individuals with health conditions that may put them at higher risk for severe illness. Now, in addition to recommending my patients stay updated with vaccinations, I’ll be talking to them about XOCOVA, so they are ready to act quickly in the event of an exposure.” The U.S. has experienced a COVID-19 summer spike every year since 2020.3 Biobot Analytics, a wastewater intelligence company with continuous and longitudinal data on pathogens from wastewater, reports that SARS-CoV-2 levels are beginning to rise, providing an early, population-level signal that another COVID-19 summer wave may be taking shape.6 Scenario modeling from The U.S. Centers for Disease Control and Prevention indicates there may be increased COVID-19 activity in regions (South and West) which did not experience a substantial level of COVID-19 activity during the most recent fall and winter months.3 “Post-exposure prophylaxis, or PEP for short, has been used successfully with influenza as well as HIV and other sexually transmitted infections, and it has the potential to help reduce the burden of COVID-19,” said David Wohl, MD, Professor of Medicine, The University of North Carolina at Chapel Hill, School of Medicine. “The availability of XOCOVA means that now, for the first time, there’s an oral option that can help prevent COVID-19 after exposure. Since XOCOVA must be taken within 72 hours of exposure, having it on hand can help ensure it is taken within the critical window between exposure and COVID-19 symptom onset.” Having XOCOVA on hand can help appropriate patients be prepared in the event of a COVID-19 exposure. Before taking XOCOVA, individuals should review the dosing and administration instructions and check the expiration date on the blister pack. They should also speak with a healthcare professional if there are any changes to their medications (including prescription and over-the-counter medicines), vitamins or supplements and verify they are not pregnant or breastfeeding. Shionogi is supporting the availability of XOCOVA through professional and consumer education and partnerships with national and regional pharmacies to ensure product availability. Select retail pharmacies across the U.S. will have XOCOVA in stock, and all retail pharmacies can order it from Shionogi’s wholesale distribution partners. Shionogi has a commercial co-pay offer for XOCOVA that helps eligible, commercially insured patients with out-of-pocket costs. Eligible patients may pay as little as $0 per prescription fill. Details about eligibility are available at https://www.xocova.com/. All components of XOCOVA are produced in the U.S. including active pharmaceutical ingredient production, drug product formulation, packaging, quality control, release testing, and supply chain and distribution. The Ongoing Impact of COVID-19 COVID-19 remains highly transmissible, driven by Omicron and its subvariants, and up to 47% of people living with an infected individual may develop COVID-19.7 The U.S. Centers for Disease Control and Prevention estimates that between October 1, 2025, and July 11, 2026, there were 4.2-12.7 million new cases in the U.S., resulting in 840,000 – 2.3 million outpatient visits, 130,000 – 250,000 hospitalizations and 14,000 - 42,000 deaths.8 Beyond acute infection, COVID-19 can have lasting impacts. People diagnosed with COVID-19 had increased rates of both new and worsening neurologic, cardiovascular, respiratory, and chronic kidney conditions during the year following infection.9-,10,11,12,13,14,15,16,17,18 COVID-19 also disproportionately affects older adults with greater risk for severe illness and death in close-community settings, such as long-term care facilities.19 About SCORPIO-PEP The global, double-blind, randomized, placebo-controlled Phase 3 study, SCORPIO-PEP, assessed the safety and efficacy of XOCOVA as post-exposure prophylaxis for COVID-19. The study was conducted from June 2023–September 2024. It included 2,387 study participants aged 12 years and older with a negative local screening test for SARS-CoV-2 infection and no symptoms at the time of enrollment, who were exposed to a person living in their household with symptomatic COVID-19. The primary analysis included 2,041 household contact participants with a central laboratory-confirmed negative SARS-CoV-2 test at baseline. More than 98% of household contacts tested positive for antibodies against SARS-CoV-2 N (nucleocapsid) or S (spike) proteins, indicating that nearly all had evidence of previous SARS-CoV-2 infection or vaccination, or both.20 Study participants were randomly assigned at a 1:1 ratio to receive XOCOVA (375 mg on Day 1 and 125 mg on Days 2-5) or placebo, once daily, and began treatment within 72 hours of when the household member with COVID-19 began showing symptoms. Participants then continued XOCOVA (125 mg) or placebo for five days. In the primary analysis population, XOCOVA significantly reduced the risk of symptomatic COVID-19 by 67% in individuals following exposure to an infected individual through Day 10 compared with placebo (ensitrelvir n=1,030; placebo n=1,011).2 The most common adverse events (regardless of causality) occurring in greater than or equal to 1% of the XOCOVA group and at a greater frequency compared to placebo were headache, diarrhea, and cough.1 There were no reports of altered taste (dysgeusia) attributed to XOCOVA in the trial.2,20 Results from the SCORPIO-PEP trial were published in the New England Journal of Medicine on May 14, 2026. SCORPIO-PEP is the first and only Phase 3 study of an oral antiviral to meet the primary endpoint of preventing COVID-19 following exposure to an infected individual. About XOCOVA XOCOVA® (ensitrelvir) is a SARS-CoV-2 main protease inhibitor created through joint research between Hokkaido University and Shionogi. SARS-CoV-2 has an enzyme called the main protease, which is essential for the replication of the virus. XOCOVA suppresses the replication of SARS-CoV-2 by selectively inhibiting the main protease. XOCOVA is approved in the U.S. and Japan for post-exposure prophylaxis of COVID‑19 in adults and adolescents 12 years of age and older following contact with an individual who has COVID-19. XOCOVA received emergency regulatory approval in Japan in November 2022 and full approval in March 2024 for the treatment of COVID-19 in adults and adolescents 12 years of age and older based on results from SCORPIO-SR, a Phase 3 study conducted in Asia during the Omicron-dominant phase of the pandemic. Results from this study were published in JAMA Network Open. XOCOVA received supplemental approval in June 2026 in Japan for pediatric patients aged 6 to under 12 years who weigh at least 20 kg for the treatment of COVID-19. XOCOVA is not approved for the treatment of COVID-19 in the U.S. INDICATION XOCOVA (ensitrelvir) is a prescription medicine used to help prevent coronavirus disease 2019 (COVID-19) in adults and children aged 12 years and older following contact with a person who has COVID-19 (post-exposure prophylaxis). It is not known if XOCOVA is safe and effective in children under age 12. IMPORTANT SAFETY INFORMATION Do not take XOCOVA if you are: allergic to ensitrelvir or any of the ingredients in XOCOVA. take any of the following medicines, as these may interact with XOCOVA and may cause severe or life-threatening side effects or death: apalutamide, carbamazepine, colchicine, dihydroergotamine, enzalutamide, eplerenone, ergotamine, finerenone, ivabradine, lomitapide, lumacaftor/ivacaftor, lurasidone, methylergonovine, phenytoin, pimozide, quinidine, rifampin, St. John’s wort, simvastatin, triazolam or voclosporin. These are not the only medicines that may cause serious or life-threatening side effects if taken with XOCOVA. XOCOVA may increase or decrease the levels of many medicines in your body. Tell your healthcare provider (HCP) about all the medicines you take because additional laboratory tests or dose changes of your other medicines may be necessary during XOCOVA use. Your HCP may tell you about specific symptoms to watch for that may indicate you need to stop or decrease the dose of some of your other medicines. Before taking XOCOVA, tell your HCP about all your medical conditions, including if you: are pregnant or plan to become pregnant. XOCOVA may harm your unborn baby. For females who are able to become pregnant, your HCP will check with you to see if you are pregnant before starting XOCOVA. Use effective birth control (contraception) during XOCOVA use and for 2 weeks after the final dose. Tell your HCP right away if you become pregnant or think you may be pregnant during XOCOVA use. are breastfeeding or plan to breastfeed. It is not known if XOCOVA passes into your breast milk. Do not breastfeed during XOCOVA use and for 2 weeks after the final dose. Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Your HCP can tell you if it is safe to take XOCOVA with other medicines. Do not start a new medicine without telling your HCP. What are the possible side effects of XOCOVA? XOCOVA may cause serious side effects, including: Allergic reactions, including severe allergic reactions (anaphylaxis). Stop taking XOCOVA and get medical help right away if you get any of the following allergic reaction symptoms: skin rash; hives; itching; swelling of the mouth, lips, tongue, or face; trouble swallowing or breathing; throat tightness; dizziness or feeling lightheaded; stomach-area (abdomen) pain; or vomiting. The most common side effects of XOCOVA include: headache, diarrhea, and cough. These are not all possible side effects of XOCOVA. For more info, ask your HCP or pharmacist. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Please see full Prescribing Information, including Patient Information, for XOCOVA. About Shionogi Inc. Shionogi Inc. is a U.S. subsidiary of Shionogi & Co., Ltd., a global research‑driven pharmaceutical company committed to bringing to market the best possible medicines to aid the health and well-being of the patients we serve. Our global expertise in infectious disease began in the 1950s and in addition to discovering and developing multiple innovative antibiotics, today our global portfolio includes an antibiotic for the treatment of serious Gram‑negative bacterial infections, antivirals for COVID-19, HIV and influenza and a therapeutic for rare fungal infections. Our U.S. infectious disease franchise includes both approved and investigational novel anti-infectives, and investigational antivirals for COVID-19 and RSV. In parallel, we are advancing a U.S. rare disease franchise that includes an approved therapy for amyotrophic lateral sclerosis (ALS), along with clinical programs in Fragile X syndrome, Jordan’s syndrome, and Pompe disease. We are also pursuing research in additional high‑unmet need therapeutic areas, including oncology, acute ischemic stroke, sleep apnea, and other quality of life areas. Learn more at shionogi.com. About Shionogi & Co., Ltd. Shionogi & Co., Ltd. is a 148-year-old global, research-driven pharmaceutical company headquartered in Osaka, Japan, that is dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.” The company currently markets products in several therapeutic areas including anti-infectives, pain, CNS disorders and cardiovascular diseases. Shionogi’s research and development currently targets two therapeutic areas: infectious diseases and diseases with unmet medical needs in pain/CNS, including Alzheimer’s disease, oncology, rare diseases, and sleep apnea. For more information on Shionogi & Co., Ltd., please visit https://www.shionogi.com/global/en. Forward-Looking Statements This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise. *Literature search conducted in May 2026 1 XOCOVA (ensitrelvir) Prescribing Information. Available from: https://www.shionogi.com/content/dam/shionogi/si/products/pdf/xocova.pdf. Accessed July 6, 2026. 2 Hayden FG, Shinkai M, et al. Ensitrelvir for COVID-19 postexposure prophylaxis in household contacts. N Engl J Med. 2026;394(19):1905-1915. doi:10.1056/NEJMoa2509306. 3 Centers for Disease Control and Prevention [Internet]. 2026 COVID-19 Summer Outlook. 2026. Available from: https://www-cdc-gov.libproxy1.nus.edu.sg/cfa-qualitative-assessments/php/data-research/2026covid-19outlook.html 4 Kawashima S, Matsui Y, et al. Ensitrelvir is effective against SARS-CoV-2 3CL protease mutants circulating globally. Biochem Biophys Res Commun. 2023;645:132-136. doi:10.1016/j.bbrc.2023.01.040. 5 Kuroda T, Nobori H, et al. Efficacy comparison of 3CL protease inhibitors ensitrelvir and nirmatrelvir against SARS-CoV-2 in vitro and in vivo. J Antimicrob Chemother. 2023;78(4):946-952. doi:10.1093/jac/dkad027. 6 Donnelly M [Internet]. Respiratory risk report: week ending in July 4, 2026. Biobot Analytics; 2026. Available from: https://biobotanalytics.substack.com/p/respiratory-risk-report-663 7 Tanaka M, Ruiz C, et al. SARS-CoV-2 transmission dynamics in households with children, Los Angeles, California. Front Pediatr. 2021;9:752993. doi:10.3389/fped.2021.752993. 8 Centers for Disease Control and Prevention [Internet]. Preliminary estimates of COVID-19 burden for 2024-2025. 2026. Available from: https://www-cdc-gov.libproxy1.nus.edu.sg/covid/php/surveillance/burden-estimates.html 9 Centers for Disease Control and Prevention [Internet]. Long COVID basics. 2026. Available from: https://www-cdc-gov.libproxy1.nus.edu.sg/long-covid/about/index.html. 10 Xie Y, Xu E, et al. Long-term cardiovascular outcomes of COVID-19. Nat Med. 2022;28(3):583-590. doi:10.1038/s41591-022-01689-3. 11 Xie Y, Xu E, et al. Risks of mental health outcomes in people with COVID-19: a cohort study. BMJ. 2022;376:e068993. doi:10.1136/bmj-2021-068993. 12 Srivastava S, Garg I. Post-COVID-19 infection: long-term effects on liver and kidneys. World J Meta-Anal. 2021;9(3):220-233. doi:10.13105/wjma.v9.i3.220. 13 Tenforde MW, Kim SS, et al. Symptom duration and risk factors for delayed return to usual health among outpatients with COVID-19 in a multistate health care systems network— United States, March-June 2020. MMWR Morb Mortal Wkly Rep. 2020;69(30):993-998. doi:10.15585/mmwr.mm6930e1. 14 Graham EL, Clark JR, et al. Persistent neurologic symptoms and cognitive dysfunction in non-hospitalized COVID-19 “long haulers.” Ann Clin Transl Neurol. 2021;8(5):1073-1085. doi:10.1002/acn3.51350. 15 Kwok WC, Chau CH, et al. Outcomes among patients with chronic obstructive pulmonary disease after recovery from COVID-19 infection of different severity. Sci Rep. 2024;14(1):13881. doi:10.1038/s41598-024-64670-9. 16 Conway SE, Healy BC, et al. COVID-19 severity is associated with worsened neurological outcomes in multiple sclerosis and related disorders. Mult Scler Relat Disord. 2022;63:103946. doi:10.1016/j.msard.2022.103946. 17 Chen IW, Chang LC, et al. Association between COVID-19 and the development of chronic kidney disease in patients without initial acute kidney injury. Sci Rep. 2025;15(1):10924. doi:10.1038/s41598-025-96032-4. 18 Sax PE, Draica F, et al. New onset conditions, exacerbations and worsening of underlying conditions following COVID-19 infection [poster presentation]. Presented at: Conference on Retroviruses and Opportunistic Infections (CROI); 2026; Denver, CO, United States. 19 Gravenstein S, Cooke C, et al. Current impact of COVID-19 in long-term care facilities in the United States: a systematic literature review [poster presentation]. Presented at: Conference on Retroviruses and Opportunistic Infections (CROI); 2026; Denver, CO, United States. 20 Hayden FG, Shinkai M, et al. Ensitrelvir for COVID-19 postexposure prophylaxis in household contacts. Supplementary Appendix. N Engl J Med. 2026;394(19). Available from: https://www-nejm-org.libproxy1.nus.edu.sg/doi/suppl/10.1056/NEJMoa2509306/suppl_file/nejmoa2509306_appendix.pdf.

Phase 3Clinical ResultDrug Approval

05 Jun 2026

·firstwordpharma.com

Biopharma bites: White House reportedly mulling Sharpless to lead FDA, plus Fulcrum's layoffs and more from InnoCare, Chiesi

Ned Sharpless on Trump admin's shortlist to lead FDA: reportFulcrum cuts 85% of workforceInnoCare's BTK inhibitor clears mid-stage lupus trialEU approves paediatric label for Chiesi's LoxujtaNed Sharpless is on Trump admin's shortlist to lead FDA: reportThe Trump administration is reportedly considering Ned Sharpless to serve as the permanent FDA commissioner, according to CNN, which cited four people familiar with the matter. He would replace Kyle Diamantas, who stepped in as interim chief following Marty Makary's resignation in May. Sharpless, who previously led the National Cancer Institute and filled in as acting FDA head for seven months under President Donald Trump's first term, is one of a "handful" of outside candidates who are being vetted for the job — including Richard Pops, who is set to retire from his 35-year role as CEO of Alkermes — though the search process is still ongoing. Since leaving the FDA, Sharpless helped found Jupiter Bioventures, a biotech venture foundry armed with $70 million to create eight to 10 oncology-focused startups. He also, alongside several former FDA leaders, penned a letter last year condemning changes to US vaccine policy proposed by Vinay Prasad, the former head of the agency's Center for Biologics Evaluation and Research.-Elizabeth EatonFulcrum cuts 85% of workforceA few days after ending work on its only clinical programme — the oral small molecule EED inhibitor pociredir for sickle cell disease — Fulcrum Therapeutics disclosed in a securities filing that it will cut 85% of its workforce, leaving it with just nine full-time employees.The restructuring plan was approved by the company's board on May 31 in an effort to reduce operating expenses and preserve capital. The workforce reduction was communicated on June 4 and is expected to be completed in the second quarter.As previously indicated, Fulcrum is undertaking a review of strategic alternatives, which may include a merger, acquisition or business combination. -Matt DennisInnoCare's BTK inhibitor clears mid-stage lupus trialUpbeat mid-stage data for InnoCare Pharma's BTK inhibitor orelabrutinib in systemic lupus erythematosus (SLE) were presented Friday at the European Alliance of Associations for Rheumatology (EULAR) congress.The Phase IIb trial randomised 187 patients with moderate-to-severe SLE to receive one of two daily doses of orelabrutinib or placebo. Results showed the higher-dose group achieved an SLE Response Index-4 (SRI-4) response rate of 57.1% versus 34.4% for placebo at week 48, meeting the study's primary endpoint. The higher-dose arm also met secondary goals for SRI-6 and British Isles Lupus Assessment Group–Based Composite Lupus Assessment (BICLA) response rates versus placebo. Additionally, 71.1% of patients receiving the higher dose had their steroid use down to ≤7.5 mg versus 43.6% of placebo recipients. Meanwhile, orelabrutinib was safe and well tolerated, with no new signals identified. The BTK inhibitor is already marketed for certain haematologic malignancies in China, Australia and Singapore under the name Hibruka. InnoCare is currently conducting a registrational Phase III trial of orelabrutinib for SLE, while investigating it for multiple sclerosis in partnership with Biogen.-Pavan KamatEU approves paediatric label for Chiesi's LoxujtaThe European Commission authorised a broader label for Chiesi's Loxujta (lomitapide) covering children aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH), in conjunction with diet and other lipid-lowering treatments, including LDL-apheresis where available. The microsomal triglyceride transfer protein (MTP) inhibitor was initially approved in the EU for adults with HoFH back in 2013.The expanded indication is supported by data from the Phase III APH-19 study. The trial met its primary endpoint, demonstrating a mean 53.5% reduction in LDL-C from baseline at week 24, with significant reductions also recorded across non-HDL-C, total cholesterol, VLDL-C, apolipoprotein B and triglycerides. Adverse events were predominantly mild and gastrointestinal or hepatic in nature; no new safety signals were identified.-Pavan Kamat

Clinical ResultPhase 3Drug ApprovalPhase 2Executive Change

100 Deals associated with Lomitapide Mesylate

External Link

KEGG	Wiki	ATC	Drug Bank
D09638	Lomitapide Mesylate	C10AX12	DB08827

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperlipidemia Type IIa	Mexico	University of Pennsylvania	09 Feb 2014
Homozygous familial hypercholesterolemia	United States	CHIESI Farmaceutici SpA	21 Dec 2012
Homozygous familial hypercholesterolemia	United States	Chiesi Ltd.	21 Dec 2012

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Familial chylomicronaemia syndrome	Phase 3	Italy	Amryt Pharma Holdings Ltd.	18 Dec 2018
Hypercholesterolemia, Familial	Phase 3	Italy	Amryt Pharma Holdings Ltd.	18 Dec 2018
Hyperlipidemias	Phase 2	United States	Aegerion Pharmaceuticals, Inc.	01 Oct 2007
Hypercholesterolemia	Phase 2	United States	Aegerion Pharmaceuticals, Inc.	01 May 2006
Colorectal Cancer	Preclinical	South Korea	Ersteq Co., Ltd.	08 Dec 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EUCTR2018-002911-80-IT (LOCHNES, Pubmed) Manual	Phase 3	Familial chylomicronaemia syndrome	18	lomitapide	rdjmbtyqrq(msbtbzjuid) = iamjrokheq xpbqmsljcu (aoxuweicim )	Positive	10 Sep 2022
NCT01915771 (CTgov)	Phase 1	-	15	Lomitapide (PK of Lomitapide)	bkurvwikmr(cfhsnyifyd) = lriljiwhrd muzufaywam (wzbdispvor, 0.556) View more	-	11 Mar 2020
				Lomitapide (PK of M1)	bkurvwikmr(cfhsnyifyd) = gmzfpxqdsl muzufaywam (wzbdispvor, 0.593) View more
NCT00943306 (CTgov)	Phase 3	Homozygous familial hypercholesterolemia	19	lomitapide	aefniwvknz(qzaytegvrp) = yawojvodlb opmhrinivu (teousiisiv, 31.35) View more	-	11 May 2018
NCT02044419 (CTgov)	Phase 1	-	32	Lomitapide (Lomitapide Sprinkled in Applesauce (Treatment A))	nuewququju(zlrcuknwhy) = vzbovyvuti bkhdjoibzt (zhhwnranir, 15.2) View more	-	13 Feb 2018
				Lomitapide (Lomitapide Sprinkled in Mashed Banana (Treatment B))	nuewququju(zlrcuknwhy) = wsinftopjv bkhdjoibzt (zhhwnranir, 13.4) View more
NCT00730236 (Phase 3, Pubmed \| Atherosclerosis)	Phase 3	Homozygous familial hypercholesterolemia	29	Lomitapide	wgotlsrtby(gswgxurbzv) = ivnhhuqday vwrxhybmnr (zdmfiyxczl )	Positive	01 Jun 2015
				lomitapide (Lipoprotein apheresis)	wgotlsrtby(gswgxurbzv) = cgfevfdokg vwrxhybmnr (zdmfiyxczl )
NCT00690443 (CTgov)	Phase 2	Hypercholesterolemia	44	Atorvastatin (Atorvastatin 20 mg)	tmetplmcme(eqiyvwxoon) = ihgmjyewpg xeaoaodxlg (fhzubbygqu, 14.35) View more	-	25 Feb 2013
				Atorvastatin+Lomitapide (Atorvastatin 20 mg + Lomitapide)	tmetplmcme(eqiyvwxoon) = mhxcytgynt xeaoaodxlg (fhzubbygqu, 26.78) View more
NCT01556906 (CTgov)	Phase 2	Homozygous familial hypercholesterolemia	6	Lomitapide	hmrhgebsnp(clwwwlteww) = xuegnxocxs bvworugxjb (bsjohrcytx, 9.311) View more	-	22 Feb 2013
NCT00730236 (Phase 3, CTgov)	Phase 3	Homozygous familial hypercholesterolemia	29	Lomitapide	rvcqweneqk(qoenchingr) = izkixtzfhg zmgsqpujlg (ndaijprwfm, 31.25) View more	-	22 Feb 2013
NCT00559962 (CTgov)	Phase 2	Hyperlipidemias	260	placebo (Placebo)	hezihayfyz(rllvoyktdt) = hhyupfuonm cmpqqxduqs (ycjhhffibz, 1.814) View more	-	22 Feb 2013
				AEGR-733 (AEGR-733 5 mg)	hezihayfyz(rllvoyktdt) = ucuwwstjcb cmpqqxduqs (ycjhhffibz, 6.297) View more
NCT00474240 (CTgov)	Phase 2	Hypercholesterolemia	157	Placebo (Placebo)	ggsntvemvf(ziofqwttqy) = nposezcxfl kizdvuopoi (uudqixivua, 11.2) View more	-	21 Feb 2013
				Atorvastatin (Atorvastatin 20 mg)	ggsntvemvf(ziofqwttqy) = abyovlcsgz kizdvuopoi (uudqixivua, 16.4) View more

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