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Last update 15 Jul 2025

Efavirenz

Last update 15 Jul 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(S)-6-chloro-4-cyclopropylethynyl-4-trifluoromethyl-1,4-dihydro-benzo[d][1,3]oxazin-2-one, 6-chloro-4-(2-cyclopropyl-1-ethynyl)-4-trifluoromethyl-(4S)-1,4-dihydro-2H-benzo[d][1,3]oxazin-2-one, Efavirenz (JAN/USP/INN)

+ [18]

Target

Action

inhibitors

Mechanism

RT inhibitors(Viral reverse transcriptase inhibitors)

Therapeutic Areas

Immune System DiseasesInfectious DiseasesUrogenital Diseases

Active Indication

HIV Infections

Inactive Indication

Acquired Immunodeficiency SyndromeAIDS-Related Opportunistic InfectionsContraception+ [5]

Originator Organization

Bristol Myers Squibb Co.

Active Organization

Teva Pharmaceuticals Europe BVMerck Sharp & Dohme (Australia) Pty Ltd.Merck Sharp & Dohme BV

+ [3]

Inactive Organization

Abbott LaboratoriesBristol-Myers Squibb Pharma EEIG

Bristol Myers Squibb Co.

+ [2]

License Organization-

Drug Highest PhaseApproved

First Approval Date

United States (17 Sep 1998),

HIV Infections

RegulationAccelerated Approval (United States), Orphan Drug (Japan)

Structure/Sequence

Molecular FormulaC14H9ClF3NO2

InChIKeyXPOQHMRABVBWPR-ZDUSSCGKSA-N

CAS Registry154598-52-4

288

Clinical Trials associated with Efavirenz

NCT06914869

/ CompletedPhase 1

A Phase I, Randomized, Open-label, Drug-drug Interaction Study to Assess the Effect of Multiple Doses of a Moderate and a Strong CYP3A Inducer on the Pharmacokinetics and Safety of Mavorixafor in Healthy Male and Female Participants

The main purpose of this study is to evaluate drug-drug interaction (DDI) of orally administered mavorixafor with cytochrome P3A (CYP3A) inducers carbamazepine (a strong CYP3A inducer) or efavirenz (a moderate CYP3A inducer) in healthy male and female participants.

Start Date18 Feb 2025

Sponsor / Collaborator

X4 Pharmaceuticals, Inc.

NCT06797674

/ Active, not recruitingPhase 1

A Clinical Study to Evaluate the Effect of Multiple Doses of Efavirenz on Single-Dose Pharmacokinetics of MK-7602 in Healthy Participants

The goal of the study is to learn what happens to levels of MK-7602 in a healthy person's body over time. Researchers will compare what happens to MK-7602 in the body when it is given with or without another medicine called efavirenz, and when it is given with or without a meal.

Start Date10 Feb 2025

Sponsor / Collaborator

Merck Sharp & Dohme LLC

NCT06823596

/ RecruitingNot ApplicableIIT

Short-term Addition of Efavirenz to Induce CARD8-mediated Reduction of Persistent Nonsuppressible HIV Viremia in People with High Adherence to ART.

Antiretroviral therapy or ART blocks HIV replication reducing plasma viral loads to undetectable levels but has no effect on persistently infected cells in the body, called the virus reservoir. These cells carry infectious HIV capable of restarting HIV replication if therapy is stopped. The reservoir is so stable forcing people to adhere life-long ART. Over 5% of ART adherent individuals continue to have residual non-suppressive viremia (NSV) detected by clinical assays (40-400 copies/ml). Residual viremia reflects a more persistent reservoir and has the potential for increased morbidity. For eg., persistent expression of HIV proteins contributes to inflammation, and can lead to comorbidities. Recently, a novel way to target this reservoir called "TACK" or "Targeted activator of cell killing" is proposed. TACK compounds only target HIV infected cells and directly kill them by inducing a natural killing program (called the inflammasome). Recently the HIV drug, Efavirenz (EFV), which was used to suppress HIV replication for decades, has now been shown to also be a TACK compound. This pilot study will evaluate the impact of Efavirenz (EFV) in reducing HIV persistence by its ability to be a TACK molecule. So in addition to blocking HIV growth, this compound when added to a current ART regimen can kill HIV infected cells in the test tube. We aim to harness this effect to determine whether the addition of EFV to the current ART regimen in people with NSV can suppress the viremia to undetectable levels by killing those cells. NSV represents the "the tip of the iceberg" of those with bigger reservoirs and represents a challenging clinical scenario in dire need of new diagnostic and therapeutic options.
This pilot study will spark larger clinical trials to advance HIV cure strategies, and will provide new tools to improve the clinical management of people living with HIV.

Start Date14 Jan 2025

Sponsor / Collaborator

University of Toronto

[+1]

100 Clinical Results associated with Efavirenz

100 Translational Medicine associated with Efavirenz

100 Patents (Medical) associated with Efavirenz

9,177

Literatures (Medical) associated with Efavirenz

31 Dec 2025·HIV Research & Clinical Practice

A combined prospective and retrospective comparative study evaluating renal outcomes after switching from TDF + FTC + EFV to TDF/3TC/DTG (TLD) vs. DTG + 3TC in virologically suppressed Thai people with HIV

Article

Author: Putcharoen, Opass ; Kanogtorn, Sathaporn ; Burimat, Phattarapon ; Patamatamkul, Samadhi

BACKGROUND:

TDF/3TC/DTG (TLD) has been adopted as the first-line therapy for all people with HIV according to the WHO 2019 and Thai HIV guidelines. As a result, people with HIV in Thailand on TDF/FTC/EFV are switched to TLD. However, increasing data demonstrate the efficacy and renal safety of the TDF-sparing dual therapy with DTG + 3TC compared with TDF-based combination ART (cART) as a switching therapy. A direct comparison of estimated glomerular filtration rates (eGFR) among people with HIV treated with TDF-based regimens who switch to TLD vs. DTG + 3TC is yet to be made.

METHODS:

We enrolled virologically suppressed people with HIV aged ≥18 years currently on TDF + FTC + EFV to either switch to TLD or DTG + 3TC at two tertiary care hospitals. The switching regimen was chosen at the physicians' discretion. The primary outcome was the change in eGFR, calculated by creatinine at 24 weeks. Secondary outcomes included changes in LDL, body weight, and BMI at 24 weeks.

RESULTS:

Among 53 recruited participants, 28 and 15 completed the second follow-up in the TLD and DTG + 3TC groups, respectively. The mean age was higher in the TLD group compared to the DTG + 3TC group (Table 1). The median time from HIV diagnosis to switching was 8.5 years. The eGFR reduction was significantly greater in the TLD group than in the DTG + 3TC group: -17.24 ± 9.24 vs. -8.4 ± 9.03 mL/min/1.73 m² (p = 0.004). All participants in both groups achieved virological suppression. There was no significant change in CD4 counts between the two groups. Switching to DTG + 3TC was associated with a significantly smaller decline in eGFR post-switch (mean difference: 6.216 mL/min/1.73 m²; 95% CI 0.169-12.263; p = 0.044) compared to those who switched to TLD.

CONCLUSIONS:

There was a significant reduction in eGFR among people with HIV who switched to TLD compared to those switched to DTG + 3TC. Changes in LDL and BMI were comparable between groups. Dual therapy with DTG + 3TC may be a preferred switching option over TLD for individuals with renal safety concerns. Further randomized prospective trials with longer follow-up are warranted to confirm these findings.

01 Oct 2025·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Discovery of novel diarypyrimidine derivatives as potent HIV-1 NNRTIs by exploiting the tolerant region I within NNRTI binding pocket

Article

Author: De Clercq, Erik ; Kang, Dongwei ; Jiang, Xiangyi ; Gao, Zhen ; Liu, Xinyong ; Gao, Heng ; Pannecouque, Christophe ; Zhan, Peng ; Ji, Xiangkai

Non-nucleoside reverse transcriptase inhibitors (NNRTIs) serve as an essential component of antiretroviral treatment regimens, but they encounter challenges such as resistance mutations, poor solubility, and adverse effects. This study presents the design, synthesis, and biological evaluation of novel diarylpyrimidine (DAPY)-typed derivatives targeting the tolerant region I within the NNRTI binding pocket (NNIBP). We introduced aliphatic ring structures with high Fsp³ value, aiming to enhance target affinity while simultaneously improving water solubility. Among them, compound 9k demonstrated excellent potency against wild-type HIV-1 (EC₅₀ = 0.0014 μM) and key single-mutant strains K103 N (EC₅₀ = 0.0041 μM) and E138K (EC₅₀ = 0.0077 μM), which outperformed the positive drugs ETR, NVP and EFV. Molecular modeling studies were conducted to elucidate the detailed interactions between RT and 9k, providing insights into the enhanced drug resistance profiles. Additionally, 9k demonstrated a notable improvement in water solubility (1.38 μg/mL, pH = 7) compared to ETR (≪ 1 μg/mL, pH = 7). These findings provide a foundation for further optimization of NNRTIs with improved anti-resistance profiles and pharmacokinetics.

01 Sep 2025·INTERNATIONAL JOURNAL OF INFECTIOUS DISEASES

Switching to bictegravir/emtricitabine/tenofovir alafenamide from efavirenz/emtricitabine/tenofovir disoproxil in virologically suppressed people with HIV: findings from a non-randomized clinical trial (EBONY study)

Article

Author: Gagliardini, Roberta ; Fusto, Marisa ; Mastrorosa, Ilaria ; Ottou, Sandrine ; Bellagamba, Rita ; Antinori, Andrea ; Tempestilli, Massimo ; Lanini, Simone ; Paulicelli, Jessica ; Duca, Giulia Del ; De Zottis, Federico ; Pinnetti, Carmela ; Plazzi, Maria Maddalena ; Mondi, Annalisa ; Grilli, Elisabetta ; Vergori, Alessandra ; Mazzotta, Valentina ; Camici, Marta

OBJECTIVES:

No previous studies specifically explored the switch from efavirenz to bictegravir (BIC)-containing three-drug antiretroviral regimens. This study aimed to evaluate the efficacy and safety outcomes of a treatment switch from efavirenz/emtricitabine/tenofovir disoproxil fumarate (EFV/FTC/TDF) given once daily (OD) or on alternate days (ATAD) to BIC/emtricitabine/tenofovir alafenamide (BIC/FTC/TAF) in virologically suppressed people with HIV (PWH).

METHODS:

A pilot, single-arm, prospective study was conducted.

RESULTS:

Overall, 234 PWH were enrolled. 217 of 234 (92.7%, 95% confidence interval [CI], 88.6-95.7%) participants had HIV-RNA <40 cp/ml at 48 weeks. Virological failure occurred in three participants, none with documented resistance, and all resuppressed without antiretroviral therapy change. After 48 weeks, a slight increase in cluster of differentiation (CD)4 cell count was observed from the baseline (+ 59 cells/mmc, 95% CI, 31; 86, P <0.001), but not in CD4/CD8 ratio. A slight increase in creatinine (mean change +0.11 mg/dl, 95% CI 0.10; 0.13, P <0.001) and a decrease in total cholesterol (mean change -8 mg/dl, 95% CI -14; -3, P = 0.001) were also observed.

CONCLUSIONS:

Our data showed that BIC/FTC/TAF demonstrated high virologic and immunologic efficacy and an excellent safety profile.

News (Medical) associated with Efavirenz

08 Jul 2025

·www.merck.com

Merck to Present New Data Highlighting Research Advancements Across its HIV Prevention and Treatment Pipeline at IAS 2025

July 8, 2025 6:45 am ET RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that new data from its research pipeline for HIV prevention and treatment will be presented at the 13th International AIDS Society Conference on HIV Science (IAS 2025) taking place July 13-17, 2025, in Kigali, Rwanda. Merck will share new scientific findings from its HIV clinical development programs, including Phase 2 data on the safety and pharmacokinetics of MK-8527, an investigational, novel nucleoside reverse transcriptase translocation inhibitor (NRTTI), dosed orally once monthly, in development for the prevention of HIV as pre-exposure prophylaxis (PrEP). HIV clinical data presented at IAS 2025 from three Phase 3 trials (P051: NCT05631093; P052: NCT05630755; P054: NCT05766501) will examine the impact of preexisting resistance-associated mutations (RAMs) in proviral DNA on the virologic response to doravirine/islatravir (DOR/ISL), with a primary focus on M184I/V in proviral DNA. Data will also be presented on the clinical development program for a once-weekly oral combination of islatravir and ulonivirine (ISL/ULO) for the treatment of adults with HIV-1 infection. Data from two Phase 1 trials evaluating the safety and tolerability of weekly ulonivirine and drug interactions between islatravir and ulonivirine dosed weekly in adults without HIV and a Phase 2b dose-ranging study utilizing a higher (20mg) dose of islatravir and 3 doses of ulonivirine in treatment naive adults living with HIV-1 will be presented. Based on results from these trials and pharmacokinetic modeling that will also be presented, a once-weekly oral combination of islatravir (2mg) and ulonivirine (200mg) (MK-8591B) is moving forward in clinical development. “As we continue to advance our HIV research, we are excited to present data from across our HIV pipeline at IAS 2025, including new data from our nucleoside reverse transcriptase translocation inhibitors development programs,” said Dr. Elizabeth Rhee, vice president, global clinical development, Merck Research Laboratories. “With daily, weekly and monthly oral regimens in development, we aim to offer choices that can help address the evolving needs of individuals living with or impacted by HIV.” Select abstracts in the IAS 2025 program include: Abstract Title and Author Date HIV PREVENTION Safety and Pharmacokinetics of Once-Monthly MK-8527: a Phase 2 Study in Adults at Low Risk of HIV-1 Exposure, Mayer, K, et al. Late-Breaker Oral Presentation Wednesday, July 16, 2025 (15:00 – 16:00 CAT) HIV TREATMENT Switching to Doravirine/Islatravir (100mg/0.25mg) Once Daily Maintained Viral Suppression in the Presence of Archived M184I/V Resistance-Associated Mutations in Proviral DNA, Diamond, T, et al. Poster Session Wednesday, July 16, 2025 (12:15 – 13:15 CAT) A Double-Blind, Active-Controlled, Phase 2b Study to Evaluate the Efficacy and Safety of Ulonivirine in Combination with Islatravir in Virologically Suppressed Adults Living With HIV-1, Molina, J.M., et al. (*) Oral Presentation ART Strategies Tuesday, July 15, 2025 (10:45 – 11:45 CAT) A Double-Blind Placebo-Controlled, Phase 1 Study to Evaluate Extended Multiple Dosing of Ulonivirine (MK-8507) in Adults Without HIV, Nussbaum, J, et al. Virtual Poster An Open-Label Phase 1 Study to Evaluate Drug Interactions Between Multiple Weekly Doses of Ulonivirine (MK-8507) and Single Doses of Islatravir in Adults Without HIV, Nussbaum, J, et al. Virtual Poster Phase 2 Once-Weekly Dose Optimization for Ulonivirine (MK-8507) in Combination with Islatravir (2mg), Pham, M, et al. Virtual Poster (*) Previous ulonivirine development program utilizing a higher (20mg) dose of islatravir. This year at IAS, Merck will host a policy symposium “Harnessing the Private Sector to Deliver HIV Prevention” on Tuesday, July 15, from 18:00 – 19:30 CAT. Merck will also host a medical symposium “Let’s Talk About It – Practical Case Discussions” on Wednesday, July 16, from 12:15 – 13:15 CAT, which will cover critical considerations when treating HIV today, as well as a deep dive into the DOR/ISL resistance profile based on Phase 3 data. Both events will be open to all registered attendees, virtually and in-person. Separately, Merck will be hosting a virtual investor event where scientific and commercial leaders will give an overview of the company’s advancing research pipeline in HIV treatment and prevention on Thursday, July 17, at 9:00AM ET. Investors, analysts, members of the media, and the general public are invited to listen to a webcast of the presentation at this weblink. For more details about Merck’s clinical development program in HIV treatment and prevention, click here. Merck’s Commitment to HIV For more than 35 years, Merck has been committed to scientific research and discovery in HIV leading to scientific breakthroughs that have helped change HIV treatment. Our work has helped pioneer the development of new options across multiple drug classes to help those impacted by HIV. Today, we are developing a series of antiviral options designed to help people manage their HIV and to help prevent HIV, with the goal of reducing the growing burden of infection worldwide. We want to ensure people are not defined by HIV, and our work focuses on transformational innovations, collaborations with others in the global HIV community, and access initiatives aimed at helping to end the HIV epidemic for everyone. Indications and usage for PIFELTRO® (doravirine) and DELSTRIGO® (doravirine, lamivudine, and tenofovir disoproxil fumarate) in the U.S. PIFELTRO is indicated in combination with other antiretroviral (ARV) agents for the treatment of HIV-1 infection in adult patients with no prior ARV treatment history or to replace the current ARV regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable ARV regimen with no history of treatment failure and no known substitutions associated with resistance to doravirine. DELSTRIGO is indicated as a complete regimen for the treatment of HIV-1 infection in adult patients with no prior ARV treatment history or to replace the current ARV regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable ARV regimen with no history of treatment failure and no known substitutions associated with resistance to the individual components of DELSTRIGO. Selected Safety Information Warning: Posttreatment Acute Exacerbation of Hepatitis B Virus (HBV) for DELSTRIGO All patients with HIV-1 should be tested for the presence of HBV before initiating ARV therapy. Severe acute exacerbations of HBV have been reported in people with concomitant HIV-1 and HBV who have discontinued products containing lamivudine or tenofovir disoproxil fumarate (TDF), which are components of DELSTRIGO. Patients coinfected with HIV-1 and HBV who discontinue DELSTRIGO should be monitored with both clinical and laboratory follow-up for at least several months after stopping DELSTRIGO. If appropriate, initiation of anti-HBV therapy may be warranted. Contraindications PIFELTRO and DELSTRIGO are contraindicated when coadministered with drugs that are strong cytochrome P450 (CYP)3A enzyme inducers (including the anticonvulsants carbamazepine, oxcarbazepine, phenobarbital, and phenytoin; the androgen receptor inhibitor enzalutamide; the antimycobacterials rifampin and rifapentine; the cytotoxic agent mitotane; and the herbal product St. John’s wort (Hypericum perforatum)), as significant decreases in doravirine plasma concentrations may occur, which may decrease the effectiveness of DELSTRIGO and PIFELTRO. DELSTRIGO is contraindicated in patients with a previous hypersensitivity reaction to lamivudine. Warnings and Precautions Severe Skin Reactions Severe skin reactions, including Stevens-Johnson syndrome (SJS)/toxic epidermal necrolysis (TEN), have been reported during the postmarketing experience with doravirine-containing regimens. Discontinue PIFELTRO or DELSTRIGO, and other medications known to be associated with severe skin reactions, immediately if a painful rash with mucosal involvement or a progressive severe rash develops. Clinical status should be closely monitored, and appropriate therapy should be initiated. New or Worsening Renal Impairment Renal impairment, including cases of acute renal failure and Fanconi syndrome, have been reported with the use of TDF. DELSTRIGO should be avoided with concurrent or recent use of a nephrotoxic agent (eg, high-dose or multiple NSAIDs). Cases of acute renal failure after initiation of high-dose or multiple NSAIDs have been reported in people living with HIV with risk factors for renal dysfunction who appeared stable on TDF. Prior to or when initiating DELSTRIGO, and during treatment, assess serum creatinine, estimated creatinine clearance, urine glucose, and urine protein in all patients. In patients with chronic kidney disease, also assess serum phosphorus. Discontinue DELSTRIGO in patients who develop clinically significant decreases in renal function or evidence of Fanconi syndrome. Discontinue DELSTRIGO if estimated creatinine clearance declines below 50 mL/min. Bone Loss and Mineralization Defects In clinical trials in adults living with HIV, TDF was associated with slightly greater decreases in bone mineral density (BMD) and increases in biochemical markers of bone metabolism. Serum parathyroid hormone levels and 1,25 Vitamin D levels were also higher. Cases of osteomalacia associated with proximal renal tubulopathy have been reported with the use of TDF. The effects of TDF-associated changes in BMD and biochemical markers on long-term bone health and future fracture risk in adults are unknown. Immune Reconstitution Syndrome Immune reconstitution syndrome can occur, including the occurrence of autoimmune disorders with variable time to onset, which may necessitate further evaluation and treatment. Drug Interactions Because DELSTRIGO is a complete regimen, coadministration with other antiretroviral medications for the treatment of HIV-1 infection is not recommended. Coadministration of PIFELTRO with efavirenz, etravirine, or nevirapine is not recommended. If DELSTRIGO is coadministered with rifabutin, take one tablet of DELSTRIGO once daily, followed by one tablet of doravirine (PIFELTRO) approximately 12 hours after the dose of DELSTRIGO. If PIFELTRO is coadministered with rifabutin, increase PIFELTRO dosage to one tablet twice daily (approximately 12 hours apart). Consult the full Prescribing Information prior to and during treatment for more information on potential drug-drug interactions. Dosage and Administration/Specific Populations Renal Impairment Because DELSTRIGO is a fixed-dose combination tablet and the dosage of lamivudine and TDF cannot be adjusted, DELSTRIGO is not recommended in patients with estimated creatinine clearance less than 50 mL/min. Adverse Reactions The most common adverse reactions with DELSTRIGO (incidence ≥5%, all intensities) were dizziness (7%), nausea (5%), and abnormal dreams (5%). The most common adverse reactions with PIFELTRO (incidence ≥5%, all intensities) were nausea (7%), dizziness (7%), headache (6%), fatigue (6%), diarrhea (6%), abdominal pain (5%), and abnormal dreams (5%). By week 96 in DRIVE-FORWARD, 2% of adult participants in the PIFELTRO group and 3% in the darunavir+ritonavir (DRV+r) group had adverse events leading to discontinuation of study medication. By week 96 in DRIVE-AHEAD, 3% of adult participants in the DELSTRIGO group and 7% in the efavirenz (EFV)/emtricitabine (FTC)/TDF group had adverse events leading to discontinuation of study medication. In DRIVE-FORWARD, mean changes from baseline at week 48 in LDL-cholesterol (LDL-C) and non-HDL-cholesterol (non-HDL-C) were pre-specified. LDL-C: -4.6 mg/dL in the PIFELTRO group vs 9.5 mg/dL in the DRV+r group. Non-HDL-C: -5.4 mg/dL in the PIFELTRO group vs 13.7 mg/dL in the DRV+r group. The clinical benefits of these findings have not been demonstrated. In DRIVE-AHEAD, mean changes from baseline at week 48 in LDL-C and non-HDL-C were pre-specified. LDL-C: -2.1 mg/dL in the DELSTRIGO group vs 8.3 mg/dL in the EFV/FTC/TDF group. Non-HDL-C: -4.1 mg/dL in the DELSTRIGO group vs 12.7 mg/dL in the EFV/FTC/TDF group. The clinical benefits of these findings have not been demonstrated. In DRIVE-SHIFT, mean changes from baseline at week 24 in LDL-C and non-HDL-C were pre-specified. LDL-C: -16.3 mg/dL in the DELSTRIGO group vs -2.6 mg/dL in the PI + ritonavir group. Non-HDL-C: -24.8 mg/dL in the DELSTRIGO group vs -2.1 mg/dL in the PI + ritonavir group. The clinical benefits of these findings have not been demonstrated. In DRIVE-AHEAD, neuropsychiatric adverse events were reported in the three pre-specified categories of sleep disorders and disturbances, dizziness, and altered sensorium. Twelve percent of adult participants in the DELSTRIGO group and 26% in the EFV/FTC/TDF group reported neuropsychiatric adverse events of sleep disorders and disturbances; 9% in the DELSTRIGO group and 37% in the EFV/FTC/TDF group reported dizziness; and 4% in the DELSTRIGO group and 8% in the EFV/FTC/TDF group reported altered sensorium. The safety of DELSTRIGO in virologically-suppressed adults was based on week 48 data from participants in the DRIVE-SHIFT trial. Overall, the safety profile in virologically-suppressed adult participants was similar to that in participants with no ARV treatment history. Serum ALT and AST Elevations: In the DRIVE-SHIFT trial, 22% and 16% of participants in the immediate switch group experienced ALT and AST elevations greater than 1.25 X ULN, respectively, through 48 weeks on DELSTRIGO. For these ALT and AST elevations, no apparent patterns with regard to time to onset relative to switch were observed. One percent of participants had ALT or AST elevations greater than 5 X ULN through 48 weeks on DELSTRIGO. The ALT and AST elevations were generally asymptomatic, and not associated with bilirubin elevations. In comparison, 4% and 4% of participants in the delayed switch group experienced ALT and AST elevations of greater than 1.25 X ULN through 24 weeks on their baseline regimen. Pregnancy/Breastfeeding There is a pregnancy exposure registry that monitors pregnancy outcomes in individuals exposed to PIFELTRO or DELSTRIGO during pregnancy. Healthcare providers are encouraged to register patients by calling the Antiretroviral Pregnancy Registry (APR) at 1-800-258-4263. Inform individuals with HIV-1 infection of the potential risks of breastfeeding, including: (1) HIV-1 transmission (in HIV-1–negative infants), (2) developing viral resistance (in HIV-1–positive infants), and (3) serious adverse reactions in a breastfed infant similar to those seen in adults. About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov). Please see Prescribing Information for PIFELTRO (doravirine) at: https://www.merck.com/product/usa/pi_circulars/p/pifeltro/pifeltro_pi.pdf and Patient Information for PIFELTRO at: https://www.merck.com/product/usa/pi_circulars/p/pifeltro/pifeltro_ppi.pdf Please see Prescribing Information for DELSTRIGO (doravirine, lamivudine, and tenofovir disoproxil fumarate) at: https://www.merck.com/product/usa/pi_circulars/d/delstrigo/delstrigo_pi.pdf and Patient Information for DELSTRIGO at: https://www.merck.com/product/usa/pi_circulars/d/delstrigo/delstrigo_ppi.pdf Media Contacts: Julie Cunningham, (617) 519-6264 Deb Wambold, (215) 779-2234 Investor Contacts: Peter Dannenbaum, (732) 594-1579 Steven Graziano, (732) 594-1583

Phase 2Clinical ResultPhase 1Phase 3

07 Jul 2025

·ir.kalvista.com

KalVista Pharmaceuticals Announces FDA Approval of EKTERLY® (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema

First new on-demand HAE treatment in over a decade, with potential to transform management of the disease Data demonstrated rapid symptom relief and attack resolution regardless of attack severity, location, age, or use of long-term prophylaxis, and well-established safety profile Management to host conference call today at 8:30 a.m. ET CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--Jul. 7, 2025-- KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the U.S. Food and Drug Administration (FDA) has approved EKTERLY® (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. EKTERLY is the first and only oral on-demand treatment for HAE. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250702871458/en/ EKTERLY Logo “The FDA approval of EKTERLY is a defining moment for people living with HAE,” said Ben Palleiko, CEO of KalVista. “EKTERLY enables people to treat attacks the moment symptoms begin, wherever they are. This approval affirms the strength of our science and deep commitment to the HAE community. I am profoundly grateful to the KalVista team for their dedication and perseverance, and to the patients and healthcare providers, as well as the HAEA and HAEi, for making this possible. EKTERLY has the potential to become the foundational treatment for HAE and our focus now is on delivering it to the people who need it.” “As the first orally administered on-demand therapy for HAE attacks, EKTERLY provides patients and physicians with an important and welcome advance in HAE treatment options,” said Anthony J. Castaldo, chief executive officer of the U.S. Hereditary Angioedema Association. Prior to EKTERLY’s approval, all on-demand HAE treatment options approved in the U.S. required intravenous or subcutaneous administration, which carries a significant treatment burden.1 Even with the use of long-term prophylaxis as a preventative therapy, most people living with HAE continue to have unpredictable attacks and require ready access to on-demand medication.1 “This is an important moment for patients, giving people living with HAE a treatment option that could provide greater independence and control over managing their condition,” said Marc A. Riedl, MD, Professor of Medicine and Clinical Director, U.S. Hereditary Angioedema Association Center at the University of California, San Diego, and an investigator for the KONFIDENT phase 3 trial. “Until now, on-demand treatment relied on injectable subcutaneous or intravenous administration, often resulting in delayed intervention. Having an oral option empowers patients to treat attacks early, which aligns with treatment guidelines and advances our goal as physicians to reduce the overall burden of disease.” The efficacy and safety of EKTERLY was established by the results from KalVista’s phase 3 KONFIDENT clinical trial, which was the largest clinical trial program ever conducted in HAE. Data from KONFIDENT was published in the New England Journal of Medicine in May 2024, showing that EKTERLY achieved significantly faster symptom relief, reduction in attack severity and attack resolution than placebo, and was well-tolerated with a safety profile similar to placebo.2 The trial randomized 136 HAE patients from 66 clinical sites across 20 countries. These results were further supported by the more real-world KONFIDENT-S open-label extension trial, which as of September 2024, showed that EKTERLY enabled patients to treat attacks in a median of 10 minutes following onset. The most recent data from KONFIDENT-S shows that beginning of symptom relief occurred in a median of 1.3 hours among attacks involving the larynx, the abdomen, and for breakthrough attacks among patients receiving long-term prophylaxis. The safety profile of EKTERLY 600 mg in KONFIDENT-S, in a much larger number of attacks (>1700), was consistent with that observed in KONFIDENT. KalVista will launch EKTERLY in the U.S. immediately and physicians can begin writing prescriptions today. As part of the Company’s commitment to supporting patients, KalVista has established KalVista Cares™, a comprehensive patient support program that offers personalized services and resources for eligible individuals. This includes assistance with navigating insurance coverage, access support, and ongoing help throughout the treatment journey. For more information, visit EKTERLY.com. Conference Call and Webcast KalVista will host a live conference call and webcast to discuss the FDA approval of EKTERLY today, July 7, 2025, at 8:30 a.m. ET. The webcast will be accessible through the Investors section of the Company’s website at ir.kalvista.com. A replay will be available shortly after the conclusion of the live event. About Hereditary Angioedema Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity. About EKTERLY® (sebetralstat) EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, is the first and only oral on-demand therapy approved by the U.S. FDA for the treatment of acute attacks of hereditary angioedema (HAE) in people 12 years of age and older. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, EKTERLY has the potential to become the foundational therapy for HAE management worldwide. INDICATION AND IMPORTANT SAFETY INFORMATION What is EKTERLY® (sebetralstat)? EKTERLY is a prescription medicine used to treat sudden (acute) attacks of hereditary angioedema (HAE) in adults and children aged 12 years of age and older. It is not known if EKTERLY is safe and effective in children under 12 years of age. IMPORTANT SAFETY INFORMATION Before taking EKTERLY, tell your healthcare provider about all of your medical conditions, including if you: Are pregnant or planning to become pregnant. It is not known if EKTERLY can harm your unborn baby. Are breastfeeding or plan to breastfeed. It is not known if EKTERLY passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby while taking EKTERLY. Have liver problems. Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking EKTERLY with certain other medicines can cause side effects or affect how well EKTERLY or the other medicines work. Especially tell your healthcare provider if you take any of the following, as their use with EKTERLY is not recommended: itraconazole, phenytoin, efavirenz. Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine. What are the possible side effects of EKTERLY? The most common side effects of EKTERLY include headache. For more information, ask your healthcare provider or pharmacist. Talk to your doctor for medical advice about side effects. You are encouraged to report side effects related to KalVista products by calling 1-855-258-4782. If you prefer, you may contact the U.S. Food and Drug Administration (FDA) directly. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Please click here for full Prescribing Information, including Patient Information. About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. In the U.S., KalVista markets EKTERLY®, the first and only oral on-demand treatment for hereditary angioedema (HAE). The Company has multiple regulatory applications under review in key global markets. For more information about KalVista, please visit www.kalvista.com and follow us on LinkedIn, X, Facebook and Instagram. Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, our expectations about the safety and efficacy of sebetralstat and our other product candidates, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2024, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. 1Busse PJ, et al. J Allergy Clin Immunol Pract. 2021;9(1):132–150.e3. doi:10.1016/j.jaip.2020.08.046. 2 Riedl MA, et al. Oral sebetralstat for on-demand treatment of hereditary angioedema attacks. N Engl J Med. 2024;391(1):32–43. View source version on businesswire.com: https://www.businesswire.com/news/home/20250702871458/en/ Ryan Baker Head, Investor Relations (617) 771-5001 ryan.baker@kalvista.com Molly Cameron Director, Corporate Communications (978) 339-3378 molly.cameron@kalvista.com Source: KalVista Pharmaceuticals, Inc.

Phase 3Drug ApprovalClinical Result

24 Jun 2025

·www.prnewswire.com

BioMarin Presents Five-Year Phase 3 Results Reinforcing Long-Term Efficacy and Safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) at International Society on Thrombosis and Haemostasis 2025 Congress

Data from longest and largest hemophilia gene therapy study demonstrates sustained factor VIII expression and bleed control, with 81.3% of individuals remaining off prophylaxis No new safety signals observed SAN RAFAEL, Calif., June 24, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data underscoring the long-term efficacy and safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) were presented at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington, D.C., June 21-25, 2025. The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained five years after treatment with ROCTAVIAN. FVIII activity remained consistent with previously reported results, and no new safety signals were observed. Across the entire trial, no participants developed FVIII inhibitors or experienced thromboembolic events, and there were no treatment-related malignancies across the five years of the study. "I am impressed that the strong efficacy and safety results for ROCTAVIAN are sustained five years after individuals with severe hemophilia A received gene therapy, with only one adult returning to prophylaxis since the last data cut," said Andrew Leavitt, M.D., director of the University of California San Francisco's program for noncancerous blood disorders and Adult Hemophilia Treatment Center. "These results highlight the role that gene therapy may play in addressing the burdens of chronic disease management in people living with hemophilia A, while maintaining excellent bleed control." Among the 134 individuals who received ROCTAVIAN in the study, the rollover population of 112 patients from the 270-902 noninterventional lead-in study had baseline annualized bleeding rate (ABR) data prospectively collected during a period of at least six months while on routine FVIII prophylaxis prior to receiving ROCTAVIAN, and two of the 112 patients discontinued the study prior to year five. After five years, FVIII activity was nearly stable compared to year four, with mean FVIII activity in the mild hemophilia range (one-stage assay = 24.0 IU/dL; chromogenic assay = 13.7 IU/dL); 73.5% of participants had FVIII levels in the mild hemophilia to normal range. The mean ABR for treated bleeds for the rollover population was 0.6 bleeds/year after year five. During year five, 77.8% of the remaining participants (n=108) in the rollover population had zero treated bleeds. At the end of year five, 81.3% (n=109) of participants remained off prophylaxis. Additionally, clinically relevant changes in health-related quality of life measures were observed over five years. "We hope these results demonstrating the long-term durability of gene therapy equip individuals with severe hemophilia A with the knowledge to make informed decisions regarding treatment," said Dawn Rotellini, Chief Operating Officer at the National Bleeding Disorders Foundation. "It is meaningful for the bleeding disorders community to see a continued commitment to highlighting the benefits of hemophilia A gene therapy." Additional data presented at the meeting described a framework for the measurement of the "hemophilia-free mind" in people who have received a ROCTAVIAN infusion, which represents an easing of the physical and psychological burden of hemophilia. Researchers leveraged clinical data as well as questionnaires to assess a number of quality of life outcomes from baseline to week 104. The analysis showed that people who received ROCTAVIAN in the Phase 3 GENEr8-1 study experienced improvements across all measures, including bleeding risk, joint pain, injection schedule, physical activity, travel, employment and education, and impact on family members and caregivers. "These five-year data reinforce our confidence in ROCTAVIAN's profile, showing that people living with severe hemophilia A can experience long-term bleed control without the need for prophylaxis, meaningfully impacting the mental and clinical burdens of this condition," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "We remain committed to supporting the medical community and individuals living with severe hemophilia A in the clinical adoption of this gene therapy so they can experience the benefits that this treatment can offer." Below are key presentations for BioMarin and ROCTAVIAN at ISTH (all times Eastern Daylight Time): Efficacy, Safety and Quality of Life 5 Years After Valoctocogene Roxaparvovec Gene Transfer Poster PB0804 Monday, June 23, 2025, 1:45 – 2:45 p.m. Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Hemophilia A Poster PB0822 Monday, June 23, 2025, 1:45 – 2:45 p.m. Impact of Valoctocogene Roxaparvovec on Hemophilia-Free Mind: A Visual Multidimensional Method Poster PB0838 Monday, June 23, 2025, 1:45 – 2:45 p.m. Mechanisms of AAV Vector Persistence Following Infant Gene Therapy in Severe Hemophilia A Dogs Oral OC 51.1 Tuesday, June 24, 2025, 9:30 – 9:45 a.m. Steady Musculoskeletal Health After Gene Therapy in Severe Hemophilia A: 5-Year Results Oral OC 69.1 Wednesday, June 25, 2025, 10:15 – 10:30 a.m. About Hemophilia A Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is an X-linked genetic condition caused by missing or defective FVIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About ROCTAVIAN ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5 (AAV5), which is determined by a blood test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis. The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The U.S. Food and Drug Administration (FDA) approved ROCTAVIAN on June 29, 2023. More information on testing to determine eligibility to receive ROCTAVIAN can be found at in the U.S. and in Germany. U.S. Important Safety Information ROCTAVIAN U.S. Important Safety Information Contraindications: Patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). Patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis, and patients with known hypersensitivity to mannitol. Infusion-related reactions including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during and for at least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN in a setting where personnel and equipment are immediately available to treat infusion-related reactions. Discontinue infusion for anaphylaxis. Hepatotoxicity: The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Perform liver health assessments prior to administration. The majority of patients treated with ROCTAVIAN experienced ALT elevations and required corticosteroids for ALT elevation. Assess patient's ability to receive corticosteroids and/or other immunosuppressive therapy that may be required for an extended period. Live vaccines should not be administered to patients while on immunosuppressive therapy. Monitor ALT weekly for at least 26 weeks and as clinically indicated, during corticosteroid therapy and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Monitor factor VIII activity levels since ALT elevation may be accompanied by a decrease in factor VIII activity. One case of autoimmune hepatitis was reported during third year follow-up in a patient with history of hepatitis C and steatohepatitis. It is recommended that patients abstain from consuming alcohol for at least 1 year after administration and thereafter limit alcohol use. Concomitant medications may cause hepatotoxicity, decrease factor VIII activity, or change plasma corticosteroid levels which may impact liver enzyme elevation and/or factor VIII activity or decrease the efficacy of the corticosteroid regimen or increase their side effects. Closely monitor concomitant medication use including herbal products and nutritional supplements and consider alternative medications in case of potential drug interactions. Thromboembolic events: Factor VIII activity above ULN has been reported following ROCTAVIAN infusion. Thromboembolic events may occur in the setting of elevated factor VIII activity above ULN. Evaluate patients for risk of thrombosis including general cardiovascular risk factors before and after administration of ROCTAVIAN. Advise patients on their individual risk of thrombosis in relation to their factor VIII activity levels above ULN and consider prophylactic anticoagulation. Advise patients to seek immediate medical attention for signs or symptoms indicative of a thrombotic event. Factor VIII inhibitors and Monitoring for inhibitors: The safety and effectiveness of ROCTAVIAN in patients with prior or active factor VIII inhibitors have not been established. Patients with active factor VIII inhibitors should not take ROCTAVIAN. Following administration, monitor patients for factor VIII inhibitors (neutralizing antibodies to factor VIII). Test for factor VIII inhibitors especially if bleeding is not controlled, or plasma factor VIII activity levels decrease. Monitor Factor VIII using the same schedule for ALT monitoring. It may take several weeks after ROCTAVIAN infusion before ROCTAVIAN-derived factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Exogenous factor VIII or other hemostatic products may also be required in case of surgery, invasive procedures, trauma, or bleeds. Consider more frequent monitoring in patients with factor VIII activity levels ≤ 5 IU/dL and evidence of bleeding, taking into account the stability of factor VIII levels since the previous measurement. Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with one-stage clotting assays compared to chromogenic substrate assays. When switching from hemostatic products prior to ROCTAVIAN treatment, physicians should refer to the relevant prescribing information to avoid the potential for factor VIII activity assay interference during the transition period. Malignancy: The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age) with regular liver ultrasound (e.g., annually) and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration. In the event that any malignancy occurs after treatment with ROCTAVIAN, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100. Most Common Adverse Reactions: Most common adverse reactions (incidence ≥ 5%) were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain. Most common laboratory abnormalities (incidence ≥ 10%) were ALT, AST, LDH, CPK, factor VIII activity levels, GGT and bilirubin > ULN. Patients also experienced adverse reactions from corticosteroid use. Isotretinoin, Efavirenz, and HIV Positive Patients: Isotretinoin is not recommended in patients who are benefiting from ROCTAVIAN. Efavirenz is not recommended in patients treated with ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection. Females and Males of Reproductive Potential: ROCTAVIAN is not intended for administration in women. There are no data on the use of ROCTAVIAN in pregnant women or regarding lactation. For 6 months after administration of ROCTAVIAN, men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception, and men must not donate semen. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to BioMarin at 1-866-906-6100. Please see the ROCTAVIAN full Prescribing Information for additional Important Safety Information. About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data presented at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), including the oral and poster presentations; ROCTAVIAN's efficacy, safety and benefits to individuals with hemophilia A, including health-related quality of life; and BioMarin's commitment to supporting the medical community and individuals living with severe hemophilia A in the clinical adoption of ROCTAVIAN. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of ROCTAVIAN; results and timing of current and planned pre-clinical studies and clinical trials of ROCTAVIAN; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission, and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® and ROCTAVIAN® are registered trademarks of BioMarin Pharmaceutical Inc. SOURCE BioMarin Pharmaceutical Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultDrug ApprovalGene Therapy

100 Deals associated with Efavirenz

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D00896	Efavirenz	J05AG03	DB00625

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Indication	Country/Location	Organization	Date
HIV Infections	United States	Bristol Myers Squibb Co.	17 Sep 1998

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Indication	Highest Phase	Country/Location	Organization	Date
Pulmonary Tuberculosis	Phase 3	Brazil	Merck Sharp & Dohme Corp.	11 Sep 2015
Pulmonary Tuberculosis	Phase 3	Côte d'Ivoire	Merck Sharp & Dohme Corp.	11 Sep 2015
Pulmonary Tuberculosis	Phase 3	France	Merck Sharp & Dohme Corp.	11 Sep 2015
Pulmonary Tuberculosis	Phase 3	Mozambique	Merck Sharp & Dohme Corp.	11 Sep 2015
Pulmonary Tuberculosis	Phase 3	Vietnam	Merck Sharp & Dohme Corp.	11 Sep 2015
Tuberculosis	Phase 3	Brazil	Merck Sharp & Dohme Corp.	11 Sep 2015
Tuberculosis	Phase 3	Côte d'Ivoire	Merck Sharp & Dohme Corp.	11 Sep 2015
Tuberculosis	Phase 3	France	Merck Sharp & Dohme Corp.	11 Sep 2015
Tuberculosis	Phase 3	Mozambique	Merck Sharp & Dohme Corp.	11 Sep 2015
Tuberculosis	Phase 3	Vietnam	Merck Sharp & Dohme Corp.	11 Sep 2015

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02777229 (NAMSAL-ANRS-12313, Pubmed \| Open Forum Infect Dis)	Phase 3	HIV Infections First line	613	Dolutegravir (DTG) 50mg with Tenofovir disoproxil fumarate and Lamivudine (TDF/3TC)	mjxpxzjjpe(jzjfccvsnb) = zlibkgyeeq rsdiwlwtgz (njzpypiogr ) View more	Positive	01 Dec 2023
				Efavirenz 400 mg (EFV400) with Tenofovir disoproxil fumarate and Lamivudine (TDF/3TC)	mjxpxzjjpe(jzjfccvsnb) = ntcqvmfxoe rsdiwlwtgz (njzpypiogr ) View more
NCT02777229 (NAMSAL ANRS 12313, Pubmed)	Phase 3	First line	613	Dolutegravir 50 mg (DTG)	ibhqmfmxto(huvukkmgff) = decreased from 7.2 to 3.0 zpbujsmqhs (zwourvmvoj ) View more	Positive	01 Nov 2023
				Low-dose efavirenz (EFV400)
NCT02777229 (NAMSAL ANRS 12313, Pubmed \| J Acquir Immune Defic Syndr)	Phase 3	HIV Infections First line	613	Dolutegravir 50 mg (DTG)	rekawdqzkg(nmrvoyfkei) = mpsvmjroph sljyubegof (sgsoqtcbpp ) View more	Positive	01 Nov 2023
				Low-dose Efavirenz (EFV400)	rekawdqzkg(nmrvoyfkei) = obovthwzgf sljyubegof (sgsoqtcbpp ) View more
NCT00964002 (FAVE, CTgov)	Phase 2	Castration-Resistant Prostatic Cancer \| Metastatic Prostate Carcinoma	61	efavirenz	vajzjtwpra = xgchrsoiks gizfjkvyjt (wtvhbsejou, hjwxkeczth - jiwfwkwunv) View more	-	16 May 2022
IAS2022	Not Applicable	Acquired Immunodeficiency Syndrome	92	Efavirenz-based regimens	xipkidhdmi(vtrqsceaja) = tkmlcwhbcu kavjhwwigo (ilvidywsmr ) View more	-	01 Jan 2022
				Dolutegravir-based ART	xipkidhdmi(vtrqsceaja) = nmemixnsxl kavjhwwigo (ilvidywsmr ) View more
NCT02273765 (Pubmed) Manual	Phase 3	Tuberculosis \| HIV Infections	460	Tenofovir Disoproxil Fumarate+lamivudine+raltegravir	rkqfmopuzv(rxirydcubc) = etgtieldko ocwvtkxujd (oiobbvtarp ) View more	Negative	01 Jun 2021
				Tenofovir Disoproxil Fumarate+lamivudine+efavirenz	rkqfmopuzv(rxirydcubc) = gktmjnwugi ocwvtkxujd (oiobbvtarp ) View more
NCT01878890 (ESCALE, CTgov)	Phase 1	Solid tumor \| Non-Hodgkin Lymphoma	25	Efavirenz 600mg (Efavirenz: 600 mg)	llpewvnnir = blyjkwgtes bhbedxroon (uuqweaqgud, cddkzgornw - bgscfjgvko) View more	-	27 Jan 2021
				Efavirenz 1200 mg (Efavirenz: 1200 mg)	llpewvnnir = mxxnwtthxl bhbedxroon (uuqweaqgud, gblpmlsjpy - pyxfmjcxae) View more
NCT04596488 (Pubmed \| BMC Infect Dis)	Not Applicable	HIV Infections	20	EFV 400 mg	ehtvpwncnx(oueqeuozfq) = Dizziness was the most common event, occurring in 70% of patients within the first 2 weeks of treatment. mgrqryrurx (qbhwtriree )	-	23 Jan 2021
NCT02777229 (NAMSAL, Pubmed \| Lancet HIV)	Phase 3	HIV Infections	820	Dolutegravir 50 mg	qvpmsgcnxa(jlcldsjnoq) = llommdvxgn asfhdtxjyy (irtgglbbtl )	Positive	01 Oct 2020
				Efavirenz 400 mg	qvpmsgcnxa(jlcldsjnoq) = hqlxmwbeoq asfhdtxjyy (irtgglbbtl )
EASD2020	Not Applicable	-	-	Palmitate	vtrwpuudnv(ovbtbqrgtz) = ouorijxroi qzqhijcigz (afbjhsxtju, 0.07)	Negative	24 Sep 2020
				Efavirenz	vtrwpuudnv(ovbtbqrgtz) = msvzpdyykx qzqhijcigz (afbjhsxtju, 0.5)

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