FDA News Month in Review: December 2024

Published on: January 4, 2025

In this edition of HCPLive’s newsletter on monthly US Food and Drug Administration (FDA) news, we cover updates from across therapeutic pipelines in December.

Welcome to our recap of this month's news and updates from the US Food and Drug Administration (FDA)!

In our December 2024 recap, we spotlight 36 pieces of pipeline news, including 16 FDA actions and 20 announcements of other pipeline updates. Be sure to check for our recap of the therapies, companies, and individuals moving the needle in healthcare during the past month, delivered on the first Saturday of each new month!

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA Regulatory Actions

FDA Approves Ustekinumab (STELARA) Biosimilar for Crohn’s, UC, Psoriasis, PsA

On December 2, 2024, Biocon Biologics announced FDA approval of ustekinumab-kfce (YESINTEK) for the treatment of Crohn’s disease (CD), ulcerative colitis (UC), plaque psoriasis (PsO), and psoriatic arthritis (PsA). Based on clinical data demonstrating similarity to reference ustekinumab in pharmacokinetics, efficacy, safety, and tolerability, this approval allows Biocon to launch the biosimilar in February 2025 under a prior settlement agreement with Janssen. This milestone underscores Biocon’s commitment to providing affordable biosimilars for patients with inflammatory diseases.

FDA Authorizes Predetermined Change Control Plan for Sleep Wearable Dreem 3S

On December 3, 2024, Beacon Biosignals announced FDA authorization of a Predetermined Change Control Plan (PCCP) for the Dreem 3S, a wearable EEG headband for sleep monitoring. Based on this authorization, Beacon can update the device’s sleep staging algorithm without requiring new 510(k) submissions, expediting improvements. This marks a significant step in advancing machine learning applications in sleep monitoring and neurological care.

Cell Therapy Gets RMAT Designation for HLHS, Improves Survival in Heart Failure With Reduced Ejection Fraction

On December 4, 2024, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Mesoblast's rexlemestrocel-L (Revascor) stromal cell therapy for potential use in children with hypoplastic left heart syndrome (HLHS). This designation follows previous Rare Pediatric Disease and Orphan Drug Designations for the therapy. Clinical data from a Phase 1/2 trial showed that rexlemestrocel-L significantly improved left ventricular volumes in HLHS patients, and recent Phase 3 data from the DREAM-HF trial indicated improved survival and clinical outcomes in adults with heart failure with reduced ejection fraction (HFrEF).

FDA Approves Crinecerfont for Congenital Adrenal Hyperplasia

On December 13, 2024, Neurocrine Biosciences announced FDA approval of crinecerfont (Crenessity) for use with glucocorticoids in adults and children aged 4 years and older with congenital adrenal hyperplasia (CAH). Based on two randomized, placebo-controlled trials, crinecerfont demonstrated significant reductions in glucocorticoid doses while maintaining androstenedione control. This approval marks an important step in advancing treatment options for individuals with the rare genetic disorder classic CAH.

FDA Approves Nemolizumab for Individuals with Atopic Dermatitis

On December 14, 2024, the FDA approved nemolizumab (Nemluvio) for patients aged 12 and older with moderate-to-severe atopic dermatitis (AD) inadequately managed with topical therapies. Based on findings from the phase 3 ARCADIA trial, nemolizumab demonstrated significant improvements in lesion clearance, itch reduction, and sleep disturbance when combined with topical corticosteroids and/or calcineurin inhibitors. This marks nemolizumab as the first monoclonal antibody targeting IL-31 receptor alpha to address AD symptoms.

FDA Approves Tapinarof 1% Cream for Adult, Pediatric Patients with Atopic Dermatitis

On December 16, 2024, Organon announced FDA approval of tapinarof (Vtama) cream 1% for treating AD in patients aged 2 years and older. Supported by the ADORING trial program, the cream demonstrated significant improvements in skin clearance, itch reduction, and long treatment-free intervals in children and adults with mild-to-severe disease. Tapinarof offers a novel aryl hydrocarbon receptor agonist option for managing this widespread inflammatory skin condition.

FDA Adds Boxed Warning to Fezolinetant (Veozah) for Liver Injury Risk

On December 16, 2024, the FDA issued a Boxed Warning for fezolinetant (Veozah) following a reported case of serious drug-induced liver injury, highlighting the risk of "rare but serious" hepatic adverse effects. Patients are advised to discontinue the medication immediately if symptoms such as jaundice, dark urine, or abnormal stool appear, and healthcare providers must conduct baseline and regular liver function tests during treatment. Despite the warning, the FDA maintains that the overall benefit-risk profile of fezolinetant remains positive for treating moderate to severe vasomotor symptoms associated with menopause.

FDA Approves Seventh Ustekinumab Biosimilar

On December 17, 2024, the FDA approved Celltrion’s biosimilar ustekinumab (Steqeyma), for subcutaneous or intravenous use in treating PsO and active PsA in adults and children, as well as CD and UC in adults. Approval was supported by a Phase 3 trial demonstrating comparable efficacy, safety, and immunogenicity to the reference product ustekinumab, with PASI score improvements in moderate to severe plaque psoriasis aligning between both treatments. Set to launch in February 2025, Steqeyma becomes the seventh FDA-approved ustekinumab biosimilar, broadening options for managing chronic inflammatory conditions.

FDA Issues CRL to Glepaglutide for Short Bowel Syndrome, Cites Insufficient Evidence

On December 19, 2024, the FDA issued a Complete Response Letter (CRL) to Zealand Pharma for glepaglutide, a long-acting GLP-2 analog, citing insufficient evidence to establish the safety and efficacy of the to-be-marketed dose for treating short bowel syndrome (SBS) dependent on parenteral support (PS). The CRL recommends an additional Phase 3 trial, which Zealand plans to initiate in 2025 to support future regulatory submissions in the US, Europe, and other regions. While Phase 3 EASE trials demonstrated reductions in PS volume and some patients achieving complete weaning, the FDA requires further confirmatory evidence for approval.

Olezarsen Earns First-Ever FDA Approval for Familial Chylomicronemia Syndrome

On December 19, 2024, the FDA approved olezarsen (TRYNGOLZA), the first-ever therapy for adults with familial chylomicronemia syndrome (FCS), to reduce triglycerides as an adjunct to a low-fat diet. This RNA-targeted medicine, developed by Ionis Pharmaceuticals, demonstrated a significant reduction in triglycerides and acute pancreatitis events in the phase 3 BALANCE trial, where the 80 mg dose lowered triglycerides by 42.5% at 6 months and achieved a 57% mean reduction at 12 months. Approval marks a breakthrough for this rare genetic condition, previously managed solely with restrictive diets, offering patients a novel, targeted treatment option.

FDA Approves First Acellular Tissue Vessel Therapy for Extremity Vascular Trauma

On December 20, 2024, the FDA approved Humacyte’s acellular tissue engineered vessel (ATEV), branded as Symvess, for use in adults with extremity arterial injuries requiring urgent revascularization, particularly when autologous vein grafting is not feasible. Symvess is a vascular conduit made from human extracellular matrix proteins and human vascular smooth muscle cells, designed to restore blood flow in patients with severe vascular trauma. Approval was based on positive data from the Phase 2/3 V005 study, which demonstrated high rates of patency and low amputation rates, offering a significant advancement in trauma care.

FDA Issues CRL for Sotagliflozin in Type 1 Diabetes and CKD

On December 20, 2024, the FDA issued a Complete Response Letter (CRL) to Lexicon Pharmaceuticals for sotagliflozin (Zynquista) as a treatment for type 1 diabetes (T1D) with chronic kidney disease (CKD), following the FDA’s advisory committee vote that the risks outweighed the benefits. Sotagliflozin, which was previously approved for heart failure under the brand name Inpefa, has faced setbacks in the US, with its prior NDA for T1D also resulting in a CRL. Despite these challenges, Lexicon remains committed to advancing its clinical pipeline, with a focus on other candidates like LX9211 for diabetic neuropathic pain.

FDA Approves Tirzepatide for Obstructive Sleep Apnea in Obesity

On December 20, 2024, the FDA approved tirzepatide (Zepbound) as the first prescription treatment for adults with moderate-to-severe obstructive sleep apnea (OSA) and obesity. In the Phase 3 SURMOUNT-OSA trial, tirzepatide reduced breathing disruptions by up to 29 per hour, with up to 20% weight loss, and significantly improved OSA symptoms, including achieving remission or mild OSA in 42% of patients after one year. Eli Lilly and Company indicated the treatment's safety profile was similar to previous studies, with gastrointestinal events being the most common side effects.

FDA Approves Concizumab for Hemophilia A or B with Inhibitors

On December 20, 2024, the FDA approved concizumab-mtci (Alhemo; Novo Nordisk), a subcutaneous injection for once-daily prophylaxis in adults and children (≥12 years) with hemophilia A or B with inhibitors. The approval was based on the Phase 3 explorer7 trial, which demonstrated an 86% reduction in annual bleeding rate (ABR) in patients using concizumab compared to no prophylaxis. This treatment, which blocks tissue factor pathway inhibitor (TFPI) to enhance thrombin production, provides a significant new option for hemophilia patients with inhibitors, particularly in hemophilia B where prophylactic choices have been limited.

FDA Approves Generic Once-Daily GLP-1 RA for Type 2 Diabetes in Pediatric, Adult Patients

On December 23, 2024, the FDA approved the first once-daily generic GLP-1 receptor agonist, referencing liraglutide 18 mg/3 mL (Victoza), for improving glycemic control in adults and children (≥10 years) with type 2 diabetes (T2D). This approval follows the success of liraglutide in the LEAD program and its subsequent label expansions for cardiovascular risk reduction and pediatric use. The generic version, developed by Hikma Pharmaceuticals, offers a more affordable treatment option for diabetes management, with common side effects including nausea, diarrhea, and vomiting.

FDA Proposes Rule for Standardized Asbestos Testing in Talc-Containing Cosmetic Products

On December 26, 2024, the FDA proposed a rule to establish standardized testing methods for detecting and identifying asbestos in talc-containing cosmetic products, aiming to protect consumers from harmful exposure to asbestos. This proposed rule, part of the Modernization of Cosmetics Regulation Act of 2022, requires manufacturers to test for asbestos using Polarized Light Microscopy (PLM) and Transmission Electron Microscopy (TEM) or provide a certificate of analysis from their talc suppliers. If finalized, the rule would make products adulterated under the Federal Food, Drug, and Cosmetic Act if they do not comply with the testing and record-keeping requirements or if asbestos is detected in talc used in cosmetics.

Pipeline Updates

FDA Clears Initiation of Phase 2 Efficacy Trial for Ruxoprubart in IgAN

On December 2, 2024, the FDA cleared the initiation of a Phase 2 clinical trial for ruxoprubart, an investigational monoclonal antibody targeting the alternative complement pathway for treating Immunoglobulin A nephropathy (IgAN). Developed by NovelMed, ruxoprubart offers a selective approach, distinguishing itself from other complement blockers by targeting the alternative pathway rather than the classical one. This approval supports ongoing research into ruxoprubart’s potential for treating multiple chronic kidney conditions, including atypical hemolytic uremic syndrome (aHUS) and ANCA-associated vasculitis (AAV), with the company seeking further partners and investors to progress to Phase 3 trials.

New Phase 3 Data Suggest Twice-Daily Tofacitinib Etocomil Effective for Atopic Dermatitis

On December 2, 2024, Minghui Pharmaceutical reported positive findings from the Phase 3 trial of tofacitinib etocomil (MH004) ointment 1.0% for mild-to-moderate AD. The twice-daily topical Janus Kinase (JAK) inhibitor significantly outperformed placebo in reducing skin inflammation, with 58.2% of patients achieving EASI-75 by week 4 compared to 19.8% on placebo (P < .0001). The ointment demonstrated a favorable safety profile, with treatment-related adverse events being mild to moderate, and no severe drug-related events reported, suggesting its potential as a safe and effective option for managing AD.

Anaphylm Sublingual Film for Anaphylaxis Receives Positive FDA Feedback

On December 2, 2024, the FDA provided positive feedback to Aquestive Therapeutics regarding Anaphylm (epinephrine) sublingual film for severe allergic reactions, including anaphylaxis. This feedback supports the company’s plans for a New Drug Application (NDA) submission in the first quarter of 2025, with no additional adult clinical trials required. If approved, anaphylm, a polymer matrix-based epinephrine prodrug, offers a novel, easy-to-carry oral alternative to traditional epinephrine devices and has shown promising results in clinical trials, with rapid symptom resolution within minutes of administration.

SURMOUNT-5: Tirzepatide Bests Semaglutide in Head-to-Head Weight Loss Trial

On December 4, 2024, Eli Lilly and Company announced tirzepatide (Zepbound) outperformed semaglutide (Wegovy) for weight loss in the head-to-head SURMOUNT-5 trial, demonstrating a 20.2% reduction in body weight compared to 13.7% with semaglutide after 72 weeks. This represents a 47% greater relative weight loss for tirzepatide, with additional superiority across five secondary endpoints. The trial, which included 751 adults with obesity or overweight and comorbidities like hypertension or dyslipidemia, positions tirzepatide as a leading option in obesity treatment, especially as the only FDA-approved dual GIP and GLP-1 receptor agonist for weight management.

Bemnifosbuvir, Ruzasvir Regimen Meets Safety, SVR Primary Endpoints in Phase 2 HCV Trial

On December 4, 2024, Atea Pharmaceuticals announced promising results from its Phase 2 trial of an 8-week regimen combining bemnifosbuvir and ruzasvir for hepatitis C virus (HCV) treatment. The regimen achieved a 98% sustained virologic response (SVR12) in treatment-adherent patients, with no serious adverse events or treatment discontinuations. The study demonstrated robust efficacy, especially in non-cirrhotic patients, and the company plans to initiate a Phase 3 program after an End-of-Phase 2 meeting with the FDA, potentially positioning the regimen as a leading short-duration, pan-genotypic treatment option for HCV.

Iptacopan Boosts Hemoglobin Levels in Adults with PNH in Phase 3b Trial

On December 6, 2024, Novaris announced the Phase 3b APPULSE-PNH trial demonstrated that twice-daily oral iptacopan (Fabhalta) improved hemoglobin (Hb) levels in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) switched from anti-C5 therapies like eculizumab or ravulizumab. The 24-week study confirmed the safety profile of iptacopan, consistent with previous data, and further data is expected to be presented at a 2025 medical meeting. Iptacopan, an oral Factor B inhibitor of the alternative complement pathway, received FDA approval in 2023 and remains the only oral monotherapy available for PNH.

FDA Receives sBLA for Guselkumab Treatment of Psoriasis, Juvenile PsA in Adolescents

On December 2, 2024, Johnson & Johnson announced submission of 2 supplemental Biologics License Applications (sBLAs) to the FDA for guselkumab (Tremfya) to treat moderate-to-severe psoriasis in children aged 6 and older and active juvenile PsA (jPsA) in those aged 5 and older. Guselkumab, an IL-23 inhibitor targeting the p19 subunit, was previously FDA-approved for adult PsO and PsA. The pediatric psoriasis sBLA is supported by the Phase 3 PROTOSTAR study, while the jPsA application is based on pharmacokinetic extrapolation from DISCOVER-1 and -2 studies, along with data from PROTOSTAR.

Intra-Cellular Therapies Submits sNDA to FDA for Lumateperone as an MDD Adjunctive Therapy

On December 3, 2024, Intra-Cellular Therapies submitted a supplemental New Drug Application (sNDA) to the FDA for lumateperone (CAPLYTA) as an adjunctive therapy for Major Depressive Disorder (MDD). Supported by two positive Phase 3 trials, lumateperone showed significant reductions in symptoms of depression on the Montgomery-Asberg Depression Rating Scale (MADRS), with a 4.9-point and 4.5-point improvement over placebo (P < .0001). With a favorable safety profile, minimal metabolic effects, and low rates of extrapyramidal symptoms, CAPLYTA could potentially become a first-line adjunctive treatment for MDD, pending FDA approval.

FDA Finds Obeticholic Acid (Ocaliva) Linked to Serious Liver Injury in Noncirrhotic PBC

On December 12, 2024, the FDA issued a safety update regarding obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC), identifying serious liver injury risks in patients without cirrhosis. This follows a 2021 restriction against its use in patients with advanced cirrhosis due to similar risks. Postmarket data revealed a higher incidence of liver transplant and death among obeticholic acid users versus placebo, prompting the FDA to recommend frequent liver function monitoring and timely discontinuation for patients with worsening liver health.

Tobevibart, Elebsiran Receive FDA Breakthrough Therapy Designation for Chronic HDV

On December 12, 2024, Vir Biotechnology announced its investigational therapies, tobevibart and elebsiran, received FDA Breakthrough Therapy and European Medicines Agency (EMA) PRIME designations for treating chronic hepatitis delta (CHD). These designations were supported by data from the Phase 2 SOLSTICE trial, where the combination therapy achieved deep and sustained suppression of HDV RNA, with 100% of participants showing significant viral load reductions by week 24 and 80% achieving undetectable levels by week 60 in the rollover cohort. Both agents demonstrated favorable safety profiles, with no study-related discontinuations, and the Phase 3 ECLIPSE program is set to begin in early 2025.

Mifepristone Notably Reduces HbA1c in Treatment Phase of CATALYST Trial

On December 12, 2024, Corcept Therapeutics announced positive Phase 4 CATALYST trial results, demonstrating that mifepristone (Korlym) significantly improves HbA1c in patients with hypercortisolism and difficult-to-control T2D. The trial's treatment phase revealed a placebo-adjusted HbA1c reduction of 1.32% (P < .0001) over 24 weeks, showcasing mifepristone’s clinical benefit for this patient population. Hypercortisolism was found in approximately 25% of patients with difficult-to-control T2D, highlighting the need for expanded screening and treatment to improve outcomes.

FDA Receives sNDA for Roflumilast Cream Treatment of Children with Atopic Dermatitis

On December 16, 2024, Arcutis Biotherapeutics submitted a supplemental New Drug Application (sNDA) to the FDA for roflumilast (Zoryve) cream 0.05% as a once-daily treatment for mild-to-moderate AD in children aged 2–5 years. Backed by data from pivotal trials, including the Phase 3 INTEGUMENT-PED study, the submission highlights significant improvements in AD severity, with 25.4% of roflumilast-treated children achieving vIGA-AD Success at 4 weeks compared to 10.7% in the placebo group (P < .0001). The cream demonstrated rapid efficacy, noticeable improvements in itch relief, and a favorable safety profile aligned with data from older populations, offering a potential new standard of care for this pediatric group.

Duvakitug Meets Primary Endpoints in Phase 2b RELIEVE UCCD Study for IBD

On December 17, 2024, Teva Pharmaceuticals and Sanofi announced positive results from the Phase 2b RELIEVE UCCD trial evaluating duvakitug, a TL1A-targeting monoclonal antibody, in patients with moderate-to-severe UC and CD. The study met its primary endpoints, with 47.8% of patients receiving a high dose of duvakitug achieving clinical remission in UC and endoscopic response in CD at week 14, representing the strongest outcomes reported for a TL1A-targeted therapy (P = .003 for UC, P < .001 for CD). Duvakitug was well tolerated, with no new safety signals, and regulatory discussions are planned to support Phase 3 trials in inflammatory bowel disease (IBD).

FDA Accepts New Drug Application for TNX-102 SL in Fibromyalgia

On December 17, 2024, Tonix Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for fibromyalgia treatment, with a PDUFA target action date to be assigned soon. TNX-102 SL, a non-opioid analgesic targeting non-restorative sleep and acting on multiple neuroreceptor subtypes, demonstrated significant pain reduction in two Phase 3 trials, RELIEF (P = .010) and RESILIENT (P = .00005). Granted Fast Track designation, TNX-102 SL aims to address the unmet needs of over 10 million adults in the U.S. with fibromyalgia, offering a novel, non-addictive treatment option.

Phase 2 Data on ESO-101 Suggests Efficacy Among Adults with Eosinophilic Esophagitis

On December 17, 2024, EsoCap announced new findings from the Phase 2 ACESO trial, highlighting ESO-101 as a promising treatment for active eosinophilic esophagitis (EoE). ESO-101, a mucoadhesive film delivery system for the corticosteroid mometasone furoate, achieved significant reductions in eosinophil counts and improved endoscopic outcomes in adults with EoE. With 48% of participants achieving histological remission and a favorable safety profile, ESO-101 shows potential to address the unmet needs in EoE management, particularly with its targeted drug delivery and excellent patient compliance.

J&J Submits sBLA for Golimumab (Simponi) for Pediatric Ulcerative Colitis

On December 16, 2024, Johnson & Johnson submitted a supplemental Biologics License Application (sBLA) to the FDA seeking expanded approval of golimumab (Simponi) for children ≥ 2 years old with moderately to severely active UC. The sBLA is supported by data from the PURSUIT program, including Phase 1 and Phase 3 studies, demonstrating the safety, efficacy, and pharmacokinetics of golimumab in pediatric patients. If approved, golimumab could offer a much-needed treatment option for children with UC, expanding its current indication for adults with the condition.

LIB Therapeutics Submits BLA for Lerodalcibep Targeting Reductions in LDL-C

On December 16, 2024, LIB Therapeutics Inc. submitted a Biologics License Application (BLA) to the FDA for lerodalcibep, a novel, adnectin-based PCSK9 inhibitor designed to reduce LDL-C in patients with atherosclerotic cardiovascular disease (ASCVD) and primary hyperlipidemia. Lerodalcibep, which offers a once-monthly, self-administered subcutaneous injection, has demonstrated significant LDL-C reductions in Phase 3 trials, including a 65% reduction in patients with heterozygous familial hypercholesterolemia (HeFH). The drug is positioned as a patient-friendly alternative to current PCSK9 inhibitors, with the potential for improved adherence due to its small volume and stability without refrigeration.

CagriSema Achieves 22.7% Weight Loss in Phase 3 REDEFINE 1 Trial

On December 20, 2024, Novo Nordisk announced topline results from the Phase 3 REDEFINE 1 trial showing CagriSema, a fixed-dose combination of cagrilintide 2.4 mg and semaglutide 2.4 mg, significantly outperformed placebo in weight loss, achieving a 22.7% reduction after 68 weeks. CagriSema demonstrated superior efficacy compared to its individual components, with 40.4% of patients achieving a ≥25% weight loss, and was generally well tolerated, with gastrointestinal adverse events being the most common.

Belapectin Misses Primary Endpoint in Phase 3 MASH Cirrhosis, Portal Hypertension Trial

On December 20, 2024, Galectin Therapeutics announced the Phase 2b/3 NAVIGATE trial of belapectin for metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis and portal hypertension failed to meet its composite primary endpoint for preventing varices. While the 2 mg/kg dose of belapectin reduced varices incidence by 43.2% in the intent-to-treat population, this difference was not statistically significant. However, in the per-protocol population, a 48.9% reduction in new varices was observed with the 2 mg/kg dose, and the drug was well tolerated with no severe adverse events. Galectin plans to analyze additional data in early 2025 for potential next steps.

Deucravacitinib Meets ACR20 Endpoint for Psoriatic Arthritis in Phase 3 Trial

On December 23, 2024, Bristol Myers Squibb announced two pivotal Phase 3 trials, POETYK PsA-1 and POETYK PsA-2, demonstrated the superiority of deucravacitinib (Sotyktu) to placebo in achieving an American College of Rheumatology 20 (ACR20) response after 16 weeks in patients with PsA. The trials met their primary endpoints, with deucravacitinib also showing efficacy in secondary PsA disease activity measures. The drug's safety profile through 16 weeks was consistent with previous studies, and long-term data from PsO trials indicated stable adverse event rates over three years of use, supporting its potential as an oral treatment for PsA.