Trending Topics & Drug Approvals: December 2025 - Prime Therapeutics
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Trending Topics & Drug Approvals: December 2025
Your source for the latest drug information highlights
December 1, 2025
Trending Topics
Hot topics
The U.S. Food and Drug Administration (FDA) has initiated steps for removal of the Boxed Warnings for cardiovascular (CV) diseases, breast cancer and probable dementia from hormone replacement therapy (HRT), also referred to as menopausal hormone therapies (MHT). These medications are used to provide relief from vasomotor symptoms (VMS) (e.g., hot flashes, night sweats) of menopause and genitourinary syndrome of menopause (GSM) (also known as vulvovaginal atrophy); certain agents are also used to prevent osteoporosis. In 2003, based on results from the Women’s Health Initiative (WHI), the FDA initiated class-wide labeling changes for estrogen plus progestogen and estrogen-alone products for these uses. Labeling updates eventually included a Boxed Warning for an increased risk of serious adverse events, including CV disorders, invasive breast cancer and probable dementia. The FDA has reconsidered the benefits and risks of these therapies; the updated label recommendations are based on a comprehensive evaluation of literature since publication of the WHI studies, drug utilization review data and public feedback. Notably, the WHI trial participants had an average age of 63 years. However, women with bothersome VMS are an average of 45 to 55 years of age. In addition to further analyses and long-term follow-up from the WHI, the Agency also held an Expert Panel meeting in July 2025 followed by a public comment period. Due to the reconsideration of the benefit to risk, the FDA has requested label changes to the prescribing information of HRT.
Language in labeling for all (systemic and local vaginal) HRT products will be updated to:
Language in labeling for all (systemic and local vaginal) HRT products will be updated to:
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Remove references to CV diseases, breast cancer and probable dementia in the boxed warning
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Remove language related to endometrial cancer except in the systemic estrogen-alone products in the boxed warning (the boxed warning for endometrial cancer for systemic estrogen-alone products will remain)
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Remove language regarding the recommendation to use the lowest effective dose for the shortest amount of time from the boxed warning
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Remove the probable dementia warning throughout the labeling
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The Agency is investigating postmarketing reports of neutralizing antibodies to ADAMTS13, including one fatality in a patient treated with Adzynma (ADAMTS13, recombinant-krhn) for congenital thrombotic thrombocytopenic purpura. The FDA states the fatality occurred in a pediatric patient and appears to be related to Adzynma. The patient exhibited progressive neurologic symptoms and neutralizing antibodies to ADAMTS13 were found approximately 10 months following initiation of prophylactic treatment with Adzynma. Assays currently do not distinguish between antibodies to recombinant ADAMTS13 and endogenous ADAMTS13. The FDA is assessing the need for regulatory action related to these events.
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The FDA has released new draft guidance to accelerate biosimilar development by simplifying biosimilarity studies and reducing unnecessary clinical testing. The draft guidance, “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies,” reduces the need for manufacturers to perform comparative human clinical efficacy studies which generally have low sensitivity compared to other analytical techniques. Instead, analytical testing will be utilized for demonstration of product differences. The intent of the new draft guidance is to improve efficiency and reduce costs required to develop biosimilars. A separate initiative is underway to improve the process for the development of interchangeable biosimilars.
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The drug shortages report for calendar year 2024, submitted annually to Congress, has been published by the FDA. During 2024, FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) collaborated with manufacturers to prevent 283 drug shortages using several approaches, including regulatory flexibility/discretion and expedited reviews/inspections. During this same timeframe, 15 new drug shortages were identified (lowest in a decade), which is substantially less than the peak of 251 new drug shortages during calendar year 2011. Ongoing drug shortages that have not been resolved from prior years are also reported (113 tracked shortages as of Dec. 31, 2024).
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The FDA has published internal filing checklists used by CDER staff to evaluate if a submitted new drug application (NDA) or biologics license application (BLA) is complete and ready for review. The checklists have been released to increase transparency and reduce the number of filing deficiencies, which can delay the approval of therapies and require additional Agency resources. Notably, the FDA states that these checklists may not be comprehensive, and that the Agency is solely responsible for determination of whether an application can be filed in accordance with legal and scientific standards.
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The Agency has announced a pilot program to enhance communication (“Meeting Minute Clarification Opportunity”) with drug developers after formal FDA meetings. The program launched in October through the Office of New Drugs and intends to aid in streamlining the drug development process by improving communication. Drug developers (also known as sponsors) can submit a question via email, and FDA staff generally will respond via email within three business days, providing clarification to meeting minutes.
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Actions to restrict the sale of unapproved ingestible prescription fluoride-containing drug products for children have been announced. The FDA has sent notices to four companies stating enforcement action will be taken against marketing of these products labeled for use in children less than three years of age, or older children at low or moderate risk of tooth decay. The Agency compiled and assessed scientific evidence and collaborated with parents, clinicians and federal partners through a public meeting and comment period. The FDA determined that these products should not be used by the aforementioned populations as fluoride may alter the gut microbiome. Furthermore, a Cochrane review concluded that fluoride did not decrease dental caries in baby teeth.
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Eighteen warning letters have been sent to owners of websites illegally marketing misbranded and unapproved botulinum toxin products. These products carry health risks, including symptoms of botulism. FDA-approved botulinum toxin products carry a Boxed Warning regarding the significant risk of serious or life-threatening side effects caused by muscle weakness leading to difficulty breathing. FDA-approved botulinum toxin products (e.g., Botox) are only available with a prescription from a licensed health care professional (HCP) and require administration by a licensed and trained HCP. Products acquired from unauthorized sources have the potential to be misbranded, adulterated, contaminated, counterfeit, and ineffective/unsafe.
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The Advisory Committee on Immunization Practices’ (ACIP) next meeting will be held on Dec. 4–5, 2025. Topics for discussion include the Centers for Disease Control and Prevention (CDC) vaccine risk monitoring evaluation, vaccine schedule history and considerations, childhood/adolescent immunization schedule, adjuvants/contaminants and hepatitis B vaccination. The CDC updated the “Autism and Vaccines” webpage to state that the claim “vaccines do not cause autism” is not an evidence-based claim as studies have not excluded the possibility that infant vaccines cause autism. The Agency states studies supporting an association have been ignored by health authorities, and a comprehensive assessment has been launched by HHS to evaluate plausible biologic mechanisms and potential causal associations. Leading medical, health and patient advocacy groups have issued a statement refuting this claim; the organizations state it is an outdated, disproven idea that vaccines cause autism.
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The FDA has approved new labeling for the Duchenne muscular dystrophy (DMD) gene therapy drug, delandistrogene moxeparvovec-rokl (Elevidys). In addition to a new Boxed Warning describing the risk of serious liver injury and acute liver failure, including fatalities, the indication is now limited to ambulatory patients with DMD who are ≥ 4 years of age with a confirmed mutation in the DMD gene. Elevidys is no longer indicated for non-ambulatory patients with DMD. A new Limitations of Use statement has also been added to assist in clinical decision-making. The label updates follow the June 2025 safety communication regarding two cases of fatal acute liver failure in non-ambulatory pediatric males with DMD after receiving Elevidys.
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A prespecified analysis of the SELECT trial (n=17,604) evaluating the association between adiposity and major adverse cardiovascular event (MACE) risk has been published in The Lancet. Cardioprotective effects of semaglutide were observed that were independent of baseline adiposity and weight loss. Patients aged ≥ 45 years with a body mass index (BMI) ≥ 27 kg/m² were randomized to once weekly subcutaneous (SC) semaglutide 2.4 mg or placebo. In the semaglutide arm, an average 4% reduction in risk of MACE was observed per 5 kg decrease in body weight (hazard ratio [HR], 0.96; 95% confidence interval [CI], 0.94 to 0.99; p=0.001) and per 5 cm smaller waist circumference (HR, 0.96; 95% CI, 0.93 to 0.99; p=0.004). In contrast, in the placebo group, only a lower baseline waist circumference demonstrated a statistically significant association with MACE reduction (HR, 0.96; 95% CI, 0.94 to 0.99; p=0.007), but body weight did not demonstrate this same association (HR, 0.99; 95% CI, 0.97 to 1.01; p=0.28). Furthermore, weight loss in the placebo group was paradoxically associated with increased MACE risk. Authors concluded that only an estimated 33% of MACE reduction was related to waist circumference suggesting that semaglutide’s cardioprotective effects are due to mechanisms beyond reducing adiposity.
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A population-based study was published in the Annals of Internal Medicine evaluating comparative gastrointestinal (GI) safety in adults with type 2 diabetes mellitus (T2DM) initiating dulaglutide, subcutaneous (SC) semaglutide and tirzepatide. Matched pairs were determined for three pairwise comparisons to assess the primary endpoint (composite of acute pancreatitis, biliary disease, bowel obstruction, gastroparesis and severe constipation) across these agents. The hazard ratio of GI events was 0.96 (95% CI, 0.87 to 1.06) in the semaglutide versus dulaglutide cohort (65,238 matched pairs), 0.96 (CI, 0.77 to 1.2) in the tirzepatide versus dulaglutide cohort (20,893 matched pairs) and 1.07 (CI, 0.9 to 1.26) in the tirzepatide versus semaglutide cohort (46,620 matched pairs), suggesting similar GI safety profiles. However, results could be potentially confounded by glycemic control and BMI.
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The FDA Adverse Event Reporting System (FAERS) has received an increased number of reports regarding allergic/hypersensitivity type reactions following infusion of specific lots of immune globulin intravenous (IV), human–slra (Asceniv) and immune globulin IV, human, 10% liquid (Bivigam). Although hypersensitivity and anaphylactic/anaphylactoid reactions are a potential risk with immune globulin products, the increased reports of reactions with these lots necessitate concern. Some patients have experienced serious adverse events requiring medical management and/or admission to the emergency room or hospital. As a result, the FDA is recommending use of these lots to cease immediately.
The American Academy of Pediatrics (AAP) has released a statement advising that acetaminophen (e.g., Tylenol) is safe for children when taken as directed and that studies have not found that acetaminophen causes autism. Furthermore, studies have not demonstrated significant or confirmed associations between use of acetaminophen during pregnancy and the child’s risk of autism, ADHD or intellectual disability. No single factor has been identified as the cause of autism; research has shown it is multifactorial and involves genetic as well as environmental factors.
The Institute for Clinical and Economic Review (ICER) has published its final evidence report on brensocatib (Brinsupri) for the treatment of non-cystic fibrosis bronchiectasis. The panel unanimously determined that current evidence is sufficient to demonstrate a net health benefit of brensocatib as an add-on therapy to usual care in comparison to usual care alone.
Drug Approvals
Specialty
Nov. 3, 2025 – doxecitine and doxribtimine (Kygevvi)
- 505(b)(2) New Drug Application (NDA) approval; Breakthrough Therapy, Orphan Drug, Priority Review, Rare Pediatric Disease; first FDA-approved treatment for thymidine kinase 2 deficiency (TK2d)
- Combination of two pyrimidine nucleosides
- Indicated for the treatment of TK2d in adults and pediatric patients with an age of symptom onset on or before 12 years
- Powder for oral solution: 2 g doxecitine and 2 g doxribtimine in a single use packet
- Recommended dosage (starting, intermediate, maintenance) is based on the patient’s body weight and is administered orally in three equally divided doses approximately six hours apart (+/- two hours) with food; titration to the next dosage level can occur after a minimum of two weeks at the current dosage level based on tolerability; product labeling provides details on the required number of packets and volume of water needed to reconstitute the powder from the packet(s); product requires preparation and administration by an adult with the ZX2000 administration kit (provided separately); can also be administered with a feeding tube; the prepared solution should be discarded 16 hours following reconstitution or after taking/giving the three daily doses, whichever occurs first
- Approval was based on data from one phase 2 clinical study, two retrospective chart review studies and an expanded access program; overall survival was compared for patients treated with Kygevvi or pyrimidine nucleosides (n=78) compared with matched untreated patients in an external control group (n=78) from published literature and one of the retrospective studies in this drug development program; at a median duration of treatment of four years (range, one day to 12 years), the overall risk of death from treatment start was decreased by 86% (95% confidence interval [CI], 61 to 96) with Kygevvi compared to matched untreated controls with 3.8% of patients in the treated arm passing away compared with 35.9% in the matched untreated arm (hazard ratio, 0.14; 95% CI, 0.04 to 0.39); the average survival time at 10 years was 9.6 years for the patients receiving Kygevvi compared with 5.7 years in the control group
- TK2d is an ultra-rare, genetic mitochondrial disorder that impairs the ability to produce and repair mitochondrial deoxyribonucleic acid (mtDNA) resulting in progressive muscle weakness and respiratory failure; prior to approval of Kygevvi, patients received only supportive care; patients with symptom onset on or before the age of 12 years have a high risk of premature death, often within three years after symptoms begin; Kygevvi works by incorporate pyrimidine nucleosides (deoxycytidine and deoxythymidine) into skeletal muscle mtDNA to restore mtDNA copy number
- Kygevvi is expected to be available from UCB in the first quarter of 2026
Nov. 13, 2025 – ziftomenib (Komzifti)
- NDA approval; Assessment Aid, Breakthrough Therapy, Orphan Drug, Priority Review
- Menin inhibitor
- Indicated for the treatment of adults with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options
- Oral capsule: 200 mg
- Recommended dosage is taken orally once daily and continued until disease progression or unacceptable toxicity; patients without confirmed disease progression or unacceptable toxicity should be treated for a minimum of six months to allow time for response
- Approval was based on an open-label, single-arm, multicenter study (KOMET-001; n=112) enrolling adults with R/R AML with an NPM1 mutation; at a median follow-up of 4.2 months (range, 0.1 to 41.2 months), the rate of complete remission (CR) plus CR with partial hematological recovery (CRh) was 21.4% (95% CI, 14.2 to 30.2) and the median duration of CR+CRh was 5 months (95% CI, 1.9 to 8.1) with 17% of patients having a CR and 4.5% of patients demonstrating CRh
- The National Comprehensive Cancer Network (NCCN) AML guidelines list revumenib (Revuforj) and Komzifti as targeted therapy options for R/R AML in adults with the NPM1 mutation; Revuforj is also a menin inhibitor and is indicated for the treatment of R/R AML with a susceptible NPM1 mutation in adult and pediatric patients ≥ 1 year of age who have no satisfactory alternative treatment options
- Boxed Warning for differentiation syndrome
- Komzifti is available from Kura Oncology
Nov. 18, 2025 – plozasrian (Redemplo)
- NDA approval; Breakthrough Therapy, Fast Track, Orphan Drug
- Apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA)
- Indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)
- Injection: 25 mg/0.5 mL solution in a single-dose pre-filled syringe
- Recommended dosage is injected subcutaneously (SC) once every 3 months by a patient and/or caregiver following proper training
- Approval was based on a randomized, placebo-controlled, double-blind, Phase 3 trial (PALISADE; n=51) that demonstrated a median difference between plozasrian and placebo in the percent change in fasting triglyceride levels from baseline to month 10 of –59% (95% CI, -90 to -28; p<0.001); this was based on the an average of two assessments that were taken two to seven days apart
- Olezarsen (Tryngolza) is an apoC-III-directed antisense oligonucleotide (ASO) that has the same indication as Redemplo; Tryngolza is administered SC once monthly by a patient and/or caregiver following proper training
- Redemplo is available from Arrowhead
Nov. 19, 2025 – sevabertinib (Hyrnuo)
- NDA approval; Accelerated Approval, Assessment Aid, Breakthrough Therapy, Orphan Drug, Priority Review, Project Orbis
- Kinase inhibitor
- Indicated for the treatment of adults with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy; Accelerated Approval was based on objective response rate and duration of response, therefore continued approval for this use may require demonstration of benefit in confirmatory trials
- Oral tablets: 10 mg
- Recommended dosage is taken orally twice daily with food until disease progression or unacceptable toxicity
- Approval was based an open-label, single-arm, multicenter, multicohort clinical study (SOHO-01); the cohort of previously treated patients who had not received HER2-targeted therapy (n=70) demonstrated an objective response rate (ORR) of 71% (95% CI, 59 to 82) with the majority of responses being partial (69%) (2.9% complete response); the median duration of response (DOR) for this cohort was 9.2 months (95% CI, 6.3 to 15); in the cohort of patients who had previously received HER2-directed antibody-drug conjugates (n=52), the ORR was 38% (95% CI, 25 to 53) with 33% of patients exhibiting a partial response and 6% having a complete response; the median DOR for this cohort was 7 months (95% CI, 5.6 to not estimable)
- According to the NCCN NSCLC guideline, zongertinib (Hernexeos) is a preferred subsequent therapy option for HER2 (ERBB2) mutation NSCLC with progression following systemic therapy for advanced or metastatic disease; Hernexeos is also a kinase inhibitor that received Accelerated Approval for adults with unresectable or metastatic non-squamous NSCLC whose tumors have HER2 (ERBB2) TKD activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy
- Hyrnuo will be available from Bayer with launch timeframe to be determined (TBD)
None
Oct. 29, 2025 – denosumab-bmwo (Osenvelt)
- Celltrion; Biologics License Application (BLA) approval; the 120 mg/1.7 mL single-dose vial for SC use of denosumab-bmwo (Osenvelt) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Xgeva)
- Previously, Osenvelt was only approved as a biosimilar to Xgeva
Oct. 29, 2025 – denosumab-bmwo (Stoboclo)
- Celltrion; BLA approval; the 60 mg/mL single-dose prefilled syringe for SC use of denosumab-bmwo (Stoboclo) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Prolia)
- Previously, Stoboclo was only approved as a biosimilar to Prolia
Oct. 29, 2025 – denosumab-bnht (Bomyntra)
Fresenius Kabi; BLA approval; the 120 mg/1.7 mL single-dose vial and single-dose prefilled syringe for SC use of denosumab-bnht (Bomyntra) have received FDA approval as interchangeable biosimilars to the same presentations of denosumab (Xgeva)
Previously, Bomyntra was only approved as a biosimilar to Xgeva
Oct. 29, 2025 – denosumab-bnht (Conexxence)
Fresenius Kabi; BLA approval; the 60 mg/mL single-dose prefilled syringe for SC use of denosumab-bnht (Conexxence) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Prolia)
Previously, Conexxence was only approved as a biosimilar to Prolia
Oct. 29, 2025 – denosumab-dssb (Ospomyv)
Samsung Bioepis; BLA approval; the 60 mg/mL single-dose prefilled syringe for SC use of denosumab-dssb (Ospomyv) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Prolia)
Previously, Ospomyv was only approved as a biosimilar to Prolia
Oct. 29, 2025 – denosumab-dssb (Xbryk)
Samsung Bioepis; BLA approval; the 120 mg/1.7 mL single-dose vial for SC use of denosumab-dssb (Xbryk) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Xgeva)
Previously, Xbryk was only approved as a biosimilar to Xgeva
Oct. 29, 2025 – denosumab-kyqq (Aukelso)
Biocon Biologics; BLA approval; the 120 mg/1.7 mL single-dose vial for SC use of denosumab-kyqq (Aukelso) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Xgeva)
Previously, Aukelso was only approved as a biosimilar to Xgeva
Oct. 29, 2025 – denosumab-kyqq (Bosaya)
Biocon Biologics; BLA approval; the 60 mg/mL single-dose prefilled syringe for SC use of denosumab-kyqq (Bosaya) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Prolia)
Previously, Bosaya was only approved as a biosimilar to Prolia
Oct. 29, 2025 – denosumab-nxxp (Bildyos)
Organon; BLA approval; the 60 mg/mL single-dose vial and single-dose prefilled syringe for SC use of denosumab-nxxp (Bildyos) have received FDA approval as interchangeable biosimilars to the same presentations of denosumab (Prolia)
Previously, Bildyos was only approved as a biosimilar to Prolia
Oct. 29, 2025 – denosumab- nxxp (Bilprevda)
Organon; BLA approval; the 120 mg/1.7 mL single-dose vial for SC use of denosumab-nxxp (Bilprevda) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Xgeva)
Previously, Bilprevda was only approved as a biosimilar to Xgeva
Oct. 29, 2025 – denosumab-qbde (Enoby)
Hikma; BLA approval; the 60 mg/mL single-dose prefilled syringe for SC use of denosumab-qbde (Enoby) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Prolia)
Previously, Enoby was only approved as a biosimilar to Prolia
Oct. 29, 2025 – denosumab-qbde (Xtrenbo)
Hikma; BLA approval; the 120 mg/1.7 mL single-dose vial for SC use of denosumab-qbde (Xtrenbo) has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Xgeva)
Previously, Xtrenbo was only approved as a biosimilar to Xgeva
Nov. 13, 2025 – pertuzumab-dpzb (Poherdy)
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BLA approval; pertuzumab-dpzb (Poherdy) is an interchangeable biosimilar to reference drug pertuzumab (Perjeta); first FDA-approved biosimilar to Perjeta
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HER2/neu receptor antagonist
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Indicated for (1) use in combination with trastuzumab (Herceptin, biosimilars) and docetaxel for treatment of adults with HER2-positive metastatic breast cancer (MBC) who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease and (2) use in combination with trastuzumab and chemotherapy as neoadjuvant treatment of adults with HER2-positive, locally advanced, inflammatory or early stage breast cancer (either > 2 cm in diameter or node positive) as part of a complete treatment regimen for early breast cancer and as adjuvant treatment of adults with HER2-positive early breast cancer at high risk of recurrence (same indications as Perjeta)
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Solution for injection: 420 mg/14 mL (30 mg/mL) in a single-dose vial (same as Perjeta)
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Recommended initial dosage is administered as an intravenous (IV) infusion, followed every three weeks by a lower dose administered as an IV infusion (same as Perjeta); infusions are administered by a health care provider (HCP) and require an observation period of 30 to 60 minutes after each infusion and prior to any subsequent administration of trastuzumab or trastuzumab hyaluronidase-oysk (Herceptin Hylecta) or a taxane; for MBC, administer Poherdy, trastuzumab or trastuzumab hyaluronidase-oysk and docetaxel every three weeks; for neoadjuvant treatment, administer Poherdy, trastuzumab or trastuzumab hyaluronidase-oysk and chemotherapy preoperatively every three weeks for three to six cycles; for adjuvant treatment, administer Poherdy, trastuzumab or trastuzumab hyaluronidase-oysk and chemotherapy postoperatively every three weeks for a total of one year (up to 18 cycles) (same as Perjeta)
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Boxed Warning for left ventricular dysfunction and embryo-fetal toxicity
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Product will be available from Organon with launch timeframe TBD
Oct. 23, 2025 – belantamab mafodotin-blmf (Blenrep)
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GlaxoSmithKline (GSK); B-cell maturation antigen (BCMA)-directed antibody and microtubule inhibitor conjugate; Orphan Drug; available only through REMS because of the risk of ocular toxicity
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New indication: in combination with bortezomib (Boruzu, Velcade) and dexamethasone for the treatment of adults with R/R multiple myeloma who have received at least two prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent
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Recommended dosage is based on actual body weight administered as an IV infusion once every three weeks in combination with bortezomib and dexamethasone for the first eight cycles, followed by dosing once every three weeks as a single agent until disease progression or unacceptable toxicity; administered by an HCP
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In August 2020, the FDA had granted Accelerated Approval to Blenrep as a monotherapy treatment for adults with relapsed or refractory multiple myeloma (RRMM) who had received at least four prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor and an immunomodulatory agent; in November 2022, GSK announced initiation of withdrawal of Blenrep from commercialization in the U.S. following a request from the FDA based on results from the DREAMM-3 phase 3 confirmatory trial that did not meet Accelerated Approval regulations; in March 2023, the FDA announced revocation of the biologics license for Blenrep as it did not meet its primary endpoint of superior progression-free survival (PFS) in the DREAMM–3 trial; on Oct. 23, 2025, Blenrep received approval for this new indication under a new BLA
Oct. 24, 2025 – revumenib (Revuforj)
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Syndax; menin inhibitor; Assessment Aid, Fast Track, Priority Review, Real-Time Oncology Review
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New indication: treatment of relapsed or refractory AML with a susceptible NPM1 mutation in adult and pediatric patients ≥ 1 year of age who have no satisfactory alternative treatment options
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Administered orally twice daily fasted or with a low-fat meal (e.g., meals with approximately 400 calories, ≤ 25% fat at approximately the same time each day); dosage is based on patient body weight (≥ 40 kg: fixed dose, < 40 kg: dose based on body surface area [BSA]); dosage is also dependent on if used concurrently with or without strong cytochrome P450 (CYP) 3A4 inhibitors; continue until disease progression or unacceptable toxicity; for patients without disease progression or unacceptable toxicity, treat for a minimum of six months to allow time for response
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Other indication: treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients ≥ 1 year of age
Oct. 27, 2025 – nivolumab and hyaluronidase-nvhy (Opdivo Qvantig)
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Bristol-Myers Squibb; combination of nivolumab (a programmed death receptor-1 [PD-1]-blocking antibody) and hyaluronidase (an endoglycosidase); the FDA also converted the Accelerated Approval of use for the treatment of adult patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan to regular approval
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New indication: treatment of adults with unresectable or metastatic MSI-H or dMMR CRC, following combination treatment with IV nivolumab (Opdivo) and ipilimumab (Yervoy)
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Recommended dosage is administered SC by an HCP in the abdomen or thigh every two weeks or every four weeks (depending on the dose); continued until disease progression or unacceptable toxicity, or up to two years
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Other indications are detailed in the product label
Nov. 6, 2025 – daratumumab and hyaluronidase-fihj (Darzalex Faspro)
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Janssen; combination of daratumumab (a CD38-directed cytolytic antibody) and hyaluronidase (an endoglycosidase); first FDA-approved treatment for patients with high-risk smoldering multiple myeloma
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New indication: treatment of adults with high-risk smoldering multiple myeloma as monotherapy
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Administered as a SC injection by an HCP into the abdomen over approximately three to five minutes according to the following schedule: weekly for weeks one to eight (eight doses total), every two weeks for weeks nine to 24 (eight doses total) and every four weeks beginning at week 25 onwards until diagnosis of multiple myeloma or for a maximum of three years
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Other indications are detailed in the product label
Nov. 18, 2025 – epcoritamab-bysp (Epkinly)
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Genmab; bispecific CD20-directed CD3 T-cell engager; Assessment Aid, Breakthrough Therapy, Orphan Drug, Priority Review; the FDA also granted traditional approval for use as monotherapy for the treatment of adults with R/R follicular lymphoma (FL) after two or more lines of systemic therapy (this was previously an Accelerated Approval)
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New indication: in combination with lenalidomide and rituximab for the treatment of adults with R/R FL
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Administered as a SC injection by an HCP into the lower part of the abdomen (preferred injection site) or the thigh according to the following schedule to reduce the incidence and severity of cytokine release syndrome (CRS): step-up doses on day one, eight and 15 of the first cycle with the first full dose given on day 22 of the first cycle, followed by the full dose on day one, eight, 15 and 22 on cycles two and three; continue dosing with the full dose on day one only of cycles four to 12; patients with FL require assessment to determine whether hospitalization or outpatient monitoring is appropriate for the first full dose given on day 22 of cycle one; administered in 28-day cycles for a total of 12 cycles or until disease progression or unacceptable toxicity, whichever occurs first
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Other indications are detailed in the product label
Nov. 19, 2025 – aflibercept (Eylea HD)
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Regeneron; vascular endothelial growth factor (VEGF) inhibitor; FDA also approved more frequent dosing regimen (every four week [monthly] dosing option) for previously approved indications of neovascular (wet) age-related macular degeneration (nAMD), diabetic macular edema (DME) and diabetic retinopathy (DR)
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New indication: treatment of macular edema following retinal vein occlusion (RVO)
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Administered as an intravitreal injection by an HCP every four weeks for the first three to five doses, followed by intravitreal injection once every eight weeks; some patients did not maintain a response with extended dosing intervals following successful response to the first three to five initial monthly doses; these patients may benefit from resuming every four week dosing
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Other indications are detailed in the product label
Nov. 19, 2025 – selumetinib (Koselugo)
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AstraZeneca; kinase inhibitor; Assessment Aid, Orphan Drug
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Expanded indication: for adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN); previously, only approved for this use in pediatric patients ≥ 1 year of age
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Administered orally twice daily based on BSA; continued until disease progression or unacceptable toxicity; capsules can be administered with or without food; oral granules can be used for patients who have difficulty swallowing whole capsules and are sprinkled on or mixed with a small amount of smooth yogurt or fruit puree (apple, banana, pear or strawberry) and consumed within 30 minutes; the capsule shells of the oral granules should not be swallowed, chewed or dissolved
Traditional
Oct. 24, 2025 – elinzanetant (Lynkuet)
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NDA approval
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Neurokinin 1 (NK1) and neurokinin 3 (NK3) receptor antagonist
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Indicated for the treatment of moderate to severe vasomotor symptoms (hot flashes) due to menopause
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Oral capsule: 60 mg
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Recommended dosage is taken orally once daily at bedtime at about the same time each day with or without food; take capsules with water and swallow whole
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Approval was based on two randomized, double-blind, placebo-controlled, multicenter clinical trials (OASIS 1 and OASIS 2); in both trials (n=796), after the first 12 weeks, women on placebo were switched to elinzanetant and all were treated for a 14-week extension for up to 26 weeks total exposure; in both studies, elinzanetant study arms demonstrated a statistically significant reduction in severity and frequency (coprimary endpoints) of moderate to severe vasomotor symptoms from baseline to weeks four and 12 compared to placebo
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Fezolinetant (Veozah) is also a non-hormonal option for menopausal vasomotor symptoms; it acts as an NK3 receptor antagonist and is also indicated for the treatment of moderate to severe vasomotor symptoms due to menopause; Veozah is an oral tablet and carries a Boxed Warning for hepatotoxicity (Lynkuet carries a Warning for hepatic transaminase elevation)
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Lynkuet is available from Bayer
Oct. 23, 2025 – clonidine oral solution (Javadin)
- 505(b)(2) NDA approval; other oral formulations of clonidine include an extended-release (ER) oral suspension (Onyda XR) and ER oral tablet (generic) for ADHD, as well as an oral tablet (generic), an oral ER tablet (Nexiclon, generics) and a transdermal weekly patch (Catapres, generics) for hypertension
- Central alpha-2 adrenergic agonist
- Indicated for the treatment of hypertension in adults to lower blood pressure; lowering blood pressure has been shown to reduce the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions
- Oral solution: 0.02 mg/mL
- Recommended dosage is administered orally twice daily (morning and bedtime, with either an equal or higher split dosage given at bedtime); take with or without food; titrated at weekly intervals, if needed, until desired response is achieved
- Javadin is available from Azurity
Oct. 24, 2025 – sotatercept-csrk (Winrevair)
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Merck; activin signaling inhibitor; expanded indication based on results of the ZENITH study
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Expanded indication: to specify that Winrevair is indicated to reduce risk of hospitalization for pulmonary arterial hypertension (PAH), lung transplantation and death; previously indicated for the treatment of adults with PAH (WHO Group 1 pulmonary hypertension) to improve exercise capacity and WHO functional class (FC) and to reduce the risk of clinical worsening events
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Administered as a weight-based SC injection every three weeks; patients/caregivers may administer after proper training; dosage modifications may be required due to increased hemoglobin and decreased platelets (check hemoglobin and platelets prior to each dose for the first five dosesor longer if unstable; monitor periodically)
Nov. 4, 2025 – linaclotide (Linzess)
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Abbvie; guanylate cyclase-C agonist; first FDA-approved therapy for irritable bowel syndrome with constipation (IBS-C) in pediatric patients
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Expanded indication: for pediatric patients ≥ 7 years of age with IBS-C; previously only indicated for this use in adults
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Administered orally once daily on an empty stomach (≥ 30 minutes prior to a meal) at approximately the same time every day; capsules should be swallowed whole; if capsules cannot be swallowed, open the capsule and administer either in applesauce or with water; can also be administered with water via a nasogastric or gastrostomy tube (administration on other soft foods or in other liquids has not been evaluated)
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Other indications are detailed in the product label
Nov. 5, 2025 – lumateperone (Caplyta)
- Intra-Cellular Therapies; atypical antipsychotic
- New indication: adjunctive therapy with antidepressants for the treatment of major depressive disorder (MDD) in adults
- Recommended dosage is administered orally once daily with or without food; dose titration is not required
- Other indications are detailed in the product label
Nov. 5, 2025 – naltrexone and bupropion (Contrave)
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Currax; combination of an opioid antagonist (naltrexone) and an aminoketone antidepressant (bupropion)
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Revised indication: in combination with a reduced-calorie diet and increased physical activity to reduce excess body weight and maintain weight reduction long term in adults with obesity or overweight in the presence of at least one weight related comorbid condition; previously, the indication included initial body mass index (BMI) levels
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Administered orally twice daily following an initial titration schedule (dosed once daily the first week); do not take with a high-fat meal due to a significant increase in systemic exposure; after 12 weeks at the maintenance dosage, evaluate response (discontinue if at least a 5% reduction from baseline body weight has not been achieved)
First generic drug launches
Oct. 17, 2025 – conjugated estrogens (Premarin)
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Novast launched generic tablets (0.3 mg, 0.45 mg, 0.625 mg, 0.9 mg, 1.25 mg) to Wyeth’s Premarin
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Mixture of estrogens; indicated for (1) treatment of moderate to severe vasomotor symptoms due to menopause, (2) treatment of moderate to severe vulvar and vaginal atrophy due to menopause, (3) treatment of hypoestrogenism due to hypogonadism, castration or primary ovarian failure, (4) treatment of breast cancer (for palliation only) in appropriately selected women and men with metastatic disease, (5) treatment of advanced androgen-dependent carcinoma of the prostate (for palliation only) and (6) prevention of postmenopausal osteoporosis
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Recommended dosage is dependent on indication for use and whether it is given continuously (with no interruption in therapy) or in cyclical regimens
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Annual sales for Premarin tablets in 2024 were $233 million
ADHD attention-deficit/hyperactivity disorder
CD cluster of differentiation
FDA Food and Drug Administration
HER2 human epidermal growth factor receptor 2
CD cluster of differentiation
FDA Food and Drug Administration
HER2 human epidermal growth factor receptor 2
RANK receptor activator of nuclear factor-kappa B
REMS Risk Evaluation and Mitigation Strategy
U.S. United States
WHO World Health Organization
Editor-In-Chief: Maryam Tabatabai, PharmD
Executive Editor: Anna Schreck Bird, PharmD
Deputy Editors: Nicole Kjesbo, PharmD, BCPS; Olivia Pane, PharmD, CDCES
The content in this publication is not a substitute for professional medical advice. For questions regarding any medical condition or if you need medical advice, please contact your health care provider.
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All brand names are property of their respective owners.
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