Drug Approvals: March 2025 - Prime Therapeutics
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Drug Approvals: March 2025
Monthly update on newly approved specialty and traditional drugs, new indications and first-time generic launches
March 26, 2025
Specialty
March 5, 2025 – revakinagene taroretcel-lwey (Encelto)
- Biologics License Application (BLA) approval
- Allogeneic encapsulated cell-based gene therapy
- Indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel)
- Implant: one single-dose implant containing 200,000 to 440,000 allogeneic retinal pigment epithelial cells expressing recombinant human ciliary neurotropic factor (rhCNTF)
- Recommended dosage is one implant per affected eye; the implant is placed via surgical intravitreal implantation under aseptic conditions by a qualified ophthalmologist; removal also requires a surgical procedure in an operating room under aseptic conditions by a qualified ophthalmologist; removal is done if vitrectomy with a complete gas fill or silicone oil fill is required or if infectious endophthalmitis occurs
- Approval was based on results from two randomized, multicenter, sham-controlled Phase 3 trials (n=115 for study 1; n=113 for study 2) that demonstrated revakinagene taroretcel-lwey significantly slowed the loss of macular photoreceptors, as measured by the rate of change in ellipsoid zone area loss from baseline over 24 months, in adults with MacTel (difference versus sham: p<0.0001 for study 1 and p=0.0186 for study 2)
- Encelto is the first and only United States (U.S.) Food & Drug Administration (FDA)-approved treatment available for this rare, progressive neurodegenerative disease of the macula that leads to degeneration of photoreceptors and irreversible vision loss; Encelto expresses rhCNTF which may promote survival and maintenance of photoreceptors
- Product will be available from Neurotech in June 2025 with pricing to follow
Feb. 19, 2025 – nilotinib d-tartrate (no trade name)
- 505(b)(2) New Drug Application (NDA) approval
- Kinase inhibitor
- Indicated for the treatment of (1) adults with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP) and (2) adults with CP and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib
- Oral capsules: 50 mg, 150 mg and 200 mg
- Recommended daily dosage is based on indication and is taken orally twice daily at approximately 12-hour intervals on an empty stomach (no food should be consumed for at least two hours before the dose and for at least one hour after); select patients may be considered for treatment discontinuation based on length of therapy and achievement of sustained molecular response
- Boxed warning for QT prolongation and sudden deaths
- Other formulations of nilotinib include oral tablets (Danziten: 71 mg and 95 mg – no mealtime restrictions) and oral capsules (Tasigna: 50 mg, 150 mg and 200 mg – mealtime restrictions); Danziten has the same indications as nilotinib d-tartrate, whereas Tasigna’s indications are similar but also include use for select pediatric patients
- Product will be available from Cipla with launch time frame and pricing to be determined (TBD)
Feb. 28, 2025 – denosumab-bmwo (Osenvelt)
- BLA approval; denosumab-bmwo (Osenvelt) is a biosimilar to reference drug denosumab (Xgeva)
- RANK ligand inhibitor
- Osenvelt is indicated for (1) prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors; (2) treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity; and (3) treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy (same indications as reference drug Xgeva)
- Solution for injection: 120 mg/1.7 mL in a single-dose vial (same as Xgeva)
- Recommended dosage is given every four weeks subcutaneously (SC) into the upper arm, upper thigh or abdomen administered by a health care professional (HCP); additional doses are given on days eight and 15 for the first month of therapy in patients being treated for giant cell tumor of bone or hypercalcemia of malignancy; calcium and vitamin D should be taken as needed to treat/prevent hypocalcemia
- Product will be available from Celltrion with availability expected in June 2025 and pricing to follow
Feb. 28, 2025 – denosumab-bmwo (Stoboclo)
- BLA approval; denosumab-bmwo (Stoboclo) is a biosimilar to reference drug denosumab (Prolia)
- RANK ligand inhibitor
- Stoboclo is indicated for the treatment (1) of postmenopausal women with osteoporosis at high risk for fracture; (2) to increase bone mass in men with osteoporosis at high risk for fracture; (3) of glucocorticoid-induced osteoporosis in men and women at high risk for fracture; (4) to increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer; and (5) to increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer (same indications as Prolia)
- Solution for injection: 60 mg/mL single-dose prefilled syringes (same as Prolia)
- Recommended dosage is given every six months as a SC injection in the upper arm, upper thigh or abdomen administered by an HCP; patients should take calcium and vitamin D daily
- Boxed warning for severe hypocalcemia in patients with advanced kidney disease
- Product will be available from Celltrion with availability expected in June 2025 and pricing to follow
March 7, 2025 – omalizumab-igec (Omlyclo)
- BLA approval; omalizumab-igec (Omlyclo) is the first and only interchangeable biosimilar to reference drug omalizumab (Xolair)
- Anti-immunoglobulin E (IgE) antibody
- Omlyclo is indicated for (1) moderate to severe persistent asthma in adults and pediatric patients ≥ 6 years of age with a positive skin test or in vitro reactivity to a perennial aeroallergen and symptoms that are inadequately controlled with inhaled corticosteroids; (2) chronic rhinosinusitis with nasal polyps (CRSwNP) in adults ≥ 18 years of age with inadequate response to nasal corticosteroids, as add-on maintenance treatment; (3) IgE-mediated food allergy in adult and pediatric patients ≥ 1 year of age for the reduction of allergic reactions (Type I), including anaphylaxis, that may occur with accidental exposure to one or more foods (to be used in conjunction with food allergen avoidance); and (4) chronic spontaneous urticaria (CSU) in adults and adolescents ≥ 12 years of age who remain symptomatic despite H1 antihistamine treatment (same indications as reference drug Xolair)
- Solution for injection: 75 mg/0.5 mL and 150 mg/mL in single-dose prefilled syringes (Xolair is also available in a 300 mg/2 mL prefilled syringe as well as a prefilled autoinjector [all three strengths] and a 150 mg lyophilized powder in a single-dose vial)
- Recommended dosage is given SC and requires initiation in a health care setting; once therapy has been established, the HCP may determine whether administration by the patient or caregiver is appropriate (depending on an assessment of risk for anaphylaxis as detailed in the product labeling); dosage for asthma, chronic rhinosinusitis with nasal polyps and IgE-mediated food allergy is given every two or four weeks based on serum total IgE level (IU/mL) measured before the start of treatment, and by body weight; dosage for CSU is given every four weeks and is not dependent on serum IgE level or body weight
- Boxed warning for anaphylaxis
- Product will be available from Celltrion with time frame TBD and pricing to follow
Feb. 28, 2025 – eculizumab (Soliris)
- Alexion; complement inhibitor; first and only treatment available for pediatric patients with generalized myasthenia gravis
- Expanded indication: the treatment of generalized myasthenia gravis (gMG) in pediatric patients ≥ 6 years of age who are anti-acetylcholine receptor (AChR) antibody positive; previously, indicated for this use in adults only
- Administered as an intravenous (IV) infusion based upon body weight; following an induction course, maintenance dosing is given every two to three weeks depending on body weight
- Other indications: (1) paroxysmal nocturnal hemoglobinuria (PNH); (2) atypical hemolytic uremic syndrome (aHUS); (3) neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive
Feb. 28, 2025 – tocilizumab-aazg (Tyenne)
- Fresenius Kabi; interleukin-6 (IL-6) receptor antagonist; biosimilar to tocilizumab (Actemra); reference drug Actemra carries additional indication for slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD)
- New indications: (1) hospitalized adults with COVID-19 who are receiving systemic corticosteroids and require supplemental oxygen, noninvasive or invasive mechanical ventilation or ECMO and (2) patients ≥ 2 years of age with chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS)
- Administered as a weight-based 60-minute IV infusion for both COVID-19 and CRS; for COVID-19, if clinical signs/symptoms worsen or do not improve after the first dose, one additional infusion may be administered at least eight hours after the initial infusion; for CRS, if no clinical improvement in signs/symptoms occurs after the first dose, up to three additional doses may be administered with an interval between doses of at least eight hours
- Other indications: select adults with (1) rheumatoid arthritis or (2) giant cell arteritis; select patients ≥ 2 years of age with (1) polyarticular or (2) systemic juvenile idiopathic arthritis
March 4, 2025 – tislelizumab-jsgr (Tevimbra)
- BeiGene; programmed death receptor-1 (PD-1)-blocking antibody
- Expanded indication: in combination with platinum-containing chemotherapy for the first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express programmed death-ligand 1 (PD-L1) (≥1) (select patients based on the presence of PD-L1 in tumor specimens); previously, indicated only for esophageal cancer as a single agent in adults with unresectable or metastatic ESCC after prior systemic chemotherapy that did not include a PD-L1 inhibitor
- Administered as an IV infusion every three weeks by an HCP with the first infusion administered over 60 minutes; if tolerated, subsequent infusions may be administered over 30 minutes; continued until disease progression or unacceptable toxicity
- Other indication: in combination with other agents as first-line treatment for adults with unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors express PD-L1 (≥1)
March 6, 2025 – furosemide (Furoscix)
- scPharmaceuticals; loop diuretic; expected to be available for this new patient population in April 2025
- Expanded indication: to include the treatment of edema in adults with chronic kidney disease, including the nephrotic syndrome; previously, only indicated for patients with chronic heart failure
- Administered SC with the single-use, on-body infusor which is pre-programmed to deliver a loading dose of furosemide over the first hour followed by a steady maintenance infusion for the subsequent four hours; not for chronic use and should be replaced with oral diuretics as soon as practical
March 20, 2025 – guselkumab (Tremfya)
- Janssen; interleukin-23 antagonist
- New indication: treatment of adults with moderately to severely active Crohn’s disease
- Administered as an induction course via IV infusion or via SC injection given every four weeks for three doses (week zero, four and eight) followed by SC maintenance dosing with frequency dependent on the dose (either started on week 16 and given every eight weeks thereafter or started at week 12 and given every four weeks thereafter); the lowest effective recommended dosage that maintains therapeutic response should be used
- Other indications: select adult patients with (1) plaque psoriasis; (2) psoriatic arthritis; or (3) ulcerative colitis
March 20, 2025 – iptacopan (Fabhalta)
- Novartis; complement factor B inhibitor; Breakthrough Therapy, Orphan Drug, Priority Review; first and only treatment approved for C3 glomerulopathy
- New indication: treatment of adults with complement 3 glomerulopathy (C3G) to reduce proteinuria
- Administered orally twice daily with or without food
- Other indications: (1) adults with PNH; (2) reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression (Accelerated Approval)
March 20, 2025 – vutrisiran (Amvuttra)
- Alnylam; transthyretin-directed small interfering RNA
- New indication: treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits
- Administered SC only by an HCP once every three months
- Other indication: polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Traditional
None
Feb. 24, 2025 – copper intrauterine system (Miudella)
- 505(b)(2) NDA approval; first hormone-free copper intrauterine system approved in the U.S. in more than 40 years
- Copper-containing intrauterine system (IUS)
- Indicated for prevention of pregnancy in female patients of reproductive potential for up to three years
- Intrauterine system: 175 mm² exposed copper surface area with a nitinol frame with copper sleeves and monofilament polymer retrieval thread, preloaded in a sterile inserter
- A single Miudella is inserted at the fundus of the uterine cavity by a trained HCP using clean technique; must be removed or replaced after three years; patient should be re-examined as clinically indicated
- Boxed warning for risk of complications due to improper insertion; only available through a Risk Evaluation and Mitigation Strategy (REMS) to ensure HCPs are trained on proper insertion before first use
- Product will be available from Sebela Women's Health with launch time frame and pricing TBD
March 13, 2025 – losartan potassium (Arbli)
- 505(b)(2) NDA approval; first and only U.S. FDA-approved ready-to-use oral liquid losartan
- Angiotensin II receptor blocker (ARB)
- Indicated for (1) treatment of hypertension, to lower blood pressure in patients > 6 years old (lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions); (2) reduction of the risk of stroke in patients with hypertension and left ventricular hypertrophy (evidence this benefit does not apply to Black patients); (3) treatment of diabetic nephropathy with an elevated serum creatinine and proteinuria in patients with type 2 diabetes and a history of hypertension
- Oral suspension: 10 mg/mL
- Recommended dosage is taken orally once daily with dosing dependent on indication, patient age (pediatric starting dosage is weight based) and patient response
- Boxed warning for fetal toxicity
- Product will be available from Scienture in the third quarter of 2025 with pricing to follow
March 17, 2025 – chlorthalidone (Hemiclor)
- 505(b)(2) NDA approval; first chlorthalidone tablet in this strength (existing tablets are 15 mg, 25 mg and 50 mg)
- Thiazide-like diuretic
- Indicated for the treatment of hypertension in adults, to lower blood pressure (lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions)
- Oral tablets: 12.5 mg
- Recommended dosage is taken orally once daily and may be increased based on individual response
- Product will be available from PRM with launch TBD and pricing to follow
Feb. 19, 2025 – emtricitabine, rilpivirine and tenofovir alafenamide (Odefsey)
- Gilead; three-drug combination of emtricitabine (FTC) and tenofovir alafenamide (TAF), both HIV nucleoside analog reverse transcriptase inhibitors (NRTIs), and rilpivirine (RPV), a non-nucleoside reverse transcriptase inhibitor (NNRTI)
- Expanded indication: to include (1) pediatric patients weighing ≥ 25 kg to < 35 kg for the treatment of HIV-1 infection as initial therapy in those with no antiretroviral treatment history with HIV-1 RNA ≤ 100,000 copies per mL and (2) pediatric patients weighing ≥ 25 kg to < 35 kg for the treatment of HIV-1 infection to replace a stable antiretroviral regimen in those who are virologically-suppressed (HIV-1 RNA < 50 copies/mL) for at least six months with no history of treatment failure and no known substitutions associated with resistance to the individual components of Odefsey; previously, indicated only for patients weighing ≥ 35 kg
- Administered as one tablet taken orally once daily with a meal
Feb. 27, 2025 – house dust mite (Dermatophagoides farinae and Dermatophagoides pteronyssinus) allergen extract (Odactra)
- ALK-Abello; allergen extract
- Expanded indication: for pediatric patients 5 to 11 years of age as immunotherapy for the treatment of house dust mite (HDM)-induced allergic rhinitis, with or without conjunctivitis, confirmed by positive in vitro testing for IgE antibodies to Dermatophagoides farinae or Dermatophagoides pteronyssinus house dust mites or by positive skin testing to licensed house dust mite allergen extracts; previously, indicated for this use in patients 12 to 65 years of age
- Administered as one tablet daily; the tablet is placed under the tongue and dissolves within 10 seconds; the patient should not swallow for at least one minute; the first dose requires administration under the supervision of an HCP with expertise in allergic diseases as the patient should be observed in the office for ≥ 30 minutes following the initial dose
March 5, 2025 – epinephrine nasal spray (Neffy)
- ARS Pharmaceuticals; alpha and beta-adrenergic receptor agonist; new strength of 1 mg/0.1 mL of epinephrine per spray also approved in conjunction with expanded pediatric population; previously, only approved as 2 mg/0.1 mL of epinephrine per spray; the 1 mg product is expected to be available by the end of May 2025
- Expanded indication: for emergency treatment of type I allergic reactions, including anaphylaxis, in pediatric patients ≥ 4 years of age who weigh ≥ 15 kg; previously, indicated only for this use in pediatric and adult patients who weighed ≥ 30 kg
- Administered intranasally with dosage for patients ≥ 4 years of age based on body weight (≥ 30 kg: one spray of 2 mg; 15 kg to < 30 kg: one spray of 1 mg); if symptoms do not improve or if symptoms worsen after the initial dose, a second dose should be given in the same nostril with a new nasal spray starting five minutes after the first dose; patients should have immediate access to two neffy nasal sprays at all times; patients should seek emergency medical assistance for the anaphylactic episode
March 17, 2025 – glucagon nasal powder (Baqsimi)
- Amphastar; antihypoglycemic agent
- Expanded indication: the treatment of severe hypoglycemia in pediatric patients 1 year of age to 3 years of age with diabetes; previously, only indicated for patients ≥ 4 years of age with diabetes
- Administered as one actuation of the intranasal device into one nostril; the dose is administered by inserting the tip into one nostril and pressing the device plunger all the way in until the green line is no longer showing (does not require inhalation); emergency assistance should be called immediately after administration; if after 15 minutes there has not been a response, another dose may be administered while waiting for emergency assistance; once the patient responds to treatment, oral carbohydrates should be given
First generic drug launches
Feb. 27, 2025 – mercaptopurine 20 mg/mL oral suspension (Purixan)
- Hikma launched a generic to Nova’s Purixan
- Nucleoside metabolic inhibitor indicated for the treatment of patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy maintenance regimen
- Recommended dosage is based on weight or body surface area (BSA) and is dosed orally once daily with dosage adjustments to maintain desirable absolute neutrophil count and for excessive myelosuppression
- In 2024, Purixan generated < $10 million in annual sales in the U.S.
March 7, 2025 – rivaroxaban 2.5 mg tablet (Xarelto)
- Lupin launched a generic to Janssen’s Xarelto
- Factor Xa inhibitor indicated (1) to reduce the risk of major cardiovascular events in patients with coronary artery disease (CAD) and (2) to reduce the risk of major thrombotic vascular events in patients with peripheral artery disease (PAD), including patients after recent lower extremity revascularization due to symptomatic PAD
- Recommended dosage is one tablet orally twice daily with or without food, in combination with low-dose aspirin once daily
- In 2024, Xarelto generated < $10 million in annual sales in the U.S.
COVID-19 coronavirus disease 2019
ECMO extracorporeal membrane oxygenation
HER2 human epidermal growth factor receptor 2
HIV human immunodeficiency virus
RANK receptor activator of nuclear factor κB
RNA ribonucleic acid
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