Drug Approvals: February 2025 - Prime Therapeutics
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Drug Approvals: February 2025
Monthly update on newly approved specialty and traditional drugs, new indications and first-time generic launches
February 28, 2025
Specialty
Jan. 21, 2025 – treosulfan (Grafapex)
- New Drug Application (NDA) approval; Orphan Drug, Standard Review
- Alkylating drug
- Indicated for (1) use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients ≥ 1 year of age with acute myeloid leukemia (AML) and (2) use in combination with fludarabine as a preparative regimen for alloHSCT in adult and pediatric patients ≥ 1 year of age with myelodysplastic syndrome (MDS)
- For injection: 1 g/vial and 5 g/vial as a lyophilized powder in a single-dose vial
- Recommended dosage is given daily and is based on body surface area (BSA); administered as a two hour intravenous (IV) infusion by a health care professional (HCP) on three consecutive days (day -4, -3, -2) in conjunction with fludarabine before hematopoietic stem cell infusion (day 0); patients should receive premedication with antiemetics before the first dose and continue antiemetics on a fixed schedule through completion of administration
- Approval was based on a randomized, active-controlled, non-United States (U.S.) trial (MC-FludT.14/L Trial II; n=476) that compared treosulfan to busulfan with fludarabine as a preparative regimen for allogeneic transplantation and demonstrated that event-free survival two years after HSCT was 64% (95% confidence interval [CI], 56 to 70.9) in the treosulfan group and 50.4% (95% CI, 42.8 to 57.5) in the busulfan group (hazard ratio [HR], 0.65; 95% CI, 0.47 to 0.90; p<0.0001 for non-inferiority, p=0.0051 for superiority)
- Grafapex provides another alkylating agent option for the preparative regimen for alloHSCT
- Boxed warning for myelosuppression
- Product will be available from Medexus in the first half of 2025 with an average wholesale price (AWP) of $732 per 1 g vial and $3,660 per 5 g vial
Feb. 11, 2025 – mirdametinib (Gomekli)
- NDA approval; Assessment Aid, Fast Track, Orphan Drug, Priority Review; first medication Food & Drug Administration (FDA)-approved for both adult and pediatric patients with neurofibromatosis type 1 plexiform neurofibromas
- Kinase inhibitor
- Indicated for the treatment adult and pediatric patients ≥ 2 years of age with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection
- Oral capsules: 1 mg and 2 mg; tablets for oral suspension: 1 mg
- Recommended dosage is based on BSA and is taken twice daily, with or without food, for the first 21 days of each 28-day cycle continued until disease progression or unacceptable toxicity
- Approval was based on an open-label, multicenter, single-arm, Phase 2b trial (ReNeu; n=114; 58 adults and 56 pediatric patients) which demonstrated a confirmed objective response rate (ORR) of 41% in adults and 52% in pediatric patients during a 24-cycle treatment phase
- Gomekli is expected to compete with selumetinib (Koselugo), which is also a kinase inhibitor indicated for the treatment of pediatric patients ≥ 2 years of age with NF1 who have symptomatic, inoperable PN; this therapy is also dosed orally twice daily in a capsule formulation
- Product is available from SpringWorks and carries an AWP of $495 per 2 mg and $247.50 per 1 mg
Feb. 14, 2025 – vimseltinib (Romvimza)
- NDA approval; Assessment Aid, Fast Track, Priority Review
- Kinase inhibitor
- Indicated for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity
- Oral capsules: 14 mg, 20 mg and 30 mg
- Recommended dosage is taken twice weekly, with a minimum of 72 hours between doses as described in the blister package
- Approval was based on a double-blind, multicenter, randomized (2:1), placebo-controlled, Phase 3 trial (MOTION; n=123) that demonstrated a statistically significant improvement in ORR at week 25 with vimseltinib compared to placebo (40% versus 0%, respectively; 95% CI, 29 to 51; p<0.0001)
- Romvimza is expected to compete with the kinase inhibitor pexidartinib (Turalio) which is similarly indicated for the treatment of adults with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery; unlike Romvimza, Turalio carries a boxed warning for hepatotoxicity
- Product will be available from Deciphera within a week of approval with pricing to follow
Feb. 3, 2025 – apomorphine hydrochloride (HCl) (Onapgo)
- 505(b)(1) NDA approval; Orphan Drug, Standard Review; new subcutaneous (SC) infusion formulation of apomorphine; existing formulations are solutions for SC injection intended for intermittent use; Onapgo is not a substitute for apomorphine products
- Dopaminergic agonist
- Indicated for treatment of motor fluctuations in adults with advanced Parkinson’s disease
- Solution for injection: 98 mg/20 mL (4.9 mg/mL) of apomorphine HCl in single-dose cartridges
- Recommended daily dosage is based on individualized titration and includes a continuous dosage and as-needed extra dose(s); treatment with trimethobenzamide is recommended to start three days prior to the first dose and be continued as long as needed to control nausea and vomiting (usually no longer than two months); patients should be trained on using the delivery system, either themselves or with the assistance of a caregiver
- Product is available from Supernus with an AWP of $345.20 per 20 mL single-dose cartridge
Feb. 21, 2025 – chenodiol (Ctexli)
- 505(b)(2) NDA approval; Fast Track, Orphan Drug, Priority Review; first FDA-approved drug to treat cerebrotendinous xanthomatosis (CTX), a rare genetic metabolic disorder leading to impaired fat metabolism
- Bile acid
- Indicated for the treatment of cerebrotendinous xanthomatosis in adults
- Oral tablets: 250 mg
- Recommended dosage is taken three times daily with or without food; baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels should be obtained in patients prior to starting therapy
- Product will be available from Mirum with launch to be determined (TBD) and pricing to follow
Jan. 24, 2025 – tocilizumab-anoh (Avtozma)
- Biologics License Application (BLA) approval; third biosimilar to tocilizumab (Actemra)
- Interleukin (IL)-6 receptor antagonist
- Indicated for adults with (1) moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to at least one DMARD(s), (2) giant cell arteritis and (3) COVID-19 who are hospitalized and receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or ECMO; also indicated for patients ≥ 2 years of age with (1) active polyarticular juvenile idiopathic arthritis and (2) active systemic juvenile idiopathic arthritis; Actemra has additional indications (cytokine release syndrome, systemic sclerosis-associated interstitial lung disease)
- Solution for injection: IV formulation (80 mg/4 mL, 200 mg/10 mL, 400 mg/20 mL in single-dose vials for further dilution prior to IV infusion) and SC formulation (162 mg/0.9 mL in a single-dose prefilled syringe or single-dose prefilled autoinjector) (same presentations as reference drug, Actemra)
- Boxed warning for risk of serious infections
- Product will be available from Celltrion with launch TBD and pricing to follow
Feb. 13, 2025 – denosumab-dssb (Ospomyv, unbranded biologic)
- BLA approval; Ospomyv is a biosimilar to reference drug denosumab (Prolia); the BLA also provides for unbranded biological product labeling for denosumab-dssb
- RANK ligand inhibitor
- Ospomyv and the unbranded biologic (denosumab-dssb) are indicated for 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 1,000 mg of calcium and ≥ 400 IU of vitamin D daily
- Boxed warning for severe hypocalcemia in patients with advanced kidney disease
- Product will be available from Samsung Bioepis with timeframe TBD and pricing to follow
Feb. 13, 2025 – denosumab-dssb (Xbryk)
- BLA approval; denosumab-dssb (Xbryk) is a biosimilar to reference drug denosumab (Xgeva)
- RANK ligand inhibitor
- Xbryk 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 SC into the upper arm, upper thigh or abdomen administered by an 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 Samsung Bioepis with timeframe TBD and pricing to follow
Feb. 14, 2025 – insulin aspart-szjj (Merilog/Merilog SoloStar)
- BLA approval; biosimilar to reference product insulin aspart (Novolog); first FDA-approved rapid acting insulin biosimilar product; third insulin biosimilar product approved (other two are long-acting insulin biosimilars)
- Rapid acting human insulin analog
- Indicated to improve glycemic control in adults and pediatric patients with diabetes mellitus; same indication as reference drug Novolog
- Solution for injection: 100 units/mL (U-100) in a 10 mL multi-dose vial and 3 mL single-patient-use SoloStar prefilled pen for SC use; Novolog (U-100) is also available in a 10 mL multi-dose vial as well as 3 mL single-patient-use FlexPen and FlexTouch prefilled pens and a 3 mL single-patient-use PenFill prefilled cartridge for the 3 mL PenFill cartridge device (Novolog is administered SC and IV)
- Product will be available from Sanofi-Aventis with launch TBD and pricing to follow
Jan. 27, 2025 – fam-trastuzumab deruxtecan-nxki (Enhertu)
- Daiichi Sankyo; human epidermal growth factor receptor 2 (HER2)-directed antibody and topoisomerase inhibitor conjugate; Assessment Aid, Breakthrough Designation, Priority Review
- Expanded indication: adults with unresectable or metastatic hormone receptor (HR)-positive, HER2-low (immunohistochemistry [IHC] 1+ or IHC 2+/in situ hybridization [ISH]-) or HER2-ultra low (IHC 0 with membrane staining) breast cancer, as determined by an FDA-approved test, that has progressed on one or more endocrine therapies in the metastatic setting; previously, only indicated for adults with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer, as determined by an FDA-approved test, who have received a prior chemotherapy in the metastatic setting, or developed disease recurrence during or within six months of completing adjuvant chemotherapy
- Administered as a weight-based dose given as an IV infusion once every three weeks (21-day cycle) until disease progression or unacceptable toxicity
- Other indications: select adult patients with breast cancer, gastric or gastroesophageal junction adenocarcinoma or solid tumors that are HER2-positive, or non-small cell lung cancer (NSCLC) whose tumors have activating HER2-mutations
Feb. 3, 2025 – ranibizumab (Susvimo)
- Genentech; vascular endothelial growth factor (VEGF) inhibitor
- New indication: for diabetic macular edema (DME) in patients who have previously responded to at least two intravitreal injections of a VEGF inhibitor
- Administered via the Susvimo ocular implant, continuously delivered with refills administered every 24 weeks (approximately six months)
- Other indication: neovascular (wet) age-related macular degeneration (AMD)
Feb. 11, 2025 – brentuximab vedotin (Adcetris)
- Seagen; cluster of differentiation (CD)30-directed antibody and microtubule inhibitor conjugate
- New indication: in combination with lenalidomide and a rituximab product for the treatment of adult patients with relapsed or refractory large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from indolent lymphoma, or high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy who are not eligible for autologous hematopoietic stem cell transplantation (auto-HSCT) or chimeric antigen receptor (CAR) T-cell therapy
- Administered as a weight-based 30-minute IV infusion by an HCP every three weeks until disease progression or unacceptable toxicity; administer granulocyte-colony stimulating factor (G-CSF) beginning with cycle one
- Other indications: select pediatric and adult patients with classical Hodgkin lymphoma, anaplastic large cell lymphoma, other CD30-expressing peripheral T cell lymphomas (PTCL) or CD30-expressing mycosis fungoides
Traditional
Jan. 30, 2025 – suzetrigine (Journavx)
- NDA approval; Breakthrough Therapy, Fast Track, Priority Review; first-in-class non-opioid analgesic
- Sodium channel blocker
- Indicated for the treatment of moderate to severe acute pain in adults
- Oral tablets: 50 mg
- Recommended starting dose is a loading dose taken on an empty stomach at least one hour before or two hours after food; beginning 12 hours after the starting dose, one tablet is taken every 12 hours with or without food; Journavx should be used for the shortest duration, consistent with individual patient treatment goals (use has not been studied beyond 14 days)
- Approval was based on two randomized, double-blind, placebo- and active-controlled trials (NAVIGATE 1 and 2) evaluating suzetrigine after abdominoplasty (n=1,118) and bunionectomy (n=1,073) surgery; in both studies, suzetrigine demonstrated a superior pain reduction compared to placebo based on the primary endpoint of time-weighted sum of the pain intensity difference from zero to 48 hours (SPID48); suzetrigine was not found to be superior to hydrocodone bitartrate/acetaminophen for SPID48 in either study
- Journavx has a novel mechanism of action and inhibits pain signals in the periphery through its selectivity for the NaV1.8 voltage-gated sodium channel, providing an acute pain management option for short-term use that does not carry the same safety risks as opioid analgesics
- Product is available from Vertex and carries an AWP of $558 per 30-count bottle of tablets
Feb. 14, 2025 – chikungunya vaccine, recombinant (Vimkunya)
- BLA approval; Accelerated Approval; second FDA-approved vaccine for prevention of chikungunya virus
- Indicated for the prevention of disease caused by chikungunya virus in individuals ≥ 12 years of age
- Injectable suspension: 0.8 mL in a prefilled syringe
- Recommended dosage is a single 0.8 mL dose administered intramuscularly (IM) by an HCP
- Product will be available from Bavarian Nordic in the first half of 2025
Feb. 14, 2025 – meningococcal groups A, B, C, W and Y vaccine (Penmenvy)
- BLA approval; Advisory Committee on Immunization Practices (ACIP) was expected to vote on recommendations for the MenABCWY vaccine in adolescents and young adults on Feb. 26, 2025; however, the meeting has been postponed to accommodate public comment in advance of the meeting
- Indicated for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroups A, B, C, W and Y in individuals 10 through 25 years of age
- Following reconstitution of a single dose, the injectable suspension is approximately 0.5 mL
- Recommended dosage is administered IM by an HCP as two doses (approximately 0.5 mL each) six months apart; appropriate medical equipment must be immediately available in the case of an anaphylactic reaction following administration
- Product will be available from GSK with launch information TBD and pricing to follow
Jan. 28, 2025 – hydrochlorothiazide (Inzirqo)
- 505(b)(2) NDA approval; Standard Review; first oral suspension formulation of hydrochlorothiazide; previously available oral solutions are no longer marketed
- Thiazide diuretic
- Indicated for (1) treatment of hypertension in adult and pediatric patients alone or in combination with other anti-hypertensive agents, to lower blood pressure (which reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarction) and (2) treatment of edema associated with congestive heart failure, hepatic cirrhosis and renal disease including nephrotic syndrome in adult and pediatric patients
- Oral suspension: 10 mg/mL
- Recommended dosage is based on indication and whether the patient is an adult or child and is taken as single or two divided doses per day
- Product will be available from Ani with launch timeframe and pricing TBD
Jan. 30, 2025 – meloxicam/rizatriptan (Symbravo)
- 505(b)(2) NDA approval; Standard Review
- Combination of the NSAID, meloxicam, and the triptan, rizatriptan
- Indicated for the acute treatment of migraine with or without aura in adults; Symbravo should only be used where a clear diagnosis of migraine has been determined; it is not indicated for the preventive treatment of migraine or for the treatment of cluster headache
- Oral tablets: 20 mg meloxicam/10 mg rizatriptan
- Recommended dosage is one tablet by mouth as needed (maximum daily dose of one tablet)
- Boxed warning for risk of serious cardiovascular and gastrointestinal events
- Product will be available from Axsome within approximately four months of approval
Feb. 7, 2025 – aztreonam/avibactam (Emblaveo)
- 505(b)(2) NDA approval; Fast Track, Priority Review, Qualified Infectious Disease Product (QIDP); first monobactam/β-lactamase inhibitor combination antibiotic therapy FDA-approved to treat complicated intra-abdominal infections, including those caused by gram-negative bacteria
- Combination of aztreonam, a monobactam antibacterial, and avibactam, a beta-lactamase inhibitor
- Indicated in combination with metronidazole for adults who have limited or no alternative options for the treatment of complicated intra-abdominal infections (cIAI) including those caused by the following susceptible gram-negative microorganisms: Escherichia coli, Klebsiella pneumoniae, Klebsiella oxytoca, Enterobacter cloacae complex, Citrobacter freundii complex and Serratia marcescens
- Emblaveo should be used only to treat infections that are proven or strongly suspected to be caused by bacteria to reduce the development of drug-resistant bacteria
- For injection: 2 g (1.5 g aztreonam and 0.5 g avibactam) of lyophilized powder for reconstitution in a single-dose vial
- Recommended dosage is based on estimated creatinine clearance with a single IV infusion loading dose followed by maintenance IV infusions beginning at the next dosing interval (every six to 12 hours) for a duration of treatment of five to 14 days
- Product will be available from Abbvie in the third quarter of 2025 with pricing to follow
Jan. 28, 2025 – semaglutide (Ozempic)
- Novo Nordisk; glucagon-like peptide 1 receptor agonist (GLP-1 RA); first GLP-1 RA to decrease the risk of worsening kidney disease and cardiovascular death in adults with type 2 diabetes mellitus (T2DM) and chronic kidney disease (CKD)
- New indication: to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease and cardiovascular death in adults with T2DM and CKD
- Administered once weekly SC in the abdomen, thigh or upper arm at any time of day, with or without meals; patients or caregivers can administer following training from an HCP
- Other indications: (1) adjunct to diet and exercise to improve glycemic control in adults with T2DM; (2) to reduce the risk of major adverse cardiovascular events (MACE) in adults with T2DM and established cardiovascular disease
Feb. 7, 2025 – moxidectin (no trade name)
- Medicines Development for Global Health; anthelmintic
- Expanded indication: to include pediatric patients aged 4 years to < 12 years and weighing ≥ 13 kg for the treatment of onchocerciasis due to Onchocerca volvulus; previously, this indication was only approved for patients aged ≥ 12 years
- Administered as a single dose taken orally with or without food with the dose dependent on patient body weight for pediatric patients ≥ 4 years of age and weighing ≥ 13 kg
First generic drug launches
Amneal launched a generic to Abbvie’s Namzaric (10 mg/14 mg, 10 mg/21 mg, 10 mg/28 mg). This combination medication containing an NMDA receptor antagonist (memantine) and an acetylcholinesterase inhibitor (donepezil) is indicated for the treatment of moderate to severe dementia of the Alzheimer’s type in patients stabilized on 10 mg of donepezil HCl once daily. It is supplied as an extended-release oral capsule. In 2024, Namzaric generated $90 million in annual sales in the U.S.
Ricon has launched a generic to Baxter’s Mesnex (400 mg). This cytoprotective agent is indicated as a prophylactic agent in reducing the incidence of ifosfamide-induced hemorrhagic cystitis. It is administered as a fractionated dosing schedule by two oral administrations of mesna tablets that follow a single bolus injection of mesna repeated on each day that ifosfamide is administered. Oral Mesnex generated less than $10 million in annual U.S. sales in 2024.
COVID-19 coronavirus disease 2019
DMARD disease-modifying antirheumatic drugs
ECMO extracorporeal membrane oxygenation
NMDA N-methyl-D-aspartate
NSAID non-steroidal anti-inflammatory drug
RANK receptor activator of nuclear factor κB
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