Drug Approvals: July 2024 - Prime Therapeutics
Drug Approvals: July 2024
Monthly update on newly approved specialty and traditional drugs, new indications and first-time generic launches
Specialty
New drugs
Jun. 20, 2024 – Piasky™ (crovalimab-akkz)
The FDA has approved Genentech’s complement C5 inhibitor, Piasky™, for the treatment of adult and pediatric patients ≥ 13 years of age with paroxysmal nocturnal hemoglobinuria (PNH) and a body weight of ≥ 40 kg. Dosing is based on the patient's actual body weight and is initiated with one loading dose via intravenous (IV) infusion, followed by four additional loading doses administered by subcutaneous (SC) injection. Maintenance doses are administered every 4 weeks SC. Piasky™ requires health care provider (HCP) administration. The open-label, phase 3 COMMODORE 2 trial (n=204) showed that in adult patients with PNH who were complement inhibitor naïve, Piasky™ was non-inferior to eculizumab in the co-primary endpoints of hemolysis control (odds ratio [OR], 1.02; 95% confidence interval [CI], 0.57 to 1.82) and transfusion avoidance (difference in proportion of patients with transfusion avoidance, -2.8%; 95% CI, -15.7 to 11.1).¹ Similar findings were observed in adolescent patients in the COMMODORE 1, 2, and 3 studies. Piasky™ received Orphan Drug designation and is expected to compete with other agents indicated for PNH: eculizumab (Soliris®), iptacopan (Fabhalta®), pegcetacoplan (Empaveli®), and ravulizumab-cwvz (Ultomiris®). Genentech launched Piasky™ in July 2024 with an average wholesale price (AWP) of $21,228 per 2 mL single-dose vial. Piasky™ is only available through the Piasky™ Risk Evaluation and Mitigation Strategy (REMS) program due to the risk of serious meningococcal infections.
Jun. 25, 2024 – Tepylute (thiotepa)
The FDA approved Tepylute, by Shorla Oncology, under a 505(b)(2) new drug application (NDA) for the treatment of adenocarcinoma of the breast or ovary. The alkylating agent will be supplied as 15 mg/1.5 mL (10 mg/mL) of solution in a single-dose vial for weight-based dosing administered IV given at 1- to 4-week intervals. Maintenance dosing is adjusted weekly based on pretreatment and subsequent blood counts and should not be given more often than once weekly. Tepylute provides a ready-for-dilution formulation of thiotepa that does not require reconstitution. Launch timeframe is to be determined, with pricing to follow.
Jun. 26, 2024 – Ohtuvayre™ (ensifentrine)
The FDA approved the first-in-class selective dual inhibitor of phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4), Ohtuvayre™, for the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adults. The aqueous suspension is supplied in unit-dose ampules and is administered twice daily via oral inhalation using a standard jet nebulizer with a mouthpiece. In the double-blind, phase 3 ENHANCE-1 (n=760) and ENHANCE-2 (n=789) trials, treatment with Ohtuvayre™ significantly improved the average forced expiratory volume in one second (FEV₁) area under the curve at 0 to 12 hours from baseline compared to placebo (ENHANCE-1: 87 mL; ENHANCE-2: 94 mL; p<0.001 for both) at week 12.² Ohtuvayre™ provides both bronchodilator and non-steroidal anti-inflammatory effects in one molecule and was studied in patients not receiving long-acting maintenance therapy or who were receiving a long-acting β₂-agonist (LABA) or long-acting muscarinic antagonist (LAMA) with or without an inhaled corticosteroid (ICS). Ohtuvayre™ may compete with other maintenance therapy medications (e.g., LAMAs, LABAs, ICS) in the COPD space. According to its manufacturer, Verona, Ohtuvayre™ is expected to be available in the third quarter of 2024 through certain accredited specialty pharmacies at an AWP of $59 per 3 mg/2.5 mL ampule.
Jul. 2, 2024 – Kisunla™ (donanemab-azbt)
Eli Lilly has received FDA approval for the amyloid beta-directed antibody, Kisunla™, for the treatment of Alzheimer’s disease. The presence of amyloid beta pathology should be confirmed before starting Kisunla™, and the therapy should only be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in the clinical trials. The monoclonal antibody is supplied as a 350 mg/20 mL (17.5 mg/mL) solution in a single-dose vial and is given every four weeks as an IV infusion over approximately 30 minutes. On amyloid PET imaging, if a reduction in amyloid plaques to minimal levels is observed, consideration should be given to stopping therapy with Kisunla™. In the randomized, double-blind, placebo-controlled, phase 3 TRAILBLAZER-ALZ 2 study (n=1,736) evaluating patients with early symptomatic Alzheimer’s disease and amyloid and tau pathology, statistically significant improvements with donanemab compared to placebo were observed in the primary endpoint (integrated Alzheimer Disease Rating Scale score) from baseline to 76 weeks for patients with low/medium tau pathology (difference, 3.25; p<0.001) and in the combined population (low/medium + high tau pathology) (difference, 2.92; p<0.001).³ Kisunla™ was reviewed under Priority Review and received Breakthrough Therapy and Fast Track designations. Kisunla™ will be one of two products on the market in this class of amyloid beta-directed antibodies that has received FDA approval for the treatment of Alzheimer’s disease. The other agent which shares its indication is lecanemab-irmb (Leqembi®). These amyloid beta-directed antibodies carry a black box warning for amyloid related imaging abnormalities (ARIA) and should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, as the agents were studied in this population. Kisunla™ is commercially available with an AWP of $834.78 per 350 mg/20 mL vial.
New biosimilars
Jun. 28, 2024 – Ahzantive™ (aflibercept-mrbb)
Formycon received FDA approval for Ahzantive™ as a biosimilar to Regeneron’s Eylea® (aflibercept). Ahzantive™ is a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of patients with: (1) neovascular (wet) age-related macular degeneration (AMD), (2) macular edema following retinal vein occlusion (RVO), (3) diabetic macular edema (DME), and (4) diabetic retinopathy (DR). Ahzantive™ is approved as a 0.05 mL of 40 mg/mL solution in a single-dose vial and is administered via intravitreal injection. Reference product Eylea® carries an additional indication for retinopathy of prematurity and is also supplied in a single-dose pre-filled syringe in addition to the single-dose vial presentation. Ahzantive™ is expected to compete with the other Eylea® biosimilars, aflibercept-yszy (Opuviz™) and aflibercept-jbvf (Yesafili™), both of which have been deemed interchangeable with the reference drug. Launch timeframe is to be determined, with pricing to follow.
Jun. 28, 2024 – Nypozi™ (filgrastim-txid)
Tanvex BioPharma has received FDA approval for Nypozi™, their filgrastim (Neupogen®) biosimilar. The leukocyte growth factor will be supplied in single-dose prefilled syringes in the strengths of 300 mcg/0.5 mL and 480 mcg/0.8 mL for IV or SC use. Nypozi™ has been approved for all the indications of reference product Neupogen®, as listed in the product label (e.g., select patients with chemotherapy-induced neutropenia, severe chronic neutropenia, peripheral blood stem cell mobilization, or radiation exposure). Direct administration of Nypozi™ in doses < 0.3 mL (180 mcg) is not recommended due to the potential for dosing errors. Nypozi™ received Orphan Drug designation and is the fourth biosimilar to Neupogen®. Launch timeframe for Nypozi™, as well as pricing information is forthcoming.
Jun. 28, 2024 – Pyzchiva® (ustekinumab-ttwe)
The FDA has approved a new ustekinumab (Stelara®) biosimilar, Pyzchiva®, from Sandoz/Samsung Bioepis. Pyzchiva® has received provisional interchangeability determination from the FDA based on an unexpired period of exclusivity for the first interchangeable biosimilar biological products. Approval was granted for all indications of the reference product: patients ≥ 6 years of age with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy, patients ≥ 6 years of age with active psoriatic arthritis, and adults with moderately to severely active Crohn's disease or moderately to severely active ulcerative colitis. The human interleukin-12 and -23 antagonist will be supplied in a single-dose prefilled syringe in the strengths of 45 mg/0.5 mL and 90 mg/mL solution for SC injection and in a 130 mg/26 mL (5 mg/mL) solution in a single-dose vial for IV infusion. Pyzchiva® is the third Stelara® biosimilar to receive FDA-approval. Other FDA-approved biosimilars to Stelara® include: ustekinumab-aekn (Selarsdi™) and ustekinumab-auub (Wezlana™), the latter of which has received interchangeability designation. Pyzchiva® is anticipated to be available in February 2025; pricing information is forthcoming.
New or expanded indications
Jun. 20, 2024 – Elevidys® (delandistrogene moxeparvovec-rokl)
The FDA has granted traditional approval to Sarepta’s adeno-associated virus vector-based gene therapy, Elevidys®, for use in ambulatory patients ≥ 4 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Additionally, the FDA has granted Accelerated Approval for use in non-ambulatory patients ≥ 4 years of age with DMD with a confirmed mutation in the DMD gene based on expression of Elevidys® micro-dystrophin in skeletal muscle. Elevidys® was previously approved under Accelerated Approval in June 2023 for ambulatory patients between the ages of 4 and 5 years with DMD with a confirmed DMD gene mutation. This gene therapy is administered as a single-dose IV infusion for both indications, with dosing based on body weight.
Jun. 21, 2024 – Krazati® (adagrasib)
Mirati’s RAS GTPase inhibitor, Krazati®, has been granted Accelerated Approval for a new indication for use in combination with cetuximab for treatment of adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC), as determined by an FDA-approved test, who have received prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. The application was reviewed under Priority Review and was granted Breakthrough Therapy designation. Krazati® was already approved for use as a single agent for treatment of adults with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. Krazati® is dosed orally twice daily until disease progression or unacceptable toxicity.
Jun. 21, 2024 – Vyvgart® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)
The FDA has granted traditional approval to Sarepta’s adeno-associated virus vector-based gene therapy, Elevidys®, for use in ambulatory patients ≥ 4 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Additionally, the FDA has granted Accelerated Approval for use in non-ambulatory patients ≥ 4 years of age with DMD with a confirmed mutation in the DMD gene based on expression of Elevidys® micro-dystrophin in skeletal muscle. Elevidys® was previously approved under Accelerated Approval in June 2023 for ambulatory patients between the ages of 4 and 5 years with DMD with a confirmed DMD gene mutation. This gene therapy is administered as a single-dose IV infusion for both indications, with dosing based on body weight.
Jun. 26, 2024 – Epkinly™ (epcoritamab-bysp)
The FDA has granted Accelerated Approval to Genmab’s bispecific CD20-directed CD3 T-cell engager, Epkinly™, for the treatment of adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. The application was reviewed under Priority Review and was granted Breakthrough Therapy designation. Epkinly™ is also approved for treatment of select adult patients with diffuse large B-cell lymphoma or high-grade B-cell lymphoma. This product is administered via SC injection in 28-day cycles, and a three step-up dosing schedule should be followed for treatment of FL. This approval makes Epkinly™ the first and only T-cell engaging bispecific antibody with SC administration for this patient population.
Traditional
New drugs
Jun. 18, 2024 – Sofdra™ (sofpironium)
The FDA approved Sofdra™ topical gel, by Botanix, for the treatment of primary axillary hyperhidrosis in adults and pediatric patients ≥ 9 years of age. This topical anticholinergic will be supplied as 12.45% of sofpironium in a 50 mL bottle with a metered dose pump and applicator. The gel is applied to clean, dry skin of the underarm once daily at bedtime. Two randomized, vehicle-controlled, phase 3 studies (CARDIGAN 1 and 2) (n=701) demonstrated Sofdra™ resulted in statistically meaningful improvements compared to vehicle in the co-primary endpoints assessing the change from baseline in gravimetric sweat production and Hyperhidrosis Disease Severity Measure-Axillary score at day 43.⁴ Sofdra™ provides a new chemical entity for this indication and is expected to compete with glycopyrronium (Qbrexza®), a topical 2.4% cloth, which shares its indication. Early access to Sofdra™ is expected in the third quarter of 2024 through a patient experience program, with broader launch occurring early in the fourth quarter of 2024. The AWP for one pump bottle of Sofdra™ is $1,161.
Jun. 28, 2024 – Chewtadzy™ (tadalafil chewable tablet)
The FDA has granted approval to Ani’s phosphodiesterase 5 (PDE5) inhibitor Chewtadzy™ for the treatment of (1) erectile dysfunction (ED); (2) the signs and symptoms of benign prostatic hyperplasia (BPH); and (3) ED and the signs and symptoms of BPH (ED/BPH). Chewtadzy™ was approved under the 505(b)(2) NDA approval pathway and will be supplied as chewable tablets in the strengths of 5 mg, 10 mg, and 20 mg. The medication is not approved for once daily use for ED as the recommended dosage for this indication cannot be achieved with the lowest tablet strength. Chewtadzy™ tablets should be chewed completely before swallowing. Chewtadzy™ is expected to compete with other formulations of tadalafil, including generic oral tablets which carry the same FDA-approved uses. Launch timeframe is to be determined, with pricing to follow.
New or expanded indications
Jun. 21, 2024 – Wakix® (pitolisant)
Wakix®, from Harmony Biosciences, has received a new indication for the treatment of excessive daytime sleepiness (EDS) in patients ≥ 6 years of age with narcolepsy, making it the first and only FDA-approved non-scheduled therapy option for this indication. The application was granted Priority Review. Previously, this histamine-3 (H3) receptor antagonist/inverse agonist was only indicated for the treatment of EDS or cataplexy in adults with narcolepsy. Wakix® is administered orally once daily in the morning upon wakening, and the initial dosing is based on a titration schedule. The maximum dosage for pediatric patients is based on body weight.
Jul. 01, 2024 – Velphoro® (sucroferric oxyhydroxide)
The FDA has expanded the indication of Fresenius’ Velphoro® to control serum phosphorus levels in patients with chronic kidney disease (CKD) on dialysis to include patients ≥ 9 years of age. This phosphate binder was previously only approved for use in adults. Velphoro® is administered orally with a twice daily starting dose for patients between 9 to 11 years of age and three times daily starting dose for patients ≥ 12 years of age. This product is available as a chewable tablet that should not be swallowed whole and must be administered with meals.
Jul. 09, 2024 – Zoryve® (roflumilast)
Zoryve® cream from Arcutis has been approved by the FDA as a 0.15% cream for the topical treatment of mild to moderate atopic dermatitis in adults and pediatric patients ≥ 6 years of age. This PDE4 inhibitor is also available as a 0.3% cream for the topical treatment of plaque psoriasis in adults and pediatric patients ≥ 6 years of age, as well as a 0.3% foam for the topical treatment of seborrheic dermatitis in adults and pediatric patients ≥ 9 years of age. For the new atopic dermatitis indication, Zoryve® 0.15% cream should be applied once daily to the affected areas. The new strength will be available as a 60-gram tube containing 1.5 mg of roflumilast per gram.
First generic drug launches
Radicava® (edaravone injection): Gland launched a generic to Mitsubishi Tanabe’s Radicava® 30 mg/100 mL and 60 mg/100 mL injection for IV use. This drug is indicated for the treatment of amyotrophic lateral sclerosis (ALS) and is administered daily for 14 days during the initial treatment cycle, followed by a 14-day drug-free period. Additional treatment cycles are comprised of daily dosing for 10 days out of each 14-day cycle, followed by 14-day drug-free periods. Radicava® generated $23 million in annual U.S. sales in 2023.
Victoza® (liraglutide injection): Teva has launched an authorized generic (AG) of Novo Nordisk’s Victoza® 18 mg/3 mL pen, a once-daily SC glucagon-like peptide-1 (GLP-1) receptor agonist indicated as an adjunct to diet and exercise to improve glycemic control in adults and pediatric patients ≥ 10 years of age with type 2 diabetes mellitus (T2DM) and to reduce the risk of major adverse cardiovascular events (MACE) in adults with T2DM and established cardiovascular disease. Victoza® generated $3,314 million in annual U.S. sales in 2023. Generic Victoza® is the first generic GLP-1 receptor agonist to be launched in the U.S.
Halog® (halcinonide topical solution): A first generic for Sun’s Halog® 0.1% topical solution has launched from Encube. This topical corticosteroid solution is indicated for the relief of the inflammatory and pruritic manifestations of corticosteroid-responsive dermatoses and is applied to the affected areas two to three times daily.
Corlanor® (ivabradine tablet): A generic for Amgen’s Corlanor® 5 mg and 7.5 mg tablets has launched from Ingenus. This hyperpolarization-activated cyclic nucleotide-gated channel blocker is indicated to reduce the risk of hospitalization for worsening heart failure (HF) in adults with stable, symptomatic chronic HF with reduced left ventricular ejection fraction. Taken orally twice daily with food, ivabradine should be dose-adjusted based on heart rate. In 2023, Corlanor® generated $153 million in annual sales in the U.S.
Endari® (l-glutamine oral powder): Novitium has launched a first generic to Emmaus’ Endari® 5-gram per packet oral powder. This amino acid product is approved to reduce the acute complications of sickle cell disease in patients ≥ 5 years of age and is dosed orally twice daily. Endari® produced $25 million in annual U.S. sales in 2023.
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COMMODORE 2 trial: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10428346/
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ENHANCE trials: https://www.atsjournals.org/doi/10.1164/rccm.202306-0944OC
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TRAILBLAZER-ALZ 2: https://jamanetwork.com/journals/jama/article-abstract/2807533