Specialty

 

New Drugs 

 

Sept. 13, 2024 - lebrikizumab-lbkz (Ebglyss) 

The FDA has approved Eli Lilly’s interleukin (IL)-13 antagonist, Ebglyss, indicated for the treatment of adult and pediatric patients ≥ 12 years of age who weigh ≥ 40 kg with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. Ebglyss can be used with or without topical corticosteroids; topical calcineurin inhibitors can be used but reserved for sensitive areas only. The monoclonal antibody will be supplied in 250 mg/2 mL single-dose prefilled pens and prefilled syringes with needle shields. The recommended dosage is a loading dose given at week zero and week two, followed by every two-week dosing until week 16 or later, when adequate clinical response is achieved. This is followed by maintenance dosing given every four weeks. Ebglyss is administered subcutaneously (SC) and can be self-administered or caregiver-administered following training in SC injection technique. Approval was based on data from two identically designed, 52-week, randomized, double-blind, placebo-controlled, phase 3 trials (ADvocate 1 [n=424] and 2 [n=427]) that demonstrated a significantly greater proportion of patients in the Ebglyss arm achieved the primary endpoint of an Investigator’s Global Assessment (IGA) score of 0 or 1 (clear or almost clear skin; range, 0 to 4) with at least two point reduction from baseline at week 16 compared to the placebo arm (p<0.001 for both studies).¹ The ADhere study provided support of Ebglyss in combination with topical corticosteroids.² Ebglyss is expected to compete with other similar biologics such as dupilumab (Dupixent) and tralokinumab-ldrm (Adbry) which are also indicated for select patients with atopic dermatitis. Ebglyss is commercially available and carries an average wholesale price (AWP) of $4,200 per 2 mL prefilled pen.  

Sept. 20, 2024 - arimoclomol (Miplyffa) 

Zevra’s Miplyffa has received FDA approval for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients ≥ 2 years of age. This is the first treatment FDA-approved for NPC, a rare genetic condition leading to progressive neurological symptoms, organ dysfunction, and early death. Product will be supplied as oral capsules in the strengths of 47 mg, 62 mg, 93 mg, and 124 mg. The recommended dosage is based on actual body weight and is given three times daily. Approval was based on a randomized, double-blind, placebo-controlled, phase 2/3, 12-month trial (n=50) in patients 2 to 19 years of age and demonstrated that Miplyffa lead to slower disease progression (assessed by NPC Clinical Severity Scale score) compared to placebo (p=0.046), corresponding to a 65% reduction in annual disease progression.³ Miplyffa was reviewed under the Priority Review pathway and received Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA. Product availability is expected within eight to 12 weeks of approval with pricing to follow. 

New Formulations 

 

Aug. 26, 2024 - bortezomib (no trade name) 

• 505(b)(2) New Drug Application (NDA) approval; Standard Review; ready-to-use for SC administration, requires dilution for intravenous (IV) administration 

• Proteasome inhibitor 

• Indicated for (1) treatment of adults with multiple myeloma; and (2) treatment of adults with mantle cell lymphoma 

• Solution for injection: 3.5 mg/1.4 mL (2.5 mg/mL) in a single-dose vial 

• Administered either as a three to five second bolus IV injection or SC injection; SC and IV routes of administration have different final concentrations  

• Product availability is expected from Shilpa Medicare Limited with launch timeframe to be determined (TBD) and pricing to follow 
   

Aug. 30, 2024 - letermovir (Prevymis) 

• 505(b) NDA approval; Orphan Drug, Priority Review; new oral pellet formulation (already available as oral tablets and IV solution); FDA also approved expanded indications  

• Cytomegalovirus (CMV) deoxyribonucleic acid (DNA) terminase complex inhibitor 

• Expanded indications for (1) prophylaxis of CMV infection and disease in CMV-seropositive recipients of an allogeneic hematopoietic stem cell transplant to include pediatric patients aged ≥ 6 months and weighing ≥ 6 kg and for (2) prophylaxis of CMV disease in kidney transplant recipients at high risk (CMV seropositive donor/CMV seronegative recipient) to include pediatric patients aged ≥ 12 years and weighing ≥ 40 kg; previously, both indications were approved for use in adults only 

• Oral pellets: 20 mg or 120 mg per packet (contents given orally mixed with soft food or via nasogastric or gastric tube) 

• Taken once daily (orally as a tablet or pellet or as an IV infusion); duration of therapy is 200 days post kidney transplant and for 100 days, which can be extended to 200 days, post stem cell transplant 

• Availability of the oral pellet formulation is expected from Merck with launch timeframe TBD and pricing to follow 
   

Sept. 12, 2024 - atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza)  

• Biologics License Application (BLA) approval; Standard Review; first SC programmed death-ligand 1 (PD-L1) cancer immunotherapy; utilizes Enhanze drug delivery technology to allow for rapid dispersal and absorption 

• Combination of atezolizumab (a PD-L1 blocking antibody) + hyaluronidase (an endoglycosidase) 

• Indicated for all IV indications of Tecentriq (e.g., select adult patients with non-small cell lung cancer [NSCLC], small cell lung cancer [SCLC], hepatocellular carcinoma, melanoma, and alveolar soft part sarcoma) 

• Solution for injection: 1,875 mg atezolizumab and 30,000 units hyaluronidase per 15 mL (125 mg/2,000 units per mL) in a single-dose vial 

• SC injection over approximately 7 minutes every three weeks in the thigh by a health care professional (HCP); different recommended dose and administration than IV Tecentriq which requires a 30-to-60-minute IV infusion 

• Product is available from Genentech with an AWP of $13.108.91 per 15 mL vial 
   

Sept. 13, 2024 - ocrelizumab and hyaluronidase-ocsq (Ocrevus Zunovo) 

• BLA approval; SC formulation of ocrelizumab (Ocrevus) administered twice per year; utilizes Enhanze drug delivery technology to allow for rapid dispersal and absorption 

• Combination of ocrelizumab (a CD20-directed cytolytic antibody) + hyaluronidase (an endoglycosidase) 

• Indicated for treatment of (1) relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults; and (2) primary progressive MS, in adults 

• Solution for injection: 920 mg of ocrelizumab and 23,000 units hyaluronidase per 23 mL (40 mg and 1,000 units per mL) in a single-dose vial 

• SC injection over approximately 10 minutes every six months into the abdomen by an HCP; patients are required to be monitored closely during all injections and for a minimum of one hour after the initial injection and for a minimum of 15 minutes after subsequent injections; different recommended dose and administration than IV Ocrevus which requires a 2 to 3.5 hours or longer IV infusion 

• Product will be available from Genentech with launch timeframe TBD and pricing to follow 

 

New Biosimilars 

 

Aug. 23, 2024 - aflibercept-ayyh (Pavblu) 

The FDA has approved Amgen’s Pavblu as a biosimilar to Regeneron’s Eylea (aflibercept). This vascular endothelial growth factor (VEGF) inhibitor is approved for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), and diabetic retinopathy; it is not approved for the indication of retinopathy of prematurity. Pavblu is administered via intravitreal injection and will be available in the same presentations as reference product Eylea, including a 2 mg (0.05 mL of 40 mg/mL) solution in a single-dose prefilled syringe and single-dose vial. Pavblu is expected to compete with the other Eylea biosimilars: aflibercept-yszy (Opuviz), aflibercept-jbvf (Yesafili), aflibercept-mrbb (Ahzantive), and aflibercept-abzv (Enzeevu). Opuviz and Yesafili have been deemed interchangeable with Eylea, while Enzeevu has been provisionally determined to be interchangeable with the reference product as it is subject to an unexpired exclusivity for the first interchangeable biosimilar products. None of the Eylea biosimilars have launched, but depending on patent litigation an Eylea biosimilar may launch “at risk” starting sometime in 2024. 
 

New or Expanded Indications 

 

Sept. 11, 2024 - guselkumab (Tremfya)  

• Janssen Biotech; IL-23 antagonist administered SC or IV  

• New indication: treatment of adults with moderately to severely active ulcerative colitis (UC)  

• Administered as an IV induction dose at weeks zero, four, and eight by an HCP followed by maintenance dosing administered SC at week 16 and every eight weeks thereafter, or a higher SC dose administered at week 12 and every four weeks thereafter (the lowest effective dose should be used); maintenance doses can be administered by a patient or a caregiver  

• Other indications: select adults with plaque psoriasis or active psoriatic arthritis  
   

Sept. 12, 2024 - dupilumab (Dupixent) 

• Regeneron; IL-4 receptor alpha antagonist administered SC; Priority Review for this expanded patient population 

• Expanded indication: add-on maintenance treatment of inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP) to include pediatric patients ≥ 12 years of age; previously, this indication was only approved for adults  

• Administered as SC injection every other week (dosage for pediatric patients ≥ 12 years of age is the same as for adults); can be administered by a patient or a caregiver; in pediatric patients 12 to 17 years of age, administer under the supervision of an adult 

• Other indications: select patients with atopic dermatitis, asthma, eosinophilic esophagitis, and prurigo nodularis 
   

Sept. 13, 2024 - certolizumab pegol (Cimzia) 

• UCB; tumor necrosis factor (TNF) blocker administered SC 

• New indication: treatment of active polyarticular juvenile idiopathic arthritis (pJIA) for patients ≥ 2 years of age  

• Administered as a SC weight-based dose beginning with a loading dose given at week zero, two, and four followed by maintenance doses beginning at week six and given every two weeks thereafter; doses range from 50 mg every two weeks to 400 mg loading doses; as there is not a dosage form that allows for self-administration of doses less than 200 mg, doses less than 200 mg require HCP administration using the vial kit; patient or caregiver (depending on patient age) can administer using the prefilled syringe 

• Other indications: select patients with Crohn's disease, rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, non-radiographic axial spondylarthritis, plaque psoriasis 
   

Sept. 16, 2024 - lanreotide (no trade name) 

• Cipla; somatostatin analog administered SC; now shares all the same indications as lanreotide (Somatuline Depot) 

• New indication: treatment of adults with carcinoid syndrome; when used, it reduces the frequency of short-acting somatostatin analog rescue therapy 

• Administered by deep SC injection every four weeks by an HCP; if patients are already being treated with lanreotide for gastroenteropancreatic neuroendocrine tumors (GEP-NETs), do not administer an additional dose for treatment of carcinoid syndrome  

• Other indications: (1) long-term treatment of select acromegalic patients and (2) treatment of select adults with GEP-NETs 
   

Sept. 17, 2024 - benralizumab (Fasenra)  

• AstraZeneca; IL-5 receptor alpha-directed cytolytic monoclonal antibody administered SC; Orphan Drug designation for new indication 

• New indication: treatment of adults with eosinophilic granulomatosis with polyangiitis (EGPA)  

• Administered as a SC injection every four weeks; monitoring of patients after administration is recommended; prefilled syringe administration requires an HCP; administration with the pen can be done by patients or caregivers following proper training 

• Other indication: add-on maintenance treatment for adults and pediatric patients ≥ 6 years of age with severe asthma and with an eosinophilic phenotype 
   

Sept. 17, 2024 - pembrolizumab (Keytruda) 

• Merck; programmed death receptor-1 (PD-1)-blocking antibody administered IV; Assessment Aid, Priority Review for this indication 

• New indication: in combination with pemetrexed and platinum chemotherapy as first-line treatment of adults with unresectable advanced or metastatic malignant pleural mesothelioma (MPM) 

• Administered as an IV infusion over 30 minutes, for MPM given as 200 mg every three weeks or 400 mg every six weeks 

• Other indications: numerous select patients with various cancers (e.g., melanoma, lung cancer, head and neck squamous cell cancer, Hodgkin lymphoma, large B-cell lymphoma, urothelial cancer, microsatellite instability-high [MSI-H] or mismatch repair deficient [dMMR] cancer, MSI-H or dMMR colorectal cancer (CRC), gastric cancer, esophageal cancer, cervical cancer, hepatocellular carcinoma, biliary tract cancer, and others)    
   

Sept. 17, 2024 - ribociclib (Kisqali) 

• Novartis; kinase inhibitor supplied as oral tablets; new indication was reviewed with the Assessment Aid pathway 

• New indication: in combination with an aromatase inhibitor (AI) for adjuvant treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative stage II and III early breast cancer at high risk of recurrence 

• Administered orally at a starting dose of 400 mg orally (two 200 mg tablets) once daily with or without food for 21 consecutive days followed by seven days off treatment 

• Other indications: treatment of adults with HR-positive, HER2-negative advanced or metastatic breast cancer in combination with (1) an AI as initial endocrine-based therapy or (2) fulvestrant as initial endocrine-based therapy or following disease progression on endocrine therapy 

• Ribociclib + letrozole co-pack (Kisqali Femara Co-Pack) also received approval for the same new indication 
   

Sept. 19, 2024 - amivantamab-vmjw (Rybrevant) 

• Janssen Biotech; bispecific epidermal growth factor receptor (EGFR)-directed and MET receptor directed antibody administered IV; reviewed under Project Orbis and utilized Assessment Aid for new indication 

• New indication: in combination with carboplatin and pemetrexed for adults with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations whose disease has progressed on or after treatment with an EGFR tyrosine kinase inhibitor 

• Administered in combination with chemotherapy weekly for four weeks, with the initial dose in week one as a split infusion over day one and day two (weeks two to four  – infusion on day one of each week; weeks five and six – no dose), then administer every three weeks starting at week seven (dosage is based on body weight); when given in combination with carboplatin and pemetrexed, infuse pemetrexed first, carboplatin second, and Rybrevant last  

• Other indications: select patients with NSCLC 

 

Traditional 

 

New Drugs 

None 
 

New Formulations 

None 
 

New or Expanded Indications 

 

Aug. 29, 2024 - smallpox and mpox (vaccinia) vaccine, live (ACAM2000)  

• Emergent BioSolutions; vaccine for active immunization  

• New indication: prevention of mpox disease in individuals determined to be at high risk for mpox infection 

• Administered as a single dose by percutaneous scarification (skin is pricked several time using a bifurcated needle that has a droplet of vaccine solution) by an HCP; formation of a major cutaneous reaction (pustule) by day six to 11 is evidence of a successful immunization; if a major cutaneous reaction does not occur, the vaccination procedure should be checked and the patient may be revaccinated using another vial or vaccine lot; upon revaccination, the patient may experience a cutaneous reaction, but the absence of one does not necessarily indicate vaccination failure 

• Other indication: prevention of smallpox disease in individuals determined to be at high risk for smallpox infection 

 

First Generic Drug Launches 

Oxcarbazepine extended release (ER) tablet (Oxtellar XR): Apotex launched a generic to Supernus’ Oxtellar XR tablets (150 mg, 300 mg, 600 mg). This drug is indicated for treatment of partial-onset seizures in patients at least six years of age. It is dosed as a single daily dose taken on an empty stomach. Oxtellar XR generated $218 million in annual U.S. sales in 2023.  

Dasatinib tablet (Sprycel): Apotex launched a generic to Bristol Myers Squibb’s Sprycel tablets (20 mg, 50 mg, 70 mg, 80 mg, 100 mg, and 140 mg). This kinase inhibitor is indicated for select adult and pediatric patients with chronic myeloid leukemia (CML) or acute lymphoblastic leukemia (ALL). Dasatinib is taken orally once daily. In 2023, Sprycel generated $2,532 million in annual sales in the U.S.  

Tazarotene cream (Tazorac): Padagis Israel launched a generic to Allergan/Allmirall’s Tazorac cream (0.05% strength). Tazarotene cream is a retinoid indicated for the topical treatment of plaque psoriasis and is applied as a thin layer to the affected area once daily in the evening. Tazorac cream generated $15 million (including both 0.05% and 0.1% strengths) in 2023 in sales.