Drug Approvals Monthly Update: February 2024

February 28, 2024

This monthly approval update provides a review of newly approved specialty drugs, recent drug launches, generic updates, new indications and news of note on drugs in the approval process.

Specialty

New drugs

2/16/2024 Amtagvi™ (lifileucel)

The United States (U.S.) Food and Drug Administration (FDA) approved the first-in-class tumor-derived autologous T cell immunotherapy, Amtagvi, by Iovance. Amtagvi is indicated for the treatment of adults with unresectable or metastatic melanoma that has been previously treated with a programmed cell death protein 1 (PD-1) blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without an MEK inhibitor. It is manufactured from T cells collected from the patient’s tumor tissue and expanded ex vivo. The processed T cells (Amtagvi) are administered as a one-time, single intravenous (IV) infusion. The Amtagvi treatment regimen, which includes lymphodepleting chemotherapy and an interleukin-2 agent (aldesleukin) to support in vivo cell expansion, will be administered at authorized treatment centers. Treatment with Amtagvi was evaluated in an open-label, single-arm trial.1,2 Based on data from 73 patient who received the recommended dose (7.5 x 109 to 72 x 109 viable cells), Amtagvi led to an objective response rate (ORR) (primary endpoint) of 31.5%, with a 3% complete response rate. The median time to initial response was 1.5 months and the median duration of response (DOR) was not reached. Amtagvi carries a boxed warning for treatment-related mortality, prolonged severe cytopenia, severe infection, and cardiopulmonary and renal impairment. The autologous therapy was granted Accelerated Approval based on the ORR, and continued approval may depend on results from confirmatory trials. Amtagvi was also granted a Priority Review as well as Fast Track, Orphan Drug and Regenerative Medicine Advanced Therapy designations. The timing for launch has not been announced. A wholesale acquisition cost (WAC) of $515,000 per patient is expected.

New indications

1/25/2024 Dupixent® (dupilumab)

The FDA expanded the indication for Dupixent for eosinophilic esophagitis (EoE) to include patients 1 to 11 years of age, weighing at least 15 kg. Regeneron’s interleukin-4 receptor alpha antagonist is administered via subcutaneous (SC) injection every other week. A clinical study in 61 patients 1 to 11 years of age and pharmacokinetic data in adult and pediatric subjects 1 to 17 years of age demonstrated that the safety profile of Dupixent was similar in pediatric and adult patients.³

1/26/2024 Gammagard Liquid® (immune globulin infusion [human])

Takeda gained FDA approval for Gammagard Liquid for improving neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). Notably, safety and effectiveness has not been studied in immunoglobulin-naïve patients with CIDP, and maintenance therapy in CIDP has not been evaluated beyond 6 months. For CIDP, Gammagard is only administered IV by an healthcare professional as an induction dose over 2 to 5 consecutive days, followed by maintenance treatment over 1 to 4 consecutive days, which is repeated every 3 weeks. Approval for CIDP was supported by the Epoch 2 study, which reported that 94.4% of patients achieved at least a 1 point decrease in the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (primary endpoint) at 6 months of treatment with Gammagard Liquid.4

2/13/2024 Onivyde® (irinotecan liposomal)

The FDA approved Ipsen’s 505(b)(2) new drug application (NDA) for Onivyde, a topoisomerase inhibitor, for use in combination with oxaliplatin, 5-fluorouracil (5-FU) and leucovorin, for the first-line treatment of adults with metastatic pancreatic adenocarcinoma. Onivyde is administered via IV infusion every 2 weeks. Approval for first-line use is based on the open-label NAPOLI 3 trial that reported a median overall survival of 11.1 months with Onivyde + oxilaplatin + 5-FU + leucovorin compared to 9.2 months with gemcitabine + Nab paclitaxel–the difference was statistically significant.5 In addition, the median progression-free survival (PFS) was 7.4 months and 5.6 months in each group, respectively (hazard ratio [HR], 0.7). Onivyde received an Orphan Drug designation for the treatment of pancreatic cancer, as well as a Fast Track designation for its use in the first-line setting.

2/15/2024 Tepmetko® (tepotinib)

EMD Serono received a traditional approval for their kinase inhibitor, Tepmetko, for the treatment of adults with metastatic non-small cell lung cancer (NSCLC) harboring MET exon 14 skipping alterations. The conversion from Accelerated Approval to a traditional approval was based on data from an additional 161 patients and an added 28 months of follow-up.6 Among 164 treatment-naïve patients, the ORR was 57% (95% confidence interval [CI], 49 to 65), with 40% of responders having a DOR ≥ 12 months. Among 149 treatment-experienced patients, the ORR was 45% (95% CI, 37 to 53), with 36% of responders having a DOR ≥ 12 months.

2/16/2024 Tagrisso® (osimertinib)

Tagrisso, a kinase inhibitor by AstraZeneca, was approved for use with pemetrexed and platinum-based chemotherapy for patients with locally advanced or metastatic NSCLC whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations. Approval was based on the open-label FLAURA 2 trial that demonstrated a statistically significant improvement in PFS when patients were treated with Tagrisso plus platinum-based chemotherapy compared to Tagrisso monotherapy (median PFS, 25.5 versus 16.7 months, respectively; HR, 0.62; 95% CI, 0.49 to 0.79; p<0.0001).7 Tagrisso was granted a Priority Review as well as Fast Track, Orphan Drug and Regenerative Medicine Advanced Therapy designations for this application.

2/16/2024 Xolair® (omalizumab)

The FDA approved Genentech’s anti- immunoglobulin E (IgE) antibody, Xolair, for the reduction of allergic reactions, including anaphylaxis, that may occur with accidental exposure to one or more foods in adult and pediatric patients aged 1 year and older with IgE-mediated food allergy. Xolair is to be used in conjunction with food allergen avoidance. This approval was supported by results from the randomized, double-blind, OUtMATCH trial that evaluated Xolair in 168 patients with allergies to peanuts and at least two other foods (e.g., milk, egg, wheat, cashew, hazelnut, walnut).8 A statistically significantly higher proportion of patients treated with Xolair for 16 to 20 weeks tolerated ≥ 600 mg of peanut protein without moderate to severe allergic symptoms compared to those who were given placebo (68% versus 5%, respectively; p<0.0001). In addition, statistically significantly more patients treated with Xolair compared to placebo (respectively) tolerated ≥ 1,000 mg of protein from milk (66% versus 11%; p<0.0001), egg (67% versus 0%; p<0.0001) or cashew (42% versus 3%; p<0.0001) without experiencing moderate to severe allergic symptoms. Xolair received a Priority Review and Breakthrough Therapy designation for this indication.

 

Traditional

New drugs

2/09/2024 Eohilia™ (budesonide)

The FDA approved the 505(b)(2) NDA for Eohilia, by Takeda, for a 12-week treatment course in adult and pediatric patients 11 years of age and older with EoE; safety and efficacy of treatment beyond 12 weeks have not been established. Eohilia is the first corticosteroid that is approved for EoE; however, others have been used off-label. Eohilia is a muco-adherent topically active viscous budesonide oral formulation that is taken orally twice daily. Initially given a Complete Response Letter (CRL) requiring an additional study, the resubmission and subsequent approval was supported by two randomized, double-blind, placebo-controlled 12-week studies that evaluated Eohilia in patients at least 11 years of age with EoE.9 In both studies significantly more patients given Eohilia achieved histologic remission (based on eosinophil count) compared to placebo (Study 1: 53.1% versus 1%, respectively; Study 2: 38% versus 2.4%, respectively). In addition, during the last 2 weeks of each study, more patients in the Eohilia group reported no dysphagia or improved dysphagia compared to the placebo group. The most common treatment-related adverse events reported in Study 1 were respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%) and headache (5%); similar was reported in Study 2. Eohilia was granted Breakthrough Therapy and Orphan Drug designations as well as a Priority Review. Eohilia became available in February 2024, with a wholesale acquisition cost of $1,875 per month.

2/13/2024 Aurlumyn™ (iloprost)

After granting a Priority Review of the 505(b)(2) NDA, the FDA approved Aurlumyn for the treatment of severe frostbite in adults to reduce the risk of finger or toe amputation. Approval was supported by an open-label, single-site, controlled trial that enrolled 47 adults with severe (stage 3 or 4) frostbite, who received IV aspirin and standard of care therapy. Aurlumyn was administered IV over 6 hours daily for up to 8 days. On day 7, none of the patients in the Aurlumyn monotherapy group (0/16) had a bone scan with findings that indicated the need for amputation, compared with 19% (3/16) and 60% (9/15) of patients in the other two groups who did not receive Aurlumyn.10 Aurlumyn was granted an Orphan Drug designation for frostbite. Eicos is expected to launch the product during Spring 2024, with pricing to follow.

2/22/2024 Exblifep® (cefepime and enmetazobactam)

Under a Priority Review, the FDA approved Exblifep, a combination of cefepime, a cephalosporin antibacterial, and enmetazobactam, a beta-lactamase inhibitor, for the treatment of patients ≥ 18 years of age with complicated urinary tract infections (cUTI) including pyelonephritis caused by designated susceptible microorganisms. Exblifep was granted Fast Track and Qualified Infectious Disease Product designations. It is administered every 8 hours by IV infusion for 7 to 14 days. FDA approval was supported by the phase 3 ALLIUM trial (n=1,034) that demonstrated Exblifep was non-inferior to piperacillin/tazobactam when treating patients with cUTI or acute pyelonephritis with gram-negative pathogens.¹¹ Among patients who were randomized to Exblifeb, 79.1% achieved a clinical cure and micro-biological eradiation compared to 58.9% of patients randomized to piperacillin/tazobactam (4 g/0.5 g). Allecra has not announced the launch timeframe or price for Exblifep.

First generic drug launches

Alrex® (loteprednol etabonate) 0.2% ophthalmic suspension: Armas launched a generic version of Bausch & Lomb’s ophthalmic corticosteroid Alrex. Bauch & Lomb also launched their own authorized generic (AG) version of the product. The FDA also approved a generic version by Lupin, which could launch at any time. Alrex is indicated for the temporary relief of the signs and symptoms of seasonal allergic conjunctivitis. It generated $31 million in U.S. annual sales in 2023.

Bromsite® (bromfenac) ophthalmic solution 0.075%: Lupin launched a generic version of Sun’s ophthalmic nonsteroidal anti-inflammatory drug (NSAID) Bromsite. Sun also launched an AG version of the product. Bromsite is indicated for the treatment of postoperative inflammation and prevention of ocular pain in patients undergoing cataract surgery. In 2023, Bromsite generated $15 million in annual U.S. sales.

Emflaza®* (deflazacort) 6 mg, 18 mg, 30 mg, 36 mg tablet: Aurobindo launched its generic version of Emflaza tablets for the treatment of Duchenne muscular dystrophy. Emflaza tablets generated $249 million in U.S. annual sales in 2023.

Gralise® (gabapentin) 300 mg, 600 mg tablets: Zydus launched its generic version of Gralise tablets. Generic versions by at least one additional manufacturer could launch in 1Q2024. Gralise is a gamma-aminobutyric acid (GABA) analog indicated for the management of postherpetic neuralgia (PHN). It generated $90 million in U.S. annual sales in 2023 for all strengths.

Korlym®* (mifepristone): Teva launched a generic version of Corcept Therapeutic’s Korlym, a cortisol receptor blocker indicated to control hyperglycemia secondary to hypercortisolism in adults with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. In 2023, Korlym generated less than $10 million in U.S. annual sales.

Pradaxa® (dabigatran etexilate) 110 mg, capsules: Apotex launched the first generic version of Boehringer Ingelheim’s Pradaxa 110 mg capsule; they also launched generic versions of the 75 mg and 150 mg capsules. Other manufacturers’ generic versions of the 75 mg and 150 mg capsules have been available since 2022. Pradaxa is a direct thrombin inhibitor indicated to reduce the risk of stroke and systemic embolism in select adults, for the treatment and prevention of deep venous thrombosis (DVT) and pulmonary in select adults, and for the treatment and prevention of venous thrombotic events (VTE) in select pediatric patients. Pradaxa capsules generated $112 million in U.S. annual sales in 2023 for all strengths.

*Specialty

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