Drug Approvals Monthly Update: January 2024

Critical updates in an ever-changing environment

January 31, 2024

This monthly approval update provides a review of newly approved specialty and traditional drugs, new indications and recent generic launches.

Specialty

New drugs

12/22/2023 Wainua™ (eplontersen) The United States (U.S.) Food and Drug Administration (FDA) approved a transthyretin-directed antisense oligonucleotide, by Ionis and AstraZeneca. It is the fourth medication approved for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Wainua was approved as a single-dose auto-injection for once-monthly self-administration of a subcutaneous (SC) dose. Published results from the open-label, single-group, phase 3 NEURO-TTRansform study demonstrated improvements in all three co-primary efficacy endpoints, which included changes from baseline to week 65/66 in serum transthyretin concentration, modified Neuropathy Impairment Score +7 (mNIS+7) composite score (scoring range, –22.3 to 346.3; higher scores indicate poorer function), and Norfolk Quality of Life Questionnaire–Diabetic Neuropathy (Norfolk QoL-DN) total score.¹ The difference in the adjusted mean percentage reduction in serum transthyretin from baseline between eplontersen and placebo was −70.4% (95% confidence interval [CI], −75.2% to −65.7%; p < 0.001), the difference in the adjusted mean change for the mNIS+7 composite score was −24.8 (95% CI, −31 to −18.6; p<0.001), the difference for Norfolk QoL-DN was −19.7 (95% CI, −25.6 to −13.8; p<0.001). Wainua will be available in the U.S. in January 2024. The annual wholesale acquisition cost (WAC) is $499,000, which is similar to the pricing for the other agents approved for this condition (Amvuttra®, Onpattro®, Tegsedi®).

New indications

1/12/24 HyQvia (immune globulin 10% [human] with recombinant hyaluronidase [human]): The FDA approved HyQvia for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment in adults. Approval of the new indication was supported by the double-blind ADVANCE-CIDP 1 trial, which demonstrated a significant reduction in CIDP relapse with HyQvia compared to placebo (difference, -21.8%; 95% CI, -34.5% to -7.9%; p=0.0045).² HyQvia is also indicated to treat primary immunodeficiency in patients 2 years and older.

1/12/24 Keytruda® (pembrolizumab): Merck’s programmed death receptor-1 (PD-1) inhibitor Keytruda was approved for use in combination with chemoradiotherapy, for the treatment of patients with International Federation of Gynecology and Obstetrics (FIGO) 2014 Stage III-IVA cervical cancer. Approval of this new indication was supported by data from the double-blind KEYNOTE-A18 trial, which demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS) in the overall study population with cervical cancer who had not previously received definitive surgery, radiation, or systemic therapy.³ In addition, an exploratory subgroup analysis revealed that, in patients with FIGO 2014 Stage III-IVA‑ disease, the PFS hazard ratio (HR) estimate was 0.59 (95% CI, 0.43 to 0.82).

1/16/24 Casgevi™ (exagamglogene autotemcel): Vertex received a new indication for their CRISPR/Cas9 gene-edited cell therapy, Casgevy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. Casgevy is the first gene therapy to utilize CRISPR-based technology to alter a patient’s hematopoietic stem cells (HSCs) to produce red blood cells (RBCs) with high levels of fetal hemoglobin (HbF). Casgevy is intended to be used as a 1-time intravenous (IV) infusion that is administered at authorized treatment centers. Treatment involves mobilization, collection, and editing of the patients HSCs, as well as myeloablative conditioning therapy prior to re-introduction of the engineered HSCs back into the patient. The ongoing, single-arm CLIMB THAL-111 study revealed that, at a median follow-up of 23.8 months (range, 16.1 to 48.1 months) after Casgevy administration, 32 of the 35 patients (91.4%; 98.3% one-sided CI, 75.7 to 100) who achieved the primary outcome of transfusion independence for 12 consecutive months.4 No patient experienced graft failure or graft rejection.

1/18/2024 Piqray® (alpelisib): The FDA expanded the indication for Novartis’ kinase inhibitor Piqray to include pre- and perimenopausal women for use in combination with fulvestrant for the treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer as detected by an FDA-approved test following progression on or after an endocrine-based regimen.5

1/19/24 Balversa® (erdafitinib): The FDA amended Balversa’s indication. It is now indicated for the treatment of adults with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy. The FDA granted a Full Approval for this revised indication; the previous indication was given an Accelerated Approval. Notably, Balversa is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or programmed death-ligand 1 (PD-L1) inhibitor therapy. Data from the BLC3001 study cohort 1 revealed a statistically significant improvement in the primary endpoint of overall survival (OS) with Balversa compared to chemotherapy (median OS, 12.1 versus 7.8 months, respectively; HR, 0.64; 95% CI, 0.47 to 0.88; p=0.005).6

 

 

Traditional

New drugs

1/05/2024 Zelsuvmi™ (berdazimer): The FDA approved Zelsuvmi for the topical treatment of molluscum contagiosum (MC) in adult and pediatric patients 1 year of age and older. Zelsuvmi is a first-in-class nitric oxide releasing antiviral. It is approved as a 10.3% berdazimer gel co-packaged with a hydrogel, which must be mixed prior to topical application. The mixed gel is applied by the patient or caregiver once daily to each MC lesion for up to 12 weeks. In the phase 3, double-blind B-SIMPLE4 trial, significantly more immunocompetent patients ≥ 6 months of age treated with berdazimer achieved the primary endpoint of complete clearance of all MC lesions at week 12 compared to patients who received vehicle (43.5% versus 24.6%, respectively; p<0.001).7 Zelsuvmi is the first topical agent for MC that is administration by the patient or caregiver and may be a more convenient alternative to Verrica’s healthcare provider-administered topical Ycanth™ (cantharidin). Ligand Pharmaceuticals plans to launch Zelsuvmi in 2H 2024, with pricing to follow.

New indications

12/20/2023 Tarpeyo® (budesonide): The FDA has revised the indication for the oral corticosteroid, Tarpeyo, for Full Approval to reduce loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. Previously, it was granted an Accelerated Approval to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥ 1.5 g/g.

Generic drug launches

Prolensa® (bromfenac): Lupin launched a generic version of Bausch & Lomb’s ophthalmic non-steroidal anti-inflammatory drug (NSAID) Prolensa. Bauch & Lomb also launched an authorized generic version of the product. Several other manufacturers may launch generic versions in July 2024 or beyond. Prolensa is indicated for postoperative inflammation and reduction of ocular pain following cataract surgery. It generated $186 million in U.S. annual sales in 2022.

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