Pediatric Indication Expanded for Imcivree
On Dec. 20, 2024, Rhythm Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Imcivree® (setmelanotide) to include children as young as two years of age. Imcivree’s initial FDA approval was in November 2020 for treating obesity due to exceptionally rare deficiencies of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) in patients six years and older. In June 2022 it gained an additional indication for a rare condition known as Bardet-Biedl Syndrome (BBS) causing hyperphagia. The recommended starting dose for pediatric patients aged two years to less than six years of age is 0.5mg (0.05mL) once daily for two weeks, followed by a weight-based maintenance dose. For full prescribing information, click here.
Updates to Trikafta Labeling
Vertex Pharmaceuticals announced that the FDA approved the expanded use of Trikafta® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages two years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation that is responsive to Trikafta based on clinical and/or in vitro data on Dec. 20, 2024. This means that 94 additional non-F508del CFTR mutations have been added to the drug labeling, expanding treatment to about 300 additional people with CF in the US. Additionally, the previous drug-induced liver injury and liver failure precaution and warning have been upgraded to a boxed warning. For revised prescribing information, click here.
Zepbound Gets Sleep Apnea Indication
On Dec. 20, 2024, the FDA approved Eli Lilly’s Zepbound® (tirzepatide) for the treatment of moderate to severe obstructive sleep apnea (OSA) in adults with obesity, to be used in combination with a reduced-calorie diet and increased physical activity. Zepbound is a dual glucose-dependent insulinotropic polypeptide (GIP) receptor and glucagon-like peptide-1 (GLP-1) receptor agonist approved in adults with obesity or who are overweight with a weight-related comorbidity. OSA is a common sleep disorder that occurs when the upper airway becomes blocked during sleep, interrupting breathing and resulting in nonrestorative sleep with symptoms including loud snoring and excessive daytime sleepiness. Excess body fat is a major reversible risk factor for OSA and its complications. The phase III studies demonstrated that the maximum tolerated dose of Zepbound, 10mg or 15mg, for 52 weeks showed 20 fewer apnea events per hour for those on positive airway pressure (PAP), and 23.8 fewer events per hour for those not on PAP when compared to placebo. This translated into a 50-60% reduction in obesity-related OSA events, which is likely related to the body weight reduction of 18-20% observed with Zepbound treatment. This marks the first medication specifically indicated for OSA; however, it does not expand the eligible population as patients with obesity were already indicated for this drug. Here is the updated prescribing information.
New Combination Approved for Metastatic Colorectal Cancer
Pfizer announced that Braftovi® (encorafenib) in combination with Erbitux® (cetuximab) and mFOLFOX6 (fluorouracil, leucovorin, and oxaliplatin) was granted accelerated approval by the FDA on Dec. 20, 2024, for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test. Braftovi is also indicated in combination with Erbitux for the treatment of adult patients with mCRC with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy so this approval provides a first-line treatment option for this specific mutation. This approval was granted under Project FrontRunner, an FDA initiative to encourage studying cancer drugs for advanced or metastatic disease in an earlier clinical setting. Phase III studies showed a 61% overall response rate in treatment naïve patients given this combination compared to a 40% response rate in the control arm of Braftovi and Erbitux only, which was statistically significant. Prescribing information may be found here.
Generic Victoza Approved
On Dec. 23, 2024, the FDA announced the approval of the first generic Victoza® (liraglutide – Novo Nordisk), Hikma’s liraglutide 18mg/3mL. This GLP-1 receptor agonist is indicated to improve glycemic control in adult and pediatric patients aged ten years and older with type two diabetes as an adjunct to diet and exercise. Teva launched an authorized generic for Victoza in June 2024. The initial dose of liraglutide is 0.6mg administered subcutaneously daily and then increased to 1.2mg daily after one week to reduce gastrointestinal symptoms. Of note, Victoza and its generics are not approved for weight management. Cost and launch information are not known at this time. Prescribing information is here.
Gemtesa Approval for Overactive Bladder in BPH
The FDA approved Gemtesa® (vibegron – Sumitomo Pharma America) on Dec. 18, 2024, for the treatment of men with overactive bladder (OAB) symptoms, such as urge urinary incontinence, urgency, and urinary frequency, who are receiving pharmacological therapy for benign prostatic hyperplasia (BPH). Originally approved in 2020 for overactive bladder, Gemtesa is a beta-3 adrenergic agonist that works to relax bladder muscles to increase capacity. More than half of men with BPH have lower urinary tract symptoms even after BPH treatment. Studies showed significant reductions in the average number of urination episodes per day and daily urgency episodes in those treated with Gemtesa. The recommended dose is 75mg orally daily. Full prescribing information may be found here.
Recalls
Adrenalin Recalled
On Dec. 20, 2024, Endo, Inc., the parent company of Par Pharmaceutical, recalled six lots of Adrenalin® Chloride Solution (Epinephrine nasal solution, USP) 30mg/30mL (1mg/mL) 30 mL vials, to the consumer level. The FDA has determined that the labeling is misbranded and easily confused with the injectable version of Adrenalin® (Epinephrine injection, USP) (1mg/mL) 30mL vial, also produced by the same company. Adrenalin Chloride Solution (Epinephrine nasal solution) is used for topical application following mucosal or sinus surgery. The recall is being carried out because labeling makes it hard to distinguish from the injectable version of the drug, which could lead to the injection of a non-sterile product. Intravenous administration of a non-sterile nasal product could lead to serious health outcomes, including delayed treatment for conditions like anaphylaxis and also the increased risk of infection. There’s also a high risk of administering an incorrect dose of epinephrine in emergencies, putting patients at risk of death if not treated properly. For more information, see here.
Systane Eye Drops Recalled
On Dec. 21, 2024, Alcon Laboratories recalled one lot of Systane® Lubricant Eye Drops Ultra PF, Single Vials On-the-Go, 25 count to the consumer level. The recall was issued because fungal material was found inside a sealed single-use vial. Systane Lubricant Eye drops are a non-prescription eye lubricant used for temporary relief of dry eye symptoms, such as burning or irritation. Contamination with fungal material could lead to infections that are threatening to vision and life-threatening in patients with a weakened immune system. For more information see here.
Updated DrugWatch Document Available
The Emerging Therapeutics department has updated DrugWatch, a document that highlights near-term pipeline drugs as well as potential new generic opportunities. The DrugWatch document is attached.