Shots:     

• PharmaShots has compiled a list of US FDA-approved drugs in the month of November 2024     

• The US FDA has approved a total of 5 new drugs including 4 new molecular entities and 1 biologic leading to the treatment of patients and advances in the healthcare industry       

• The major highlighted drug was Jazz Pharmaceuticals’ Ziihera securing accelerated approval for treating HER2+ Biliary Tract Cancer 

1. Syndax Pharmaceuticals’ Revuforj (Revumenib) Receives the US FDA’s Approval for R/R Acute Leukemia 

Product Name: Revuforj 

Active ingredient: Revumenib 

Company: Syndax Pharmaceuticals 

Disease: Relapsed/Refractory Acute Leukemia    

Date: Nov 15, 2024  

Shots:   

• Based on P-I/II (AUGMENT-101) study, the US FDA has approved Revuforj to treat r/r acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in individuals (age≥1yr.) 

• In 104 efficacy evaluable patients, data showed CR + CRh was 21%, with median duration of 6.4mos. & median time to CR or CRh of 1.9mos.; 23% underwent HSCT post Revuforj. 

• The company will release 110 & 160mg tablets in Nov across the US and 25mg tablets for patients with wt. <40kg in H1’25. An oral formulation will available under expanded access program for those with <40kg before the availability of 25mg tablets 

2. Jazz Pharmaceuticals’ Ziihera (Zanidatamab-hrii) Secures the US FDA’s Accelerated Approval to Treat HER2-Positive (IHC 3+) Biliary Tract Cancer (BTC) 

Product Name: Ziihera 

Active ingredient: Zanidatamab-hrii 

Company: Jazz Pharmaceuticals 

Disease: Biliary Tract Cancer    

Date: Nov 20, 2024 

Shots:   

• The US FDA’s accelerated approval of Ziihera (50mg/mL, IV) was based on P-IIb (HERIZON-BTC-01) study in patients (n=87) with HER2+, locally advanced unresectable or metastatic BTC across 2 arms, based on tumor IHC status 

• Efficacy in 62 HER2+ BTC patients (arm 1 of HERIZON-BTC-01) showed a 52% ORR & 14.9mos. mDoR. Continued approval depends on P-III (HERIZON-BTC-302) confirmatory trial of zanidatamab + SoC vs SoC alone in 1L HER2+ BTC 

• Zanidatamab is also being studied P-III (HERIZON-GEA-01) trial with CT & with/without tislelizumab for 1L HER2+ GEAs as well as P-III (EmpowHER-303) trial with physician's choice CT HER2+ mBC patients, progressed on or intolerant to trastuzumab deruxtecan 

3. AOP Health’s Rapiblyk (landiolol) Receives the US FDA’s Approval for Atrial Fibrillation and Atrial Flutter in the Critical Care Setting 

Product Name: Rapiblyk 

Active ingredient: landiolol 

Company: AOP Health 

Disease: Atrial Fibrillation & Atrial Flutter    

Date: Nov 22, 2024 

Shots:   

• The US FDA has granted approval to Rapiblyk for treating severe heart condition supraventricular tachycardia (atrial fibrillation and atrial flutter) in critical care setting 

• Approval was supported by 5 clinical trials assessing the safety & efficacy of Rapiblyk (9.3 to 74.6 mcg/kg/min) vs PBO in adults (n=317) with supraventricular tachycardia, showing 40-90% vs 0-11% reduction in the heart rate & 9.9% vs 1% AEs 

• Rapiblyk, adrenergic receptor antagonist with beta 1/2 selectivity, lowers heart rate without affecting blood pressure. It is approved in the EU for treating supraventricular tachycardia, incl. atrial fibrillation & flutter, and managing non-compensatory sinus tachycardia 

4. BridgeBio Pharma’s Attruby (Acoramidis) Secures the US FDA’s Approval to Treat ATTR-CM 

Product Name: Attruby 

Active ingredient: Acoramidis 

Company: BridgeBio Pharma 

Disease: Transthyretin Amyloid Cardiomyopathy   

Date: Nov 22, 2024 

Shots:   

• The US FDA has approved Attruby (oral stabilizer of Transthyretin) to reduce cardiovascular death & associated hospitalization in ATTR-CM patients. The MAA is also under review in the EU, with decision anticipated in 2025 

• Approval was based on P-III (ATTRibute-CM) study of Attruby vs PBO in ATTR-CM patients (n=632). It achieved its 1EP, showing a 42% reduced ACM & recurrent CVH events, 50% reduced cumulative frequency of CVH events at 30mos. & a Win Ratio of 1.8 for a composite of ACM, CVH, NT-proBNP & 6-minute walk distance 

• Bayer holds exclusive commercial rights of Attruby in the EU & will get $500M under their royalty agreement 

Note: 

According to the FDA's November 2024 approval list, the following drug was also approved; however, no PR was available:    

• Iomervu 

Related Post: Insights+: The US FDA New Drug Approvals in October 2024