Shots:     

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

• The US FDA has approved a total of 10 new drugs including 4 new molecular entities and 6 biologics leading to the treatment of patients and advances in the healthcare industry       

• The major highlighted drug was BMS’ Opdivo Qvantig securing approval for treating G/GEJ Cancers 

1. Merus Reports the US FDA’s Accelerated Approval of Bizengri (Zenocutuzumab-zbco) for NRG1+ Pancreatic Adenocarcinoma and NSCLC 

Product Name: Bizengri 

Active ingredient: Zenocutuzumab-zbco 

Company: Merus 

Disease: Pancreatic Adenocarcinoma and NSCLC   

Date: Dec 04, 2024  

Shots:   

• The US FDA has granted accelerated approval to Bizengri for the treatment of advanced, unresectable or metastatic NRG1+ pancreatic adenocarcinoma or NSCLC. It will be commercialized by Partner Therapeutics in the US under previously signed agreement 

• Approval was based on eNRGy study assessing the safety, tolerability, PK/PD, immunogenicity & anti-tumor activity of zenocutuzumab to treat NRG1+ solid tumors incl. pancreatic cancer (n=30) & NSCLC (n=64) in patients, progressed on prior treatment 

• Study demonstrated an ORR of 40% & DoR range from 3.7 to 16.6mos. in pancreatic cancer patients while ORR of 33% & mDoR of 7.4mos. in NSCLC patients 

2. Neurocrine’s Crenessity (Crinecerfont) Secures the US FDA’s Approval to Treat Adults and Pediatrics with Congenital Adrenal Hyperplasia (CAH) 

Product Name: Crenessity 

Active ingredient: Crinecerfont 

Company: Neurocrine Biosciences 

Disease: Congenital Adrenal Hyperplasia 

Date: Dec 13, 2024  

Shots:   

• The US FDA’s approval of Crenessity as an adjunctive treatment to glucocorticoid replacement to treat CAH was based on a global P-III (CAHtalyst Pediatric: n=103, age: 4-17yrs. & Adult: n=182, age: 18-58yrs.) trials. It has also received PRV on approval 

• Pediatric study achieved its 1 & 2EP, depicting ~4x greater reductions in androstenedione at wk.4 & GC doses (with improved androgen levels) at wk.28 as well as ~12x greater reduction in 17-OHP vs PBO 

• Adult study also met its 1 & 2EP, showing 63% vs 18% (PBO) achieving physiologic GC doses with ~2x greater steroid dose reduction at wk.24, ~8x greater reduction in androstenedione levels at wk.4 and ~37x greater reduction in 17-OHP vs PBO 

3. Checkpoint Therapeutics’ Unloxcyt (Cosibelimab-ipdl) Receives the US FDA’s Approval for Cutaneous Squamous Cell Carcinoma (cSCC) 

Product Name: Unloxcyt 

Active ingredient: Cosibelimab-ipdl 

Company: Checkpoint Therapeutics 

Disease: Metastatic or Locally Advanced Cutaneous Squamous Cell Carcinoma 

Date: Dec 13, 2024  

Shots:   

• Checkpoint has received the US FDA’s approval for Unloxcyt (1,200mg, IV, over 60 minutes, Q3W) to treat metastatic or locally advanced cSCC in adults, ineligible for curative surgery or radiation 

• Approval was based on the P-I (CK-301-101) trial in solid tumor patients (incl. cSCC: N=109), showing ORR of 48% with mDoR of 17.7mos. in locally advanced cSCC patients (n=31) & ORR of 47% with mDoR not achieved in metastatic disease patients (n=78) 

• Unloxcyt is a human, anti-PD-L1 IgG1 mAb which blocks the interaction between PD-L1 & T cell receptors (PD-1 & B7.1) producing an anti-tumor immune response 

4. Galderma’s Nemluvio (Nemolizumab) Secures the US FDA’s Approval to Treat Moderate-To-Severe Atopic Dermatitis (AD) 

Product Name: Nemluvio 

Active ingredient: Nemolizumab 

Company: Galderma 

Disease: Moderate-to-Severe Atopic Dermatitis   

Date: Dec 13, 2024  

Shots:   

• The US FDA has approved Nemluvio + TCS ± calcineurin inhibitors (TCI) to treat mod. to sev. atopic dermatitis in patients unresponsive to topical therapies 

• Approval was based on the P-III (ARCADIA) study of the combination vs PBO in patients (n=1,728; ≥12yrs.), showing significant skin clearance in both co-1EPs of IGA score (0/1) & 75% EASI reduction at wk.16.; 2EPs of itch relief & improved sleep disturbance, were also met 

• Nemluvio has received the CHMP's positive opinion in Dec 2024 for AD & prurigo nodularis. Submissions in Australia, Singapore, Switzerland, Canada, Brazil & South Korea are under review, with more expected in 2025 

5. Xcovery Holdings Receives US FDA Approval for Ensacove (Ensartinib) to Treat ALK-Positive Locally Advanced or Metastatic NSCLC 

Product Name: Ensacove 

Active ingredient: Ensartinib 

Company: Xcovery Holdings 

Disease: ALK-Positive, Locally Advanced or Metastatic NSCLC 

Date: Dec 18, 2024  

Shots:   

• Xcovery Holdings has received the US FDA’s approval for Ensacove (225mg, oral, QD) for ALK+, locally advanced or metastatic NSCLC in patients not previously treated with ALK-targeted therapy 

• Approval was based on a global P-III (eXalt3) study assessing ensartinib vs crizotinib in 290 patients, demonstrating significantly improved PFS with mPFS of 25.8 vs 12.7mos. (1EP) & similar OS for both arms (2EP)  

• Ensartinib is an ALK tyrosine kinase inhibitor (TKI) developed by Xcovery & Betta Pharmaceuticals 

6. Ionis’ Tryngolza (Olezarsen) Receives the US FDA’s Approval to Treat Adults with Familial Chylomicronemia Syndrome (FCS) 

Product Name: Tryngolza 

Active ingredient: Olezarsen 

Company: Ionis Pharmaceuticals 

Disease: Familial Chylomicronemia Syndrome 

Date: Dec 19, 2024  

Shots:   

• The US FDA has approved Tryngolza (80mg, QM, self-administered via an auto-injector) as an adj. for reducing triglycerides (TG) in FCS patients, based on a P-III (BALANCE) study. Further submission is under review in the EU, with more filings planned  

• The P-III study involved FCS patients with fasting TG levels of ≥880mg/dL. Trial showed PBO-adjusted mean reduction in TG levels of 42.5% (6mos.) & 57% (12mos.) plus reduced AP events at 12mos. with 5% (1 pt) on Tryngolza having 1 episode vs 30% (7 pts) on PBO having 11 AP episodes 

• Tryngolza (RNA-targeted drug) reduces apoC-III production responsible for regulating TG & is being evaluated in 3 P-III trials (CORE, CORE2 & ESSENCE) to treat sHTG  

7. The US FDA Approves Vertex’s Alyftrek for the Treatment of Cystic Fibrosis 

Product Name: Alyftrek 

Active ingredient: Vanzacaftor/Tezacaftor/Deutivacaftor 

Company: Vertex Pharmaceuticals   

Disease: Cystic Fibrosis 

Date: Dec 20, 2024  

Shots:   

• The US FDA has approved Alyftrek (QD, CFTR modulator) to treat patients (≥6yrs.) with cystic fibrosis (CF), having at least one F508del mutation or another CFTR gene mutation. Submissions are under review in the EU, UK, Canada, Switzerland, Australia & New Zealand 

• Approval was supported by pivotal P-III CF program involving >1,000 patients across 20+ countries, with results reported as conclusion of the studies & highlighted at the North American Cystic Fibrosis Conference in Sep 2024 

• P-III studies in CF patients (≥12yrs.) showed that Alyftrek met its 1EP of ppFEV1 non-inferiority & 2EP of SwCl improvement vs Trikafta. In children (6–11yrs.), the drug demonstrated safety (1EP) as well as benefits in ppFEV1 & SwCl  

8. Novo Nordisk’s Alhemo Receives the US FDA’s Approval as a Prophylactic Treatment of Hemophilia A or B with Inhibitors 

Product Name: Alhemo 

Active ingredient: Concizumab-mtci 

Company: Novo Nordisk 

Disease: Hemophilia A or B 

Date: Dec 20, 2024  

Shots:   

• The US FDA has approved Alhemo injection (QD) as a prophylactic treatment to reduce the frequency of bleeding episodes in hemophilia A or B patients (≥12yrs.) with inhibitors 

• Approval was based on the P-III (explorer7) study assessing Alhemo's efficacy and safety by comparing annual bleeding rates (ABR) in patients aged 12+ with hemophilia A or B with inhibitors 

• Study showed Alhemo reduced annual bleeding rates (ABR) by 86%, with mean ABR of 1.7 vs 11.8. The overall median ABR was 0 vs 9.8 without prophylaxis, and 64% of patients on Alhemo had no spontaneous and traumatic bleeds in the first 24wks., vs 11% without prophylaxis 

9. BeiGene’s Tevimbra Plus Chemotherapy Secures the US FDA’s Approval as a 1L Treatment of G/GEJ Cancers 

Product Name: Tevimbra + CT 

Active ingredient: Tislelizumab-jsgr 

Company: BeiGene 

Disease: G/GEJ Cancers 

Date: Dec 26, 2024  

Shots:   

• The US FDA has approved Tevimbra + Pt & fluoropyrimidine-based CT as a 1L treatment of inoperable or metastatic HER2- G/GEJ adenocarcinoma with PD-L1 (≥1) expression. Another BLA for 1L locally advanced unresectable or metastatic ESCC is under review 

• Approval was based on P-III (RATIONALE-305) study of Tevimbra + CT vs PBO in patients (n=997) that achieved 1EP, showing OS benefits with a 20% reduction in the death risk & mOS of 15 vs 12.9mos. 

• Pooled safety analysis included data from 2 studies (RATIONALE-302 & BGB-A317-303) in 1,972 patients plus 5 trials (BGB-A317-208, BGB-A317-204, BGB-A317-203, BGB-A317-102 & BGB A317_Study_001) in patients with ESCC (n=307) & advanced or recurrent tumors (n=1,665) 

10. BMS’ Opdivo Qvantig SC Injection Secures the US FDA’s Approval for Solid Tumor Opdivo Indications 

Product Name: Opdivo Qvantig 

Active ingredient: Nivolumab; Hyaluronidase-nvhy 

Company: BMS 

Disease: G/GEJ Cancers 

Date: Dec 27, 2024  

Shots:   

• The US FDA has approved Opdivo Qvantig (nivolumab & hyaluronidase-nvhy) SC injection for previously approved solid tumor Opdivo indications, incl. as monotx., maintenance therapy after Opdivo-Yervoy combination & with CT or cabozantinib 

• Approval was based on P-III (CheckMate-67T) study assessing Opdivo Qvantig (nivolumab: 1,200mg & hyaluronidase: 20,000 units, SC, Q4W; n=248) vs Opdivo (3mg/kg, IV, Q2W; n=247) in adults (N=495) with advanced or metastatic ccRCC treated previously with systemic therapy 

• Study depicted noninferiority in co-1EPs of Cavgd28 (GMR: 2.10) and Cminss (GMR: 1.77). The ORR was 24% vs 18%, showing comparable efficacy 

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