Shots:     

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

• The US FDA has approved a total of 7 new drugs including 4 new molecular entities and 3 biologics leading to the treatment of patients and advances in the healthcare industry       

• The major highlighted drugs were Roche’s Tecentriq Hybreza & Ocrevus Zunovo for the treatment of various cancers as well as relapse & progressive multiple sclerosis, respectively  

1. Roche’s Tecentriq Hybreza Receives the US FDA’s Approval for Cancer Treatment 

Product Name: Tecentriq Hybreza 

Active ingredients: Atezolizumab and Hyaluronidase-tqjs 

Company: Roche 

Date: Sep 12, 2024   

Disease: Lung, Liver, Skin & Soft Tissue Cancers 

Shots:   

• The US FDA has approved Tecentriq Hybreza (SC) to treat all IV indications, incl. lung, liver, skin & soft tissue cancers. Regulatory reviews across other regions are underway 
• The approval was supported by P-IB/III (IMscin001) study assessing Tecentriq Hybreza (SC) vs Tecentriq (IV) in locally advanced or metastatic NSCLC patients (n=371) failed on Pt therapy and P-II (IMscin002) assessing patient preference b/w SC vs IV in those (n=179) with PD-L1+ve resected Stage II-IIIB NSCLC & untreated Stage IV NSCLC 
• IMscin001 trial showed consistent Tecentriq levels in blood with safety profile aligning with IV formulation. In the IMscin002 trial, 71% preferred SC over IV, with 79% opting to continue treatment with it 

2. Eli Lilly’s Ebglyss (Lebrikizumab-lbkz) Receives the US FDA’s Approval for Treating Moderate-to-Severe Atopic Dermatitis 

Product Name: Ebglyss 

Active ingredient: Lebrikizumab-lbkz 

Company: Eli Lilly 

Date: Sep 13, 2024   

Disease: Moderate-to-Severe Atopic Dermatitis 

Shots:    

• The US FDA has approved Ebglyss to treat moderate-to-severe AD in adults & children (≥12yrs.) weighing 88 pounds (40kg). Lilly holds its exclusive rights outside the EU, while Almirall has licensed rights in the EU 
• The approval was supported by P-III (ADvocate 1 & 2) studies of Ebglyss alone and P-III (ADhere) study of Ebglyss + topical corticosteroids in over 1000 adults & children (12-18yrs.) with mod. to sev. AD 
• ADvocate 1 & 2 studies showed clear or almost-clear skin in 38% vs 12% (77% maintained it for 1yr. with QM dosing & 48% maintained it after switching to PBO) and 43% vs 12% had itch relief at 16wks. (85% maintained for 1yr. with QM dosing & 66% maintained it post switching to PBO) 

3. Roche Reports the US FDA’s Approval of Ocrevus Zunovo for Treating Relapse and Progressive Multiple Sclerosis 

Product Name: Ocrevus Zunovo 

Active ingredients: Ocrelizumab & Hyaluronidase-ocsq 

Company: Roche 

Date: Sep 13, 2024   

Disease: Relapse & Progressive Multiple Sclerosis 

Shots:    

• The US FDA has approved Ocrevus Zunovo (ocrelizumab & hyaluronidase-ocsq), a twice-a-year, 10-minute SC injection, to treat relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS) 
• The approval was supported by the pivotal P-III (OCARINA) study assessing the PK, safety, clinical & radiological efficacy of the Ocrevus SC vs IV in patients (n=236) with RMS or PPMS 
• The study depicted that SC Ocrevus Zunovo had similar blood levels, safety & efficacy to the IV form in RMS & PPMS. It suppressed relapse activity & MRI lesions by 97% over 48wks. Over 92% of them preferred SC formulation 

4. Zevra Therapeutics Receives the US FDA’s Approval of Miplyffa (Arimoclomol Citrate) for Treating Niemann-Pick Disease Type C (NPC) 

Product Name: Miplyffa 

Active ingredient: Arimoclomol Citrate 

Company: Zevra Therapeutics 

Date: Sep 20, 2024   

Disease: Niemann-Pick Disease Type C 

Shots:    

• The US FDA has awarded priority review voucher & approved Miplyffa (47mg to 124mg, oral, TID) + miglustat to treat neurological manifestations in adults & pediatrics (≥2yrs.) with NPC, with its launch planned within 8-12wks.   
• Approval was based on trial of Miplyffa + miglustat vs PBO in NPC patients (2-19yrs.), showing halted disease progression with 0.2 vs 1.9-point reduction at 12mos. Data from a 48mos. OLE trial provided additional evidence of improved outcomes 
• Zevra has launched AmplifyAssist, offering insurance education, copay assistance, disease information, therapy management counseling & support for prescription refills for the eligible patients in need 

5. IntraBio’s Aqneursa Receives the US FDA’s Approval for Treating Niemann-Pick Disease Type C 

Product Name: Aqneursa 

Active ingredient: Levacetylleucine 

Company: IntraBio 

Date: Sep 24, 2024   

Disease: Niemann-Pick Disease Type C 

Shots:    

• The US FDA has granted approval to Aqneursa (levacetylleucine) for treating adults & pediatric patients (≥15kg) with neurological manifestations of Niemann-Pick disease type C (NPC) 

• Approval was supported by pivotal P-III (IB1001-301) study assessing the effect of Aqneursa vs PBO on neurological symptoms & functioning in pediatric (≥4yrs.) & adults (n=60) with confirmed NPC 

• Study reached all 1 & 2EPs, depicting improved neurological symptoms & functional benefits at 12wks. as well as greater improvement in fSARA scores, with a mean difference of -0.4. Results were published in the New England Journal of Medicine 

6. BMS’ Cobenfy (Xanomeline and Trospium Chloride) Receives the US FDA Approval for Treating Schizophrenia 

Product Name: Cobenfy 

Active ingredients: Xanomeline Tartrate and Trospium Chloride  

Company: BMS 

Date: Sep 26, 2024   

Disease: Schizophrenia 

Shots:    

• The US FDA has granted approval to BMS’ Cobenfy for treating schizophrenia in adults, based on its EMERGENT studies 
• The EMERGENT studies comprised of P-III (EMERGENT-2 & 3) trials, evaluating the safety, efficacy & tolerability of Cobenfy vs PBO in schizophrenic adults for over 5wks., as well as two open-label trials assessing long-term safety & tolerability of Cobenfy for over 1yr. 
• Studies reached the 1EPs, showing reduced schizophrenia symptoms with 9.6 (-21.2 vs -11.6 in EMERGENT-2) and 8.4 (-20.6 vs -12.2 in EMERGENT-3) point reductions in PANSS total scores at wk.5. EMERGENT-2 study also depicted a change of 0.6 (-1.2 vs -0.7) in CGI-S score at wk.5 (2EP) 

7. GE HealthCare Reports the US FDA’s Approval of Flyrcado (Flurpiridaz F 18) PET Radiotracer for Improved CAD Diagnosis 

Product Name: Flyrcado 

Active ingredient: Flurpiridaz F-18 

Company: GE HealthCare  

Date: Sep 27, 2024   

Disease: Coronary Artery Disease 

Shots:    

• The US FDA has approved Flyrcado injection as a PET MPI agent to diagnose CAD, with its launch planned in early 2025  
• The effectiveness of Flyrcado was assessed under the P-III (AURORA) study in comparison with both invasive coronary angiography as a standard and SPECT MPI to identify CAD   
• In addition, GE HealthCare secured exclusive global commercialization rights of the flurpiridaz F 18 from Lantheus in 2017, funding its development until approval. Lantheus will partner on commercialization through a joint steering committee & is entitled to get royalties based on sales milestones 

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