The US FDA New Drug Approvals in September 2024
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PharmaShots has compiled a list of US FDA-approved drugs in the month of September 2024
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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
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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
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- 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
Product Name: Ebglyss
Active ingredient: Lebrikizumab-lbkz
Company: Eli Lilly
Date: Sep 13, 2024
Disease: Moderate-to-Severe Atopic Dermatitis
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- 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)
Product Name: Ocrevus Zunovo
Active ingredients: Ocrelizumab & Hyaluronidase-ocsq
Company: Roche
Date: Sep 13, 2024
Disease: Relapse & Progressive Multiple Sclerosis
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- 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
Product Name: Miplyffa
Active ingredient: Arimoclomol Citrate
Company: Zevra Therapeutics
Date: Sep 20, 2024
Disease: Niemann-Pick Disease Type C
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- 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
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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)
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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
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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
Product Name: Cobenfy
Active ingredients: Xanomeline Tartrate and Trospium Chloride
Company: BMS
Date: Sep 26, 2024
Disease: Schizophrenia
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- 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)
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
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A passionate content writer with expertise in delivering high-quality and engaging content, Dipanshu is a keen reader and a versatile writer. Dipanshu dedicatedly covers news ranging from biopharma, life sciences, biotech, and MedTech to diagnostics and animal health companies. He can be contacted at connect@pharmashots.com