Groundbreaking Drug Launches:
Notable mentions
Beyond these twelve groundbreaking drugs, few others are generating significant excitement in the market. In this report, we’ve highlighted ten such drugs that are making waves across various therapeutic areas, including haemophilia, cholesterol management, cancer, multiple sclerosis, and hereditary angioedema. Below is the list of these ten drugs:
Fenebrutinib
Fenebrutinib is an orally available, small-molecule, agammaglobulinemia tyrosine kinase (Bruton's tyrosine kinase) inhibitor, developed by Genentech for the treatment of multiple sclerosis. Expectations for BTK inhibitors in multiple sclerosis (MS) dropped after disappointing results in relapsing-remitting MS, but Roche hopes fenebrutinib will succeed with its unique non-covalent reversible profile, with Phase III results expected in 2025, while Sanofi recently revived the class with an unexpected Phase III win in secondary progressive MS.
Enlicitide decanoate
Enlicitide Decanoate, an oral cyclic peptide that inhibits proprotein convertase subtilisin-kexin type 9 (PCSK9), developed by Merck Sharp & Dohme for the treatment of hypercholesterolemia and hyperlipoproteinemia type IIa. Merck is expected to announce Phase III results this year for enlicitide decanoate, a promising first-in-class oral PCSK9 inhibitor with once-daily dosing. This could improve cholesterol management for statin users and create a new revenue stream as Keytruda’s patent approaches expiration.
Fitusiran
Fitusiran is a synthetic, double-stranded small interfering ribonucleic acid (siRNA) that targets antithrombin III, developed by Alnylam Pharmaceuticals in collaboration with Sanofi for the treatment of haemophilia A and B. Phase 3 trials shows that fitusiran significantly reduces bleeding rates, prevents bleeding in half of participants, enables safe surgeries for hemophilia A/B, and mitigates thrombotic and liver risks, with further studies in the ATLAS program; its monthly/bimonthly dosing, along with other non-factor and gene therapies like Alhemo and HYMPAVZI, is expected to evolve the hemophilia B treatment landscape, while the FDA has set a target action date of March 28, 2025, for its NDA application.
Vepdegestrant
Vepdegestrant, an orally bioavailable, estrogen receptor (ER) targeted PROTAC™ protein degrader developed by Arvinas for the treatment of breast cancer. Breast cancer will be a key oncology focus in 2025. Pfizer’s Ibrance sales are declining as newer CDK4/6 inhibitors take over, but the company hopes to return to success with vepdegestrant, an oral estrogen receptor-degrader. Two important Phase III results are expected this year: one for monotherapy in HR+ HER2- metastatic breast cancer patients previously treated with a CDK4/6 inhibitor, and another combining vepdegestrant with a CDK4 inhibitor in frontline patients.
Ivonescimab
Ivonescimab is a bispecific antibody targeting an anti-programmed death 1 (PD-1) and anti-vascular endothelial growth factor (VEGF), developed by Akeso Biopharma, for the treatment of colorectal cancer, gynaecological cancer, liver cancer, non-small cell lung cancer, glioblastoma, ovarian cancer, small cell lung cancer, pancreatic ductal adenocarcinoma, triple-negative-breast-cancer and other solid tumours such as gastric cancer, biliary tract cancers, head and neck cancer, squamous cell cancer and pancreatic cancer. Ivonescimab shows promising results as a treatment for PD-L1 positive NSCLC, outperforming pembrolizumab by reducing disease progression or death risk by 49% in a Phase 3 trial, with FDA approval expected in 2025 due to its dual targeting of PD-1 and VEGF pathways.
UGN-102
UGN-102 is a hydrogel-based sustained release formulation of mitomycin C, a DNA synthesis inhibitor, developed by UroGen Pharma, for the treatment of superficial (non-muscle invasive) bladder cancer and upper urinary tract urothelial cancer. This is a first-in-class non-surgical treatment combining mitomycin with UroGen's reverse thermal gel technology, which showed significant efficacy in Phase 3 trials and could become the first FDA-approved non-surgical option for LG-IR-NMIBC if approved in 2025..
Plozasiran
Plozasiran, a subcutaneously administered RNA interference (RNAi) therapeutic that targets apolipoprotein C-III (apoC-III), is being developed by Arrowhead Pharmaceuticals for the treatment of dyslipidemia, hypertriglyceridemia, and familial chylomicronemia syndrome. As Arrowhead's first-in-class RNAi drug, plozasiran is designed for severe hypertriglyceridemia (SHTG) and familial chylomicronemia syndrome (FCS), showing significant triglyceride reductions in clinical trials with the potential for a 2025 launch.
Donidalorsen
Donidalorsen is a second-generation, ligand-conjugated, antisense oligonucleotide inhibiting plasma prekallikrein (PKK), developed by Ionis Pharmaceuticals, for the treatment of hereditary angioedema and COVID-2019 infections. Donidalorsen reduces prekallikrein production to lessen attack frequency and severity, offering less frequent dosing and potentially fewer side effects, with FDA approval expected in 2025.
Tolebrutinib
Tolebrutinib is an orally available small molecule, developed by Sanofi, for the treatment of inflammation, multiple sclerosis, myasthenia gravis, and other CNS disorders. Tolebrutinib is a BTK inhibitor for non-relapsing secondary progressive MS, is nearing FDA approval with a 2030 sales forecast of $1.4 billion, following a Phase 3 trial showing a 31% delay in disability progression, despite some liver enzyme elevations.
Telisotuzumab vedotin
Telisotuzumab vedotin is an antibody-drug conjugate designed to target c-Met overexpression in non-small cell lung cancer (NSCLC). As a first-in-class therapy, it is expected to potentially reach the market in 2025 following AbbVie’s Biologics License Application (BLA) submission to the FDA in September 2024.