És bo fer una ullada a quins són els medicaments que previsiblement s'aprovaran l'any 2024, i els de Nature diuen que són aquests:
Medicaments per aprovar el 2024
Biologic name | Sponsor | Properties | Indication | Timing |
|---|---|---|---|---|
Zolbetuximab | Astellas | Claudin 18.2-targeted mAb | Gastric cancer | January |
Lifileucel | Iovance | Tumour-infiltrating lymphocyte therapy | Melanoma | February |
Resmetiroma | Madrigal/Synta | Thyroid hormone receptor β agonist | NASH | March |
Sotatercepta | Merck & Co./Acceleron | Fusion protein ligand trap for TGF-β superfamily | PAH | March |
mRNA-1345a | Moderna | mRNA-based vaccine | RSV prevention | April |
Donanemaba | Eli Lilly | Amyloid-β-targeted mAb | Alzheimer disease | Q1 |
EB-101a | Abeona | Gene therapy with COL7A2 transgene | RDEB | May |
Patritumab deruxtecana | Merck & Co. | HER3-targeted ADC | NSCLC | June |
Imetelstat | Geron | Telomerase inhibitor | Transfusion-dependent anaemia with MDS | June |
Tarlatamaba | Amgen | DLL3 × CD3 T-cell engager antibody | SCLC | June |
Fidanacogene elaparvoveca | Pfizer/Spark | AAV-based gene therapy with factor IX transgene | Hemophilia B | Q2 |
Bentracimaba | Laboratoires SERB | Ticagrelor-neutralizing antibody | Drug toxicity | 1H |
Crovalimaba | Roche | C5-targeted mAb | PNH | July |
Danicopana | AstraZeneca/Alexion | Factor D inhibitor | PNH | July |
Midomafetaminea | MAPS | MDMA | PTSD | August |
Xanomeline plus trospium | Karuna/BMS | Muscarinic receptor modulators | Schizophrenia | September |
Acoramidis | BridgeBio | TTR stabilizer | TTR amyloidosis | December |
Marstacimab | Pfizer | TFPI-targeted mAb | Haemophilia A and B | Q4 |
Afamitresgene autoleucela | Adaptimmune | MAGE-A4-targeted autologous, engineered T cell therapy | Synovial sarcoma | 2024 |
Fig. 1 | 30 years of novel FDA approvals. Annual numbers of new molecular entities (NMEs) and biologics license applications (BLAs) approved by the FDA’s Center for Drug Evaluation and Research (CDER). See Table 1 for new approvals in 2023. Products approved by the Center for Biologics Evaluation and Research (CBER), including vaccines and gene therapies, are not included in this drug count (Table 2). Source: FDA.
Fig. 3 | CDER approvals by modality. Small molecules, including peptides of up to 40 amino acids in length, and oligonucleotides are approved as new molecular entities (NMEs). Protein-based candidates are approved through biologics license applications (BLAs). mAb, monoclonal antibody; siRNA, small interfering RNA. Source: Nature Reviews Drug Discovery.
Vertex and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; Casgevy) especially is the first CRISPR–Cas9-based gene editor to secure a green light from the FDA, winning an approval for sickle cell disease (SCD). Exa-cel is an ex vivo gene-edited cell therapy: blood cells are harvested from patients, genetically modified at the BCL11a transcription factor to re-enable fetal haemoglobin production, and then re-infused into patients. The therapeutically upregulated fetal haemoglobin compensates for the defects in β-haemoglobin that cause the diseases. Clinical data shows that the gene therapy has curative potential, although longer-term data are needed to assess the durability of the effect.
When Harvard Medical School and HHMI’s Stuart Orkin and colleagues discovered the role of BCL11a in fetal haemoglobin production in 2008, it was unclear how to drug the transcription factor. The arrival of CRISPR–Cas9 gene-editing system in 2012 provided a path forward for haemoglobinopathies. The development of the programme was “remarkably fast”, said Orkin. “It is a perfect example of how the ecosystem can work.”
Vertex and CRISPR have priced the one-off treatment at $2.2 million. It also requires a harsh preconditioning chemotherapy regimen, to make room for the edited cells. The therapy will consequently remain out of reach for many patients. “This is not the end game,” says Orkin, who has his eye on next-generation gene editors and small molecules that might be more accessible.
Table 1 | CDER approvals in 2023
Drug (brand name) | Sponsor | Properties | Indication |
|---|---|---|---|
Lecanemab (Leqembi)a | Eisai/Biogen | Amyloid-β-targeted mAb | Alzheimer disease |
Bexagliflozin (Brenzavvy) | Theracosbio | SGLT2 inhibitor | Glycaemic control in type 2 diabetes mellitus |
Pirtobrutinib (Jaypirca) | Loxo/Eli Lilly | BTK inhibitor | Mantle cell lymphoma |
Elacestrant (Orserdu) | Stemline | ER antagonist | ER-positive, HER2-negative, ESR1-mutant breast cancer |
Daprodustat (Jesduvroq) | GSK | HIF-PH inhibitor | Anaemia caused by CKD for adults on dialysis |
Velmanase alfa (Lamzede)a | Chiesi | Recombinant α-mannosidase | Non-CNS manifestations of α-mannosidosis |
Sparsentan (Filspari) | Travere | Endothelin and angiotensin II receptor antagonist | Proteinuria in primary IgA nephropathy |
Omaveloxolone (Skyclarys) | Reata/Biogen | Mechanism unknown, NRF2 activator | Friedrich’s ataxia |
Zavegepant (Zavzpret) | Pfizer | CGRP receptor antagonist | Migraine |
Trofinetide (Daybue) | Acadia | Mechanism unknown | Rett syndrome |
Retifanlimab (Zynyz)a | Incyte | PD1-targeted mAb | Merkel cell carcinoma |
Rezafungin (Rezzayo) | Cidara | Echinocandin antifungal | Candidemia and invasive candidiasis |
Leniolisib (Joenja) | Pharming | PI3Kδ inhibitor | Activated PI3Kδ syndrome |
Tofersen (Qalsody) | Biogen | SOD1-targeted ASO | SOD1 amyotrophic lateral sclerosis |
Pegunigalsidase alfa (Elfabrio)a | Chiesi | PEGylated recombinant α-galactosidase Α | Fabry disease |
Fezolinetant (Veozah) | Astellas | Neurokinin 3 receptor antagonist | Hot flashes caused by menopause |
Perfluorohexyloctane (Miebo) | Bausch + Lomb | Semifluorinated alkane | Dry eye disease |
Epcoritamab (Epkinly)a | Genmab/AbbVie | CD20 × CD3 T-cell engager | DLBCL and high-grade B-cell lymphoma |
Sulbactam, durlobactam (Xacduro) | Entasis | β-lactam antibacterial plus a β-lactamase inhibitor | Hospital-acquired and ventilator-associated bacterial pneumonia caused by susceptible ABC |
Nirmatrelvir, ritonavir (Paxlovid) | Pfizer | SARS-CoV-2 main protease inhibitor plus a CYP3A inhibitor | Mild-to-moderate COVID-19 |
Flotufolastat F18 (Posluma) | Blue Earth | Radioactive diagnostic agent | PET imaging in prostate cancer |
Sotagliflozin (Inpefa) | Lexicon | SGLT1/2 inhibitor | Heart failure |
Glofitamab (Columvi)a | Genentech | CD20 × CD3 T-cell engager | DLBLC or large B-cell lymphoma |
Ritlecitinib (Litfulo) | Pfizer | JAK3 inhibitor | Alopecia areata |
Rozanolixizumab (Rystiggo)a | UCB | FcRn-targeted mAb | AChR- or MuSK-antibody-positive gMG |
Somatrogon (Ngenla)a | Pfizer | Human growth hormone analogue | Growth hormone deficiency |
Nirsevimab (Beyfortus)a | AstraZeneca | RSV F protein-targeted mAb | RSV lower respiratory tract disease |
Quizartinib (Vanflyta) | Daiichi Sankyo | FLT3 kinase inhibitor | AML |
Lotilaner (Xdemvy) | Tarsus | Ectoparasiticide | Demodex blepharitis |
Zuranolone (Zurzuvae) | Sage | GABAA receptor PAM | Postpartum depression |
Avacincaptad pegol (Izervay) | Iveric/Astellas | C5-targeted aptamer | Geographic atrophy secondary to AMD |
Talquetamab (Talvey)a | Janssen | GPRC5D × CD3 T-cell engager | Multiple myeloma |
Elranatamab (Elrexfio)a | Pfizer | BCMA × CD3 T-cell engager | Multiple myeloma |
Palovarotene (Sohonos) | Ipsen | Retinoic acid receptor agonist | Fibrodysplasia ossificans progressiva |
Pozelimab (Veopoz)a | Regeneron | C5-targeted mAb | CHAPLE disease |
Motixafortide (Aphexda) | Biolinerx | CXCR4 inhibitor | Hematopoietic stem cell mobilization for autologous transplantation in multiple myeloma |
Momelotinib (Ojjaara) | GSK | JAK1/2, ALK2 inhibitor | Myelofibrosis in adults with anaemia |
Gepirone (Exxua) | Fabre-Kramer | 5HT1A receptor agonist | Major depressive disorder |
Cipaglucosidase alfa (Pombiliti)a | Amicus | Recombinant α-glucosidase | Pompe disease |
Nedosiran (Rivfloza) | Novo Nordisk | LDHA-targeted siRNA | Primary hyperoxaluria type 1 |
Etrasimod (Velsipity) | Pfizer | S1P receptor modulator | Ulcerative colitis |
Zilucoplan (Zilbrysq) | UCB | Complement C5 inhibitor | AChR-antibody positive gMG |
Bimekizumab (Bimzelx)a | UCB | IL-17A/F-targeted mAb | Plaque psoriasis |
Vamorolone (Agamree) | Santhera | Corticosteroid | Duchenne muscular dystrophy |
Mirikizumab (Omvoh)a | Eli Lilly | IL-23-targeted mAb | Ulcerative colitis |
Toripalimab (Loqtorzi)a | Coherus | PD1-targeted mAb | Nasopharyngeal carcinoma |
Fruquintinib (Fruzaqla) | Takeda | VEGFR1/2/3 kinase inhibitor | Colorectal cancer |
Taurolidine, heparin (Defencath) | Cormedix | Thiadiazinane antimicrobial plus an anticoagulant | Incidence of catheter-related bloodstream infections |
Repotrectinib (Augtyro) | Bristol Myers Squibb | ROS1 and TRK kinase inhibitor | ROS1-positive NSCLC |
Efbemalenograstim alfa (Ryzneuta)a | Evive | Recombinant leukocyte growth factor | Neutropenia |
Capivasertib (Truqap) | AstraZeneca | AKT kinase inhibitor | Breast cancer |
Nirogacestat (Ogsiveo) | Springworks | γ-secretase inhibitor | Desmoid tumours |
Iptacopan (Fabhalta) | Novartis | Complement factor B inhibitor | Paroxysmal nocturnal haemoglobinuria |
Birch triterpenes (Filsuvez) | Chiesi | Mechanism unknown | Epidermolysis bullosa |
Eplontersen (Wainua) | Ionis/AstraZeneca | TTR-targeted ASO | hATTR with polyneuropathy |
