CDER approved 50 novel drugs in 2024, record number of biosimilars
A report released by the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) notes that the Center approved 50 novel drugs in 2024, as well as a record number of biosimilars and a significant number of therapies with orphan drug designation.While the number of drugs approved by CDER in 2024 was lower than the 55 novel drugs approved in 2023, it ties 2020 in having the third highest number of novel approved drugs within the last 5 years. CDER has approved an average of 47 novel drugs per year over the last decade, reaching a high of 59 novel drug approvals in 2018 and a low of 22 drugs approved in 2016. (RELATED: CDER notches uptick in novel drug approvals in 2023, Regulatory Focus 9 January 2024)
“Our 2024 drug approvals include therapies for a variety of diseases and conditions, including rare diseases. FDA approved a significant number of rare disease therapies with orphan drug designation for patients with few to no treatment options,” Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER) at FDA, said in an executive summary.
CDER’s approvals in 2024 were drugs that aided in the diagnosis, prevention, and treatment of infectious diseases, neurological conditions, opioid use, lung diseases, cancers, and diseases associated with the heart, blood and kidneys. CDER said that 37 of 50 (74%) approved drugs were approved on the first review cycle, and 34 of 50 drugs (68%) were approved in the US before other countries. The Center also improved in 2024 with regard to the percentage of novel drugs that met or exceeded their Prescription Drug User Fee Act (PDUFA) goal dates compared with the previous year (94% vs 89%).
Overall, 26 of 50 (52%) novel drug approvals in 2024 were for rare diseases. Cavazzoni said the number of drugs granted orphan drug designation comprising more than half of approvals highlights the Center’s “steadfast commitment to address the huge unmet medical need in rare diseases.”
CDER noted that 24 of 50 (48%) novel drugs approved in 2024 were first-in-class, which included the combination medication Anktiva (nogapendekin alfa inbakicept-pmln) for certain cases of invasive bladder cancer, Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) for the treatment of neurological symptoms associated with Niemann-Pick disease type C, Bizengri (zenocutuzumab-zbco) to treat certain types of advanced unresectable or metastatic pancreatic adenocarcinoma, Cobenfy (xanomeline, trospium chloride) as a treatment for schizophrenia in adults, Crenessity (crinecerfont) to treat classic congenital adrenal hyperplasia, Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy in children and adults, Hympavzi (marstacimab-hncq) to prevent bleeding episodes in patients with certain types of hemophilia A and B, Imdelltra (tarlatamab-dlle) for treating extensive-stage small-cell lung cancer, Iqirvo (elafibranor) for treating primary biliary cholangitis, the optical imaging agent Lumisight (pegulicianine) for use intraoperatively to detect cancerous tissue in patients with breast cancer, Nemluvio (nemolizumab-ilto) for treating prurigo nodularis, Niktimvo (axatilimab-csfr) to treat chronic graft-versus-host disease, Revuforj (revumenib) as a treatment for certain types of relapsed or refractory acute leukemia, Rezdiffra (resmetirom) for patients with noncirrhotic non-alcoholic steatohepatitis, Rytelo (imetelstat) for treatment of low- to intermediate-1 risk myelodysplastic syndromes, Tryngolza (olezarsen) to reduce triglycerides in patients with familial chylomicronemia syndrome, Tryvio (aprocitentan) as a treatment for hypertension, Voydeya (danicopan) as an add-on therapy for extravascular hemolysis resulting from paroxysmal nocturnal hemoglobinuria, Vyloy (zolbetuximab-clzb) as a treatment for gastric cancer, Winrevair (sotatercept-csrk) to treat pulmonary arterial hypertension, Xolremdi (mavorixafor) for use in patients with WHIM syndrome, Zelsuvmi (berdazimer) for the treatment of molluscum contagiosum, and Ziihera (zanidatamab-hrii) to treat certain kinds of biliary tract cancer.
In addition, 33 of 50 (66%) used at least one expedited review program, with 22 of 50 (44%) granted fast track status, 18 of 50 (36%) designated as a breakthrough therapy, 28 of 50 (56%) granted priority review status, and 7 of 50 (14%) approved under accelerated approval.
CDER approved 18 biosimilars, which included 8 biosimilars for 3 reference products without an existing biosimilar, and approved their 60th biosimilar in 2024 since the first biosimilar was approved in 2015.
FDA’s Center for Biologics Evaluation and Research (CBER) also released a list of 10 therapies that received Biological License Application (BLA) approval in 2024. CBER’s list of approved therapeutics in 2024 includes treatments for aromatic L-amino acid decarboxylase deficiency (Kebilidi), relapsed or refractory B-cell precursor acute lymphoblastic leukemia (Aucatzyl), a subset of patients with unresectable or metastatic synovial sarcoma (Tecelra), primary humoral immunodeficiency in children 2 years and younger (Yimmugo), a subset of patients with moderate to severe hemophilia B (Beqvez), children with certain types of metachromatic leukodystrophy (Lenmeldy), a subset of patients with unresectable or metastatic melanoma (Amtagvi), adolescents with transfusion-dependent ß-thalassemia (Casgevy), as well as indications for the active immunization for the prevention of invasive disease due to certain Streptococcus pneumoniae serotypes (Capvaxive) and lower respiratory tract disease due to respiratory syncytial virus in adults 60 years and older (Mresvia).
In their report, CDER highlighted the collaborative process that occurs when they receive a drug application, which involves experts in science, regulation, and policy at CDER and other parts of FDA, and said they perform a timely analysis of the safety and effectiveness of the drug while also considering the indication, labeling, safety issues, and external input from interested parties.
“We take our regulatory decision-making seriously, because we know our decisions affect the health and well-being of patients and consumers nationwide,” the report concluded.
New Drug Therapy Approvals 2024
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