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2024 is an extraordinary year for drug discovery, with 21 small molecule drugs approved by the FDA pushing the boundaries of innovation and addressing some of the most critical medical challenges. Among these, rare diseases have experienced a transformative year, with drugs for rare conditions making up 23.8% of the approvals, reflecting a strong commitment to addressing unmet medical needs. Cancer drugs also made groundbreaking progress, comprising 19% of the total, ranking second, while blood diseases showed further exploration potential. Below are the 21 significant small molecule drugs approved by the FDA in 2024:
Rezdiffra is an oral selective thyroid hormone receptor-β (THR-β) agonist. Its active ingredient, resmetirom, reduces liver fat accumulation, scarring, and inflammation by activating thyroid hormone receptors in the liver. It is the first drug in 40 years to be approved by the FDA for treating adults with moderate-to-advanced liver fibrosis associated with metabolic dysfunction-related fatty liver disease (MASH), to be used alongside diet and exercise.
Tryvio is an endothelin receptor antagonist that inhibits endothelin (a vasoconstrictor peptide) from binding to its smooth muscle cell receptors, causing vasodilation and lowering blood pressure. It is the first and only endothelin receptor antagonist to inhibit the binding of endothelin-1 (ET-1) to both ETA and ETB receptors, marking the first new oral antihypertensive treatment approved in nearly 30 years.
Duvyzat is a histone deacetylase (HDAC) inhibitor used to reduce inflammation and muscle loss in patients with DMD aged six years and older. It is the first non-steroidal drug approved for treating all DMD gene mutations.
Zelsuvmi is a nitric oxide donor with antiviral properties, approved for treating molluscum contagiosum in adults and pediatric patients aged 1 year and older. It is the first new drug approved for treating molluscum contagiosum by the FDA and can be used in home or non-clinical settings.
Zevtera is an intravenous cephalosporin antibiotic that rapidly kills various Gram-positive and Gram-negative bacteria. It is used to treat bloodstream infections (SAB) in adults, including right-sided infective endocarditis; acute bacterial skin and skin structure infections (ABSSSI); and community-acquired bacterial pneumonia (CABP) in both adults and children aged 3 months to 18 years.
Ohtuvayre is a first-in-class dual inhibitor of PDE3 and PDE4, designed to provide both bronchodilation and anti-inflammatory effects with a single compound. It is the first inhaled product in over 20 years to be approved for COPD maintenance treatment with a novel mechanism of action.
Voranigo is a first-in-class IDH1 and IDH2 inhibitor, approved for use in patients aged 12 and older with grade 2 astrocytomas or oligodendrogliomas harboring IDH1 or IDH2 mutations. It is the first systemic therapy approved for this indication.
Leqselvi is an oral JAK1/JAK2 inhibitor designed to block the immune signals attacking hair follicles, promoting hair regrowth in adults with severe alopecia areata. It has received FDA breakthrough therapy designation.
Flyrcado is a radioactive diagnostic agent used in positron emission tomography (PET) myocardial perfusion imaging (MPI) to detect coronary artery disease (CAD). It is the first PET MPI drug approved for CAD detection.
Ojemda is an oral brain-penetrant selective RAF kinase inhibitor, approved for the treatment of children aged 6 months and older with recurrent or refractory low-grade gliomas with BRAF fusions, rearrangements, or V600 mutations.
Iqirvo is a peroxisome proliferator-activated receptor (PPAR) agonist approved for the treatment of primary biliary cholangitis, marking the first new drug approved in nearly a decade for this rare liver disease.
Aqneursa is a modified amino acid designed to cross the blood-brain barrier and restore mitochondrial and lysosomal function in patients with Niemann-Pick type C disease. It is FDA-approved for rare pediatric conditions and has received orphan drug, pediatric rare disease, and fast-track designations.
Cobenfy combines xanomeline and trospium chloride to activate brain muscarinic receptors while reducing peripheral muscarinic effects. It is the first cholinergic receptor-targeting antipsychotic approved for schizophrenia treatment.
Vafseo is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor used to treat anemia in adult patients with chronic kidney disease (CKD). It has been approved in 37 countries.
Sofdra is an anticholinergic agent that blocks sweat signals, providing relief for patients aged 9 and older with primary axillary hyperhidrosis. It is the first FDA-approved treatment for this condition.
Inavolisib is a highly selective PI3Kα inhibitor used in combination with palbociclib and fulvestrant for advanced HR+HER2- breast cancer with PIK3CA mutations.
Company: Zevra Therapeutics
Miplyffa is a first-in-class oral co-inducer of the heat shock response (HSP) designed for Niemann-Pick type C disease treatment. It is the first FDA-approved drug for NPC.
Xolremdi is the first CXCR4 antagonist developed specifically for WHIM syndrome treatment, a rare immune disorder, and was granted breakthrough therapy designation.
Livdelzi is an oral PPAR-δ agonist approved in combination with UDCA for PBC patients who do not respond to UDCA. It offers statistically significant improvements in key biomarkers compared to a placebo.
Voydeya is a first-in-class oral small molecule approved as an adjunct therapy to complement C5 inhibitors like Ultomiris for treating PNH-related extravascular hemolysis.
Lazcluze is a highly selective, third-generation oral EGFR tyrosine kinase inhibitor (TKI) with the ability to cross the blood-brain barrier, approved for treating EGFR-exon 19 deletions or L858R mutations in NSCLC.
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