Emcitate-Tiratricol
Emcitate (tiratricol) is an oral thyroid hormone analog approved for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. MCT8 deficiency is a rare genetic disorder caused by mutations affecting monocarboxylate transporter 8, which plays an important role in transporting thyroid hormones into cells. Tiratricol can […]
Atebrioz-Zilurgisertib
Atebrioz (zilurgisertib) is an oral activin receptor-like kinase 2 (ALK2) inhibitor indicated to reduce the volume of new heterotopic ossification in adults and pediatric patients 12 years of age and older with fibrodysplasia ossificans progressiva (FOP). FOP is a rare genetic disorder in which connective tissues such as muscles, tendons, and ligaments progressively transform into […]
Juvmo-Tavapadon
Juvmo (tavapadon) is an oral dopamine agonist indicated for the treatment of Parkinson’s disease in adults. Tavapadon is a selective partial agonist of dopamine D1 and D5 receptors, offering a differentiated mechanism from dopamine agonists that primarily target D2 and D3 receptors. Juvmo can be used as monotherapy in patients with early Parkinson’s disease or […]
Lyrfigtu-Lirafugratinib
Lyrfigtu (lirafugratinib) is an oral kinase inhibitor indicated for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement. Lirafugratinib targets fibroblast growth factor receptor signaling, a pathway that can contribute to tumor growth and progression in cancers driven by FGFR2 alterations. Lyrfigtu provides a biomarker-driven treatment […]
Pixclara-Floretyrosine F 18
Pixclara (floretyrosine F18) is a radioactive diagnostic agent used with positron emission tomography (PET) to help differentiate recurrent or progressive glioma from treatment-related changes in adults and pediatric patients 1 month of age and older. The fluorine-18-labeled imaging agent is taken up through amino acid transport pathways, allowing areas of increased tracer uptake to be […]
Etcamah-Camizestrant
Etcamah (camizestrant) is an oral selective estrogen receptor degrader (SERD) and estrogen receptor antagonist indicated, in combination with a CDK4/6 inhibitor, for adults with hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer with an ESR1 mutation detected during aromatase inhibitor and CDK4/6 inhibitor therapy. Camizestrant works by binding to and degrading estrogen receptors, helping […]
Zanvastro-Zilganersen
Zanvastro (zilganersen) is an antisense oligonucleotide indicated for the treatment of Alexander disease in pediatric and adult patients. It targets glial fibrillary acidic protein (GFAP) messenger RNA to reduce the production of abnormal GFAP protein, which accumulates in the brain and contributes to disease progression. Administered by intrathecal injection, Zanvastro is designed to target an […]
Xenoview-Hyperpolarized Xe-129
Xenoview provided a functional alternative in lung imaging, where standard scans show structure well but reveal little about which regions actually ventilate. Its xenon nuclei are hyperpolarized to make them detectable by MRI, producing regional ventilation maps without ionizing radiation. Inhaled during a single breath hold, it is approved for MRI evaluation of lung ventilation […]
Sunlenca-Lenacapavir
Sunlenca (lenacapavir) introduced an entirely new mechanism for patients with multidrug-resistant HIV, who can exhaust every available class and be left without a viable regimen. As the first capsid inhibitor approved, it disrupts multiple stages of the viral lifecycle, so no existing resistance pathway applies. Subcutaneous injections every six months follow oral loading, used with […]
Krazati-Adagrasib
Krazati (adagrasib) followed sotorasib into the KRAS G12C space, arriving as the second approved inhibitor of a target once considered undruggable. Adagrasib binds the mutant protein irreversibly and covalently, and its longer half-life and central nervous system penetration distinguish it pharmacologically from sotorasib. The twice-daily tablet treats adults with KRAS G12C-mutated locally advanced or metastatic […]
