Ojemda-Tovorafenib
Ojemda is the first FDA-approved targeted therapy for children 6 months and older with relapsed or treatment-resistant pediatric low-grade glioma (pLGG) caused by certain BRAF gene changes.
Xolremdi-Mavorixafor
Xolremdi is a once-daily pill that helps people aged 12 years and older with WHIM syndrome by increasing important white blood cells to strengthen the body’s defense against infections.
Rytelo-Imetelstat
Rytelo is a prescription treatment for adults with low-to-intermediate-1 risk myelodysplastic syndromes (MDS) who have anemia and need frequent blood transfusions, and who haven’t responded to or can’t take erythropoiesis-stimulating agents (ESAs).
Iqirvo-Elafibranor
Iqirvo is used with ursodeoxycholic acid to treat primary biliary cholangitis. It works by targeting key pathways in the liver, including PPAR alpha and PPAR delta.
Sofdra-Sofpironium
Sofdra is a topical anticholinergic gel for adults and children aged 9 and up suffering from underarm excessive sweating (primary axillary hyperhidrosis). Applied at bedtime, it helps reduce sweat production by blocking signals to sweat glands
Leqselvi-Deuruxolitinib
Leqselvi is an oral tablet prescribed for adults with severe alopecia areata, a condition that causes widespread hair loss. It works by blocking specific Janus kinase enzymes involved in the autoimmune process, leading to significant hair regrowth over 30% of patients saw at least 80% scalp coverage in clinical trials
Ohtuvayre-Ensifentrine
Ohtuvayre is an inhaled medication for adults with moderate to severe COPD, offering a new option beyond traditional steroids and bronchodilators. It targets two enzymes (PDE3 and PDE4) to both open airways and reduce lung inflammation
Grafapex-Treosulfan
Grafapex is used with fludarabine to prepare adults and children (≥1 year) with AML or MDS for stem cell transplant. It helps replace traditional regimens, offering similar effectiveness with potentially fewer side effects
Journavx-Suzetrigine
Journavx is a non‑opioid oral painkiller approved for adults with moderate‑to‑severe acute pain. It blocks sodium‑channel pain signals in the body, relieving pain without addiction risk.
Gomekli-Mirdametinib
Gomekli treats adults and children (≥2 years) with NF1-related plexiform neurofibromas that can’t be fully removed. It inhibits MEK enzymes to slow tumor growth and reduce tumor size.
