The FDA has approved Simponi (golimumab) for children with moderately to severely active ulcerative colitis (UC) who weigh at least 15 kilograms (33 pounds). Simponi is a lab-made antibody (biologic medicine) that works by blocking… Read more
Forzinity (elamipretide) is the first medicine to receive accelerated approval from the FDA for improving muscle strength in people with Barth syndrome who weigh at least 66 pounds (30 kilograms). Barth syndrome is an extremely… Read more
Vyjuvek (beremagene geperpavec-svdt) is the first topical gene therapy approved to heal wounds in adults and children with dystrophic epidermolysis bullosa (DEB). DEB is a rare genetic skin condition in which a faulty COL7A1 gene… Read more
Repatha (evolocumab) is an injectable medicine that helps lower levels of low-density lipoprotein cholesterol (LDL), often called “bad” cholesterol. Approved in 2015, Repatha can now be used in adults with poorly controlled LDL who haven’t been… Read more
Modeyso (dordaviprone) is a new oral medicine that treats a rare and aggressive brain tumor, called diffuse midline glioma (DMG), that has a tumor mutation known to promote tumor growth, called an H3 K27M mutation.… Read more
Alhemo (concizumab-mtci) is a medicine approved to help prevent bleeding in people aged 12 or older with hemophilia A or B who don’t have inhibitors to certain blood clotting proteins. Hemophilia is a rare condition… Read more
Skytrofa (lonapegsomatropin-tcgd) is a long-acting version of human growth hormone (hGH). It is already approved for children with growth hormone deficiency (GHD) and is now approved for adults with GHD. It slowly releases somatropin, the… Read more
Doptelet (avatrombopag) is a prescription medicine that helps the body make more platelets (cells that help stop bleeding). First approved in 2018 for adults, it is now also approved by the FDA for children aged… Read more
July 11, 2025 — The FDA has given full approval to Moderna’s COVID-19 shot, Spikevax, allowing it to be used in children 6 months to 11 years old, with some limits on who can get… Read more
Epkinly (epcoritamab-bysp) is a type of cancer treatment called a bispecific antibody that helps your immune system find and destroy certain cancer cells by connecting immune cells (T cells) to cancer cells (B cells). Epkinly… Read more