| FDA approves therapy for rare blood disorder in pediatric patients 12 years and older | 1/5/2026 | Cablivi | caplacizumab-yhdp |
| FDA approves first oral treatment for anemia in thalassemia, an inherited blood disorder | 1/5/2026 | Aqvesme | mitapivat |
| FDA approves first drug to treat serious complication of stem cell transplant | 1/5/2026 | Yartemlea | narsoplimab-wuug |
| FDA approves first prescription oral medicine for iron deficiency in pediatric patients ages 10 and older | 12/22/2025 | Accrufer | ferric maltol |
| FDA approves drug to improve functional capacity and symptoms in adults with rare inherited heart condition | 12/22/2025 | Myqorzo | aficamten |
| FDA approves drug to treat chronic, progressive lung disease | 12/19/2025 | Jascayd | nerandomilast |
| FDA approves drug for type of abnormally fast heart rhythm | 12/15/2025 | Cardamyst | etripamil |
| FDA approves a new treatment for primary immunoglobulin A nephropathy | 11/25/2025 | Voyxact | sibeprenlimab-szsi |
| FDA approves drug to reduce triglycerides in adults with familial chylomicronemia syndrome | 11/18/2025 | Redemplo | plozasiran |
| FDA approves first interchangeable biosimilar to Perjeta to treat certain types of breast cancer | 11/13/2025 | Poherdy | pertuzumab-dpzb |
| FDA approves 1st drug for children 7 years and older with irritable bowel syndrome with constipation | 11/5/2025 | Linzess | linaclotide |
| FDA approves 1st drug for thymidine kinase 2 deficiency, a very rare mitochondrial disease | 11/3/2025 | Kygevvi | doxecitine and doxribtimine |
| FDA approves drug to treat idiopathic pulmonary fibrosis | 10/7/2025 | Jascayd | nerandomilast |
| FDA approves new treatment for acromegaly, a rare endocrine disorder | 9/26/2025 | Palsonify | paltusotine |
| FDA approves drug to treat adults with persistent or chronic immune thrombocytopenia | 9/2/2025 | Wayrilz | rilzabrutinib |
| FDA approves treatment for serious liver disease known as ‘MASH’ | 8/15/2025 | Wegovy | semaglutide |
| FDA approves first treatment for adults with complement 3 glomerulopathy, a rare kidney disease, to reduce proteinuria | 3/20/2025 | Fabhalta | iptacopan |
| FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors | 12/20/2024 | Alhemo | concizumab-mtci |
| FDA approves drug to reduce triglycerides in adult patients with familial chylomicronemia syndrome | 12/19/2024 | Tryngolza | olezarsen |
| FDA approves drug for heart disorder caused by transthyretin-mediated amyloidosis | 11/25/2024 | Attruby | acoramidis |
| FDA approves new treatment for women with uncomplicated UTIs | 10/23/2024 | Orlynvah | sulopenem etzadroxil and probenecid |
| FDA approves imaging drug for evaluation of myocardial ischemia and infarction | 9/27/2024 | Flyrcado | flurpiridaz F 18 |
| FDA approves new drug for hypoparathyroidism, a rare disorder | 8/12/2024 | Yorvipath | palopegteriparatide |
| FDA approves treatment for adults with Alzheimer’s disease | 7/02/2024 | Kisunla | donanemab-azbt |
| FDA approves treatment for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults | 6/24/2024 | Vyvgart Hytrulo | efgartigimod alfa and hyaluronidase-qvfc |
| FDA approves first interchangeable biosimilars to Eylea to treat macular degeneration and other eye conditions | 5/20/2024 | Yesafili and Opuviz | aflibercept-jbvf and aflibercept-yszy |
| FDA approves first drug for WHIM syndrome, a rare disorder | 4/29/2024 | Xolremdi | mavorixafor |
| FDA approves imaging drug to assist in detection of cancerous tissue following lumpectomy | 4/17/2024 | Lumisight | pegulicianine |
| FDA approves first interchangeable biosimilars to Prolia and Xgeva to treat certain types of osteoporosis and prevent bone events in cancer | 3/5/2024 | Jubbonti and Wyost | Denosumab-bbdz |
| FDA approves new drug under special pathway for patients receiving hemodialysis | 11/15/2023 | Defencath | taurolidine and heparin |
| FDA approves first biosimilar to Actemra to treat adult and pediatric arthritis | 9/29/2023 | Tofidence | tocilizumab-bavi |
| FDA approves multiple generics of ADHD and BED treatment | 8/28/2023 | Vyvanse | lisdexamfetamine dimesylate |
| FDA approves first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease) | 8/18/2023 | Veopoz | pozelimab-bbfg |
| FDA approves first treatment for Fibrodysplasia Ossificans Progressiva | 8/17/2023 | Sohonos | palovarotene |
| FDA approves first treatment for molluscum contagiosum | 7/24/2023 | Ycanth | cantharidin |
| FDA approves first treatment for pediatric functional constipation | 6/12/2023 | Linzess | linaclotide |
| FDA approves treatment for relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma | 5/19/2023 | Epkinkly | epcoritamab-bysp |
| FDA approves first oral treatment for moderately to severely active Crohn’s disease | 5/18/2023 | Rinvoq | upadacitinib |
| FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene | 4/25/2023 | Qalsody | tofersen |
| FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome | 3/24/2023 | Joenja | leniolisib |
| FDA approves first treatment for Rett Syndrome | 3/13/2023 | Daybue | trofinetide |
| FDA approves first treatment for Friedreich’s ataxia | 2/28/2023 | Skyclarys | omaveloxolone |
| FDA approves first enzyme replacement therapy for rare alpha-mannosidosis | 2/17/2023 | Lamzede | velmanase alfa-tycv |
| FDA approves drug combination treatment for adults with asthma | 1/10/2023 | Airsupra | albuterol-budesonide |
| FDA approves imaging drug to help identify lung cancer lesions | 12/16/2022 | Cytalux | pafolacianine |
| FDA approves first treatment for prurigo nodularis | 9/28/2022 | Dupixent | dupilumab |
| FDA approves treatment to improve kidney function in adults with hepatorenal syndrome | 9/14/2022 | Terlivaz | terlipressin |
| FDA approves topical treatment addressing repigmentation in vitiligo in patients aged 12 and older | 7/19/2022 | Opzelura | ruxolitinib |
| FDA approves treatment for chronic weight management in pediatric patients aged 12 years and older | 6/24/2022 | Qsymia | phentermine and topiramate |
| FDA approves treatment for weight management in patients with Bardet-Biedl Syndrome aged 6 or older | 6/16/2022 | Imcivree | setmelanotide |
| FDA Approves Oral Form for the treatment of adults with amyotrophic lateral sclerosis (ALS) | 5/12/2022 | Radicava ORS | edaravone |
| FDA approves new drug to improve heart function in adults with rare heart condition | 4/29/2022 | Camzyos | mavacamten |
| FDA approves drug for treatment of seizures associated with rare disease in patients two years of age and older | 3/18/2022 | Ztalmy | ganaxolone |
| FDA approves drug for adults with rare form of bone marrow disorder | 2/28/2022 | Vonjo | pacritinib |
| FDA approves treatment for anemia in adults with rare inherited disorder | 2/17/2022 | Pyrukynd | mitapivat |
| FDA approves treatment for adults with rare type of anemia | 2/4/2022 | Enjaymo | sutimlimab-jome |
| FDA approves add-on therapy to lower cholesterol among certain high-risk adults | 12/22/2021 | Leqvio | inclisiran |
| FDA approves drug to treat, help prevent types of blood clots in certain pediatric populations | 12/20/2021 | Xarelto | rivaroxaban |
| FDA approves Tezspire as maintenance treatment for severe asthma | 12/17/2021 | Tezspire | tezepelumab-ekko |
| FDA approves drug to treat sickle cell disease in patients aged 4 up to 11 years | 12/17/2021 | Oxbryta | voxelotor |
| FDA approves first drug to decrease urine protein in IgA nephropathy, a rare kidney disease | 12/15/2021 | Tarpeyo | budesonide |
| FDA approves naloxone injection to counteract opioid overdoses | 10/15/2021 | ZIMHI | naloxone hydrochloride |
| FDA approves add-on drug for adults with rare form of blood vessel inflammation | 10/13/2021 | Tavneos | avacopan |
| FDA approves add-on drug for ages 10 & up with rare forms of high cholesterol | 9/24/2021 | Repatha | evolocumab |
| FDA approves new use of transplant drug based on real-world evidence | 7/16/2021 | Prograf | tacrolimus |
| FDA approves drug to reduce risk of serious kidney and heart complications in adults with chronic kidney disease associated with type 2 diabetes | 7/9/2021 | Kerendia | finerenone |
| FDA approves therapy for pediatric patients with serious rare blood disease | 6/7/2021 | Ultomiris | ravulizumab-cwvz |
| FDA approves drug to treat smallpox | 6/4/2021 | Tembexa | brincidofovir |
| FDA approves second PSMA-targeted PET imaging drug for men with prostate cancer | 5/27/2021 | Pylarify | piflufolastat F 18 |
| FDA approves new treatment for adults with serious rare blood disease | 5/14/2021 | Empaveli | pegcetacoplan |
| FDA approves add-on therapy for patients with genetic form of severely high cholesterol | 4/1/2021 | Praluent | alirocumab |
| FDA approves first treatment for disease that causes recurrent inflammation in sac surrounding heart | 3/18/2021 | Arcalyst | rilonacept |
| FDA approves add-on therapy for patients with genetic form of severely high cholesterol | 2/11/2021 | Evkeeza | evinacumab-dgnb |
| FDA approves treatment for ebola virus | 12/21/2020 | Ebanga | Ansuvimab-zykl |
| FDA approves weight management drug for patients aged 12 and older | 12/4/2020 | Saxenda | liraglutide |
| FDA approves first treatment for weight management for people with certain rare genetic conditions | 11/27/2020 | Imcivree | setmelanotide |
| FDA’s approval of Veklury (remdesivir) for the treatment of COVID-19—the science of safety and effectiveness | 10/22/2020 | Veklury | remdesivir |
| FDA approves weekly therapy for adult growth hormone deficiency | 9/1/2020 | Sogroya | somapacitan |
| FDA approves new treatment for rare hereditary disease | 8/23/2018 | Takhzyro | lanadelumab |
