Orserdu-Elacestrant
Orserdu is a once-daily selective estrogen receptor degrader (SERD) approved for ER-positive, HER2-negative, ESR1-mutated, advanced or metastatic breast cancer in adults after endocrine therapy.
Jesduvroq-Daprodustat
Jesduvroq is an oral HIF-PHI approved for the treatment of anemia in adults with chronic kidney disease on dialysis. It was voluntarily withdrawn in the U.S. for non-safety-related reasons but remains FDA-listed.
Lamzede-Velmanase Alfa-Tycv
Lamzede is the first enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis. It improves enzyme activity and reduces the buildup of serum oligosaccharides.
Filspari-Sparsentan
Filspari is an oral dual endothelin and angiotensin receptor blocker approved to reduce proteinuria and slow kidney function decline in adults with IgA nephropathy.
Daybue-Trofinetide
Daybue is the first FDA-approved oral treatment for Rett syndrome in patients 2 years of age and older. It targets neurological function to address core symptoms of the disorder.
Skyclarys-Omaveloxolone
Skyclarys is the first FDA-approved treatment for Friedreich’s ataxia in patients aged 16 years and older. It helps reduce physical impairment associated with the condition.
Zavzpret-Zavegepant
Zavzpret is a fast-acting nasal spray for the acute treatment of migraine in adults. It provides pain relief and functional recovery, with effects starting as early as 30 minutes after use.
Sohonos-Palovarotene
Sohonos is a daily oral capsule for patients (8 + for females, 10 + for males) with fibrodysplasia ossificans progressiva (FOP), a condition causing abnormal bone growth. It slows new bone formation by activating retinoic acid receptors.
Veopoz-Pozelimab-Bbfg
Veopoz is an antibody injection that treats CHAPLE disease, a rare genetic disorder causing protein loss and chronic intestinal inflammation. It works by blocking complement C5, helping to stabilize protein levels and reduce hospital visits in both children and adults.
Aphexda-Motixafortide
Aphexda, used together with filgrastim, is a stem cell mobilizer for patients with multiple myeloma undergoing autologous transplant. By blocking CXCR4 receptors, it increases stem cells circulating in the blood for effective collection.
