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.
Ojjaara-Momelotinib
Ojjaara is an oral medication for adults with intermediate or high-risk myelofibrosis and anemia. It targets JAK1, JAK2, and ACVR1 to reduce spleen size, relieve symptoms, and improve anemia.
Exxua-Gepirone
Exxua is an extended-release oral antidepressant for adults with major depressive disorder. It selectively activates serotonin 5‑HT1A receptors, offering symptom relief with low risk of sexual side effects.
Velsipity-Etrasimod
Velsipity is a once-daily pill for adults with moderately to severely active ulcerative colitis. It modulates S1P receptors to reduce inflammation and maintain remission.
Pombiliti-Cipaglucosidase Alfa-Atga
Pombiliti, used with Opfolda (miglustat), treats adults with late-onset Pompe disease who aren’t responding to enzyme replacement therapy. It delivers an enzyme to break down muscle-accumulated glycogen, improving muscle strength and lung function.
Rivfloza-Nedosiran
Rivfloza is a once-monthly injection for children more than 2 years old and adults with primary hyperoxaluria type 1. It lowers urine oxalate levels by silencing a liver enzyme, helping protect kidney function.
