Rare & genetic: new FDA approvals
Brand, generic name, approval date and the use the FDA approved it for. No recommendations. Updated 2026-10-12.
Weight loss & diabetesCancerBrain & mental healthInfections & vaccinesHeart & bloodImmune & inflammatoryKidney, liver & lungEyeWomen's & hormonal healthPain & anesthesiaDiagnostic & imaging agentsRare & geneticOther
EmcitateRare & genetic
tiratricolTo treat peripheral thyrotoxicosis in patients with MCT8 deficiencyAtebriozRare & genetic
zilurgisertibTo reduce the volume of total new heterotopic ossification in adults and pediatric patients 12 years and older with fibrodysplasia ossificans progressivaPasatruRare & genetic
garetosmab-grts · Regeneron Pharmaceuticals, Inc.To reduce new heterotopic ossification and clinician-assessed disease flare-ups in adults with fibrodysplasia ossificans progressivaSimtriyoRare & genetic
centanafadineTo treat attention-deficit hyperactivity disorderAvlayahRare & genetic
tividenofusp alfa-eknm · Denali Therapeutics Inc..To treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II)YuviwelRare & genetic
navepegritide · AscendisTo increase linear growth in pediatric patients 2 years and older with achondroplasia with open epiphysesLoargysRare & genetic
pegzilarginase-nbln · Immedica Pharma AbTo treat hyperarginemia in adults and pediatric patients two years and older with Arginase 1 Deficiency, in conjunction with dietary protein restrictionZycuboRare & genetic
copper histidinate · Sentynl Theraps Inc.To treat Menkes diseaseKygevviRare & genetic
doxecitine and doxribtimine · Ucb Inc.To treat thymidine kinase 2 deficiency in patients who start to show symptoms when they are 12 years old or youngerForzinityRare & genetic
elamipretide · Stealth BiotherapsTo improve muscle strength in patients with Barth syndrome weighing at least 30 kgDawnzeraRare & genetic
donidalorsen · Ionis Pharms Inc.To prevent attacks of hereditary angioedemaSephienceRare & genetic
sepiapterin · Ptc TherapTo treat hyperphenylalaninemia in patients with sepiapterin-responsive phenylketonuria, in conjunction with a phenylalanine-restricted dietEkterlyRare & genetic
sebetralstat · KalvistaTo treat acute attacks of hereditary angioedemaAndembryRare & genetic
garadacimab-gxii · Csl Behring LLCTo prevent attacks of hereditary angioedemaAqneursaRare & genetic
levacetylleucineTo treat Niemann-Pick disease type CMiplyffaRare & genetic
arimoclomol · Zevra DenmarkTo treat Niemann-Pick disease type CXolremdiRare & genetic
mavorixafor · X4 PharmsTo treat WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis)DuvyzatRare & genetic
givinostat · Italfarmaco SpaTo treat Duchenne muscular dystrophy in individuals aged 6 years and older