FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 970 total
Drug information is sourced from FDA labels and prescribing information. Always verify current prescribing information with your pharmacist or prescriber before starting, stopping, or changing any medication.
Bizengri
zenocutuzumab-zbco
Partner Therapeutics, Inc.
1 INDICATIONS AND USAGE BIZENGRI® is a bispecific HER2- and HER3-directed antibody indicated for the treatment of: Adults with advanced, unresectable …
Retrovir
zidovudine
Glaxo Wellcome Inc.
1 INDICATIONS AND USAGE RETROVIR is a nucleoside analogue reverse transcriptase inhibitor indicated for: • Treatment of Human Immunodeficiency Virus (…
Komzifti
ziftomenib
Kura Oncology, Inc.
1 INDICATIONS AND USAGE KOMZIFTI is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susc…
Zilbrysq
ZILUCOPLAN
UCB, Inc.
1 INDICATIONS AND USAGE ZILBRYSQ is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine re…
Galzin
Zinc acetate
Eton Pharmaceuticals, Inc
INDICATIONS AND USAGE Zinc acetate therapy is indicated for maintenance treatment of patients with Wilson’s disease who have been initially treated wi…
Vyloy
zolbetuximab-clzb
Astellas Pharma Global Development Inc.
1 INDICATIONS AND USAGE VYLOY, in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment o…
Zometa, Zabel
Zoledronate
Novartis Pharmaceuticals Corp.
Treatment of hypercalcemia of malignancy.…
Papzimeos
zopapogene imadenovec-drba
Precigen, Inc.
1 INDICATIONS AND USAGE PAPZIMEOS is indicated for the treatment of adults with recurrent respiratory papillomatosis. PAPZIMEOS™ is a non-replicating …