FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 968 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.
Rivfloza
NEDOSIRAN
Novo Nordisk
1 INDICATIONS AND USAGE RIVFLOZA is indicated to lower urinary oxalate levels in children 2 years of age and older and adults with primary hyperoxalur…
Arranon
nelarabine
Novartis Pharmaceuticals Corp.
1 INDICATIONS AND USAGE ARRANON is indicated for the treatment of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL…
Jascayd
NERANDOMILAST
Boehringer Ingelheim Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE JASCAYD is a phosphodiesterase 4 (PDE4) inhibitor indicated for: The treatment of idiopathic pulmonary fibrosis in adult patie…
LAMPIT
nifurtimox
Bayer HealthCare Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE LAMPIT is indicated in pediatric patients (birth to less than 18 years of age and weighing at least 2.5 kg) for the treatment …
Danziten
nilotinib
Azurity Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE DANZITEN is a kinase inhibitor indicated for the treatment of: • Adult patients with newly diagnosed Philadelphia chromosome p…
NYMALIZE
nimodipine
Arbor Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE NYMALIZE is indicated for the improvement of neurological outcome by reducing the incidence and severity of ischemic deficits …
Ofev
NINTEDANIB
Boehringer Ingelheim Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE OFEV is a kinase inhibitor indicated in adults for: Treatment of idiopathic pulmonary fibrosis (IPF) ( 1.1 ) Treatment of chro…
Imaavy
NIPOCALIMAB
Janssen Biotech, Inc.
1 INDICATIONS AND USAGE IMAAVY is indicated for the treatment of generalized myasthenia gravis (gMG) in adult and pediatric patients 12 years of age a…
Zejula
niraparib
GlaxoSmithKline, LLC.
1 INDICATIONS AND USAGE ZEJULA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated: • for the maintenance treatment of adult patients with ad…
Ogsiveo
nirogacestat
SpringWorks Therapeutics (EMD Serono / Merck KGaA)
1 INDICATIONS AND USAGE OGSIVEO is indicated for adult patients with progressing desmoid tumors who require systemic treatment. OGSIVEO is a gamma sec…
Alinia
Nitazoxanide
Romark Laboratories, L.C.
Treatment of diarrhea caused by Cryptosporidium parvum and Giardia lamblia…
Harliku
nitisinone
Cycle Pharmaceuticals Ltd.
1 INDICATIONS AND USAGE HARLIKU TM is indicated for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU). HARLIKU …
INOmax
Nitric oxide
Mallinckrodt Hospital Products
1 INDICATIONS AND USAGE INOmax ® is indicated to improve oxygenation and reduce the need for extracorporeal membrane oxygenation in term and near-term…
OPDIVO
NIVOLUMAB
E.R. Squibb & Sons, L.L.C.
1 INDICATIONS AND USAGE OPDIVO QVANTIG is a combination of nivolumab, a programmed death receptor-1 (PD-1)-blocking antibody, and hyaluronidase, an en…
OPDIVO� and YERVOY�
nivolumab and ipilimumab
Bristol-Myers Squibb Company
YERVOY� (ipilimumab), in combination with nivolumab, is indicated for the first-line treatment of adult patients with unresectable malignant pleural…
Opdualag
nivolumab and relatlimab-rmbw
Bristol-Myers Squibb Company
1 INDICATIONS AND USAGE OPDUALAG™ is indicated for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metasta…
Spinraza
NUSINERSEN
Biogen Inc.
1 INDICATIONS AND USAGE SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a surviv…
Aucatzyl
obecabtagene autoleucel
Autolus Incorporated, US subsidiary of Autolus Limited
1 INDICATION AND USAGE AUCATZYL is indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (AL…
OCALIVA
obeticholic acid
Intercept Pharmaceuticals, Inc.
Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as mo…
Anthim
obiltoxaximab
Elusys Therapeutics, Inc.
1 INDICATIONS AND USAGE ANTHIM ® is a monoclonal antibody directed against the protective antigen of Bacillus anthracis . It is indicated in adult and…
GAZYVA
obinutuzumab
Genentech Inc., a member of the Roche Group
1 INDICATIONS AND USAGE GAZYVA is a CD20-directed cytolytic antibody indicated: in combination with chlorambucil, for the treatment of patients with p…
Sandostatin LAR
Octreotide
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE SANDOSTATIN LAR DEPOT 10 mg, 20 mg, and 30 mg is indicated in patients in whom initial treatment with Sandostatin Injection ha…
MYCAPSSA (formerly Octreolin)
octreotide (oral)
Chiesi USA, Inc.
Mycapssa (octreotide) is indicated for the long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with o…
Bylvay
ODEVIXIBAT
Ipsen Biopharmaceuticals, Inc.
1 INDICATIONS AND USAGE BYLVAY is an ileal bile acid transporter (IBAT) inhibitor indicated for: Progressive Familial Intrahepatic Cholestasis (PFIC) …
ARZERRA
ofatumumab
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE Chronic Lymphocytic Leukemia (CLL) ARZERRA (ofatumumab) is indicated: in combination with chlorambucil, for the treatment of p…
Ocuflox Ophthalmic Solution
Ofloxacin
Allergan, Inc.
INDICATIONS AND USAGE Ofloxacin Otic Solution 0.3% is indicated for the treatment of infections caused by susceptible isolates of the designated micro…
Lynparza
olaparib
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE Lynparza is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated: Ovarian cancer • for the maintenance treatment of adult…
Lartruvo
olaratumab
Eli Lilly and Company
In combination with doxorubicin, for the treatment of adult patients with soft tissue sarcoma (STS) with a histologic subtype for which an anthracycli…