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New Drug Applications

The New Drug Application (NDA) is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the United States.
The purpose of a NDA is to provide enough information to permit the FDA to reach the following key decisions

  • Whether the drug is safe and effective in its proposed use(s), and whether the benefits of the drug outweigh the risks
  • Whether the drug's proposed labeling (package insert) is appropriate and what it should contain
  • Whether the methods used in manufacturing the drug and the controls used to maintain the drug's quality are adequate to preserve the drug's identity, strength, quality, and purity

For more information on New Drug Applications, please visit the FDA's How Drugs are Developed and Approved page.


Company: Biogen
Treatment for: Alzheimer's Disease

Aducanumab (BIIB037) is an investigational human recombinant monoclonal antibody (mAb) in development for the treatment of early Alzheimer’s disease (AD).


Company: Blueprint Medicines Corporation
Treatment for: Thyroid Cancer

Pralsetinib is an oral selective RET kinase inhibitor in development for the treatment of patients with advanced or metastatic RET mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancers.

Furoscix (furosemide)

Company: scPharmaceuticals, Inc.
Treatment for: Congestive Heart Failure

Furoscix (furosemide) is a subcutaneous formulation of the approved diuretic furosemide delivered via a wearable, pre-programmed on-body drug delivery system. Furoscix is in development for treatment of congestion in patients with heart failure, and has the potential to provide an outpatient alternative for the treatment of worsening heart failure due to congestion.


Company: Mezzion Pharma Co. Ltd.
Treatment for: Single Ventricle Heart Disease

Udenafil is a long acting, highly selective phosphodiesterase-5 inhibitor in development for the treatment of patients who have undergone the Fontan operation for single ventricle heart disease.

VP-102 (cantharidin) Topical Solution

Company: Verrica Pharmaceuticals Inc.
Treatment for: Molluscum Contagiosum

VP-102 (cantharidin) is a topical terpenoid in development for the treatment of molluscum contagiosum.


Company: Sarepta Therapeutics, Inc.
Treatment for: Duchenne Muscular Dystrophy

Casimersen is a phosphorodiamidate morpholino oligomer in development for the treatment of patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.

E4/DRSP (estetrol and drospirenone)

Company: Mayne Pharma Group Limited
Treatment for: Contraception

E4/DRSP (estetrol and drospirenone) is a novel combined hormonal oral contraceptive pill in development for the prevention of pregnancy.


Company: Orphazyme A/S
Treatment for: Niemann-Pick Disease, type C

Arimoclomol is an investigational Heat-Shock Protein amplifier in development for the treatment of Niemann-Pick disease Type C (NPC).


Company: Zealand Pharma A/S
Treatment for: Hypoglycemia

HypoPal® Rescue Pen (dasiglucagon) is a glucagon analog in development for the treatment of severe hypoglycemia.

Zimhi (naloxone hydrochloride) Injection

Company: Adamis Pharmaceuticals Corporation
Treatment for: Opioid Overdose

Zimhi (naloxone hydrochloride) is a high-dose formulation of the approved opioid antagonist naloxone in development for the treatment of opioid overdose.

Zokinvy (lonafarnib)

Company: Eiger BioPharmaceuticals, Inc.
Treatment for: Progeria and Progeroid Laminopathies

Zokinvy (lonafarnib) is an oral farnesyltransferase inhibitor (FTI) in development for the treatment of Progeria and Progeroid Laminopathies.


Company: Sanofi
Treatment for: Cold Agglutinin Disease (CAD)

Sutimlimab is a first-in-class selective inhibitor of complement C1s in development for the treatment of hemolysis in adult patients with cold agglutinin disease (CAD).


Company: Rhythm Pharmaceuticals, Inc.
Treatment for: Obesity

Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist in development for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.

lisocabtagene maraleucel

Company: Bristol-Myers Squibb Company
Treatment for: Large B-Cell Lymphoma

Lisocabtagene maraleucel (liso-cel) is an investigational chimeric antigen receptor (CAR) T-cell therapy in development for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL).

CC-486 (azacitidine)

Company: Bristol Myers Squibb
Treatment for: Acute Myeloid Leukemia

CC-486 (azacitidine) is an investigational oral hypomethylating agent in development for the maintenance treatment of adult patients in remission with acute myeloid leukemia.

See also: Generic Approvals, New Drug Approvals, Recent Additions to, Alphabetical Listing of all New Drug Applications, FDA Approval Process

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