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.

ZS-9 (sodium zirconium cyclosilicate)

Company: ZS Pharma, Inc.
Treatment for: Hyperkalemia

ZS-9 (sodium zirconium cyclosilicate) is a crystalline lattice potassium ion-trap in development for the treatment of hyperkalemia.

Dyanavel XR (amphetamine)

Company: Tris Pharma, Inc.
Treatment for: ADHD

Dyanavel XR (amphetamine) is an extended-release oral suspension formulation of amphetamine in development for the treatment of children with ADHD.

Orkambi (ivacaftor and lumacaftor)

Company: Vertex Pharmaceuticals Incorporated
Treatment for: Cystic Fibrosis

Orkambi (ivacaftor and lumacaftor) is a CFTR potentiator and CFTR corrector combination designed to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation.

Zalviso (sufentanil) Sublingual Microtablet System

Company: AcelRx Pharmaceuticals, Inc.
Treatment for: Pain

Zalviso (sufentanil sublingual microtablet system) is a patient-activated, non-invasive analgesic system for the management of moderate-to-severe acute pain in adult patients in the hospital setting.

Zinbryta (daclizumab high-yield process)

Company: AbbVie, Inc. and Biogen
Treatment for: Multiple Sclerosis

Zinbryta (daclizumab high-yield process) is an investigational humanized monoclonal antibody in development for the treatment of relapsing forms of multiple sclerosis (MS).

AuriPro (ciprofloxacin)

Company: Otonomy, Inc.
Treatment for: Otitis Media

AuriPro (ciprofloxacin) is a sustained-exposure otic fluoroquinolone formulation in development for the treatment of middle ear effusion in pediatric patients undergoing tympanostomy tube placement (TTP) surgery.


Company: Prosensa Holding N.V.
Treatment for: Duchenne Muscular Dystrophy

Drisapersen is an investigational antisense oligonucleotide in development for the treatment of Duchenne Muscular Dystrophy.


Company: Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment for: Reversal Agent for Pradaxa

Idarucizumab is a humanized antibody fragment, or Fab, being investigated as a specific reversal agent for the anticoagulant effect of dabigatran (Pradaxa).


Company: Ultragenyx Pharmaceutical Inc.
Treatment for: Glucose Transporter Type-1 Deficiency Syndrome

Triheptanoin is a synthetic triglyceride compound in development for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS).

Reolysin (pelareorep)

Company: Oncolytics Biotech Inc.
Treatment for: Ovarian Cancer, Pancreatic Cancer, Malignant Glioma

Reolysin (pelareorep) is a proprietary formulation of the human reovirus (Respiratory Enteric Orphan Virus) in development for the treatment of various cancers and cell proliferative disorders.


Company: The Medicines Company
Treatment for: Platelet Aggregation Inhibition

Cangrelor is an intravenous antiplatelet drug in development for use in patients undergoing percutaneous coronary intervention (PCI) and those that require bridging from oral antiplatelet therapy to surgery.

Androxal (enclomiphene)

Company: Repros Therapeutics Inc.
Treatment for: Hypogonadism -- Male

Androxal (enclomiphene) is a non-steroidal estrogen receptor antagonist in development for secondary hypogonadism in overweight men wishing to restore normal testicular function.

Gencaro (bucindolol)

Company: ARCA biopharma, Inc.
Treatment for: Congestive Heart Failure

Gencaro (bucindolol) is an investigational and pharmacologically unique beta-blocker and mild vasodilator in development for the treatment of chronic heart failure.

Macrilen (macimorelin acetate)

Company: Aeterna Zentaris Inc.
Treatment for: Adult Human Growth Hormone Deficiency

Macrilen (macimorelin) is a ghrelin agonist in development for use in evaluating adult growth hormone deficiency (AGHD).

emtricitabine and tenofovir alafenamide

Company: Gilead Sciences, Inc.
Treatment for: HIV Infection

Emtricitabine/tenofovir alafenamide is an investigational nucleoside analog HIV-1 reverse transcriptase inhibitor (NRTI) and nucleotide reverse transcriptase inhibitor (NtRTI) fixed-dose combination in development for the treatment of HIV-1 infection.

Lyxumia (lixisenatide)

Company: Sanofi
Treatment for: Diabetes Type 2

Lyxumia (lixisenatide) is a once-daily prandial GLP-1 receptor agonist in development for the treatment of adults with type 2 diabetes mellitus.

Praluent (alirocumab)

Company: Sanofi and Regeneron Pharmaceuticals, Inc.
Treatment for: High Cholesterol

Praluent (alirocumab) an investigational monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) in development for the treatment of patients with high cholesterol.

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

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