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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.

Tofersen

Company: Biogen Inc.
Treatment for: Amyotrophic Lateral Sclerosis

Tofersen is an antisense drug in development for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).

ABBV-951 (foscarbidopa and foslevodopa)

Company: AbbVie Inc.
Treatment for: Parkinson's Disease

ABBV-951 (foscarbidopa/foslevodopa) is a solution of carbidopa and levodopa prodrugs for continuous subcutaneous delivery that is being investigated for the treatment of motor fluctuations in patients with advanced Parkinson's disease.

Eplontersen

Company: Ionis Pharmaceuticals
Treatment for: Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN)

Eplontersen is a LIgand-Conjugated Antisense (LICA) medicine in development for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).

Lumryz (sodium oxybate) - formerly FT218

Company: Avadel Pharmaceuticals plc
Treatment for: Narcolepsy

Lumryz (sodium oxybate) is a once-nightly formulation of the approved central nervous system depressant sodium oxybate in development for the treatment of excessive daytime sleepiness and cataplexy in adults with narcolepsy.

ADX-2191 (methotrexate)

Company: Aldeyra Therapeutics, Inc.
Treatment for: Primary Vitreoretinal Lymphoma

ADX-2191 (methotrexate) is an intravitreal formulation of the folate analog metabolic inhibitor methotrexate in development for the treatment of primary vitreoretinal lymphoma.

Omecamtiv mecarbil

Company: Cytokinetics, Inc.
Treatment for: Heart Failure with Reduced Ejection Fraction

Omecamtiv mecarbil is a first in class, small molecule cardiac myosin activator in development for the treatment of heart failure with reduced ejection fraction (HFrEF).

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.

Elranatamab

Company: Pfizer Inc.

Elranatamab is a B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb) in development for the treatment of patients with relapsed or refractory multiple myeloma.

Pozelimab

Company: Regeneron Pharmaceuticals, Inc.
Treatment for: CHAPLE

Pozelimab is a complement C5 inhibitor in development for the treatment of adults and children with CHAPLE disease.

CSF-1 (pilocarpine hydrochloride) Ophthalmic Solution

Company: Orasis Pharmaceuticals
Treatment for: Presbyopia

CSF-1 (pilocarpine) is a low dose ophthalmic formulation of the approved muscarinic cholinergic agonist pilocarpine in development to improve near vision for people with presbyopia.

Fezolinetant

Company: Astellas Pharma Inc.
Treatment for: Menopausal Disorders, Hot Flashes

Fezolinetant is a selective neurokinin 3 (NK3) receptor antagonist in development for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause.

Nyxol (phentolamine mesylate) Ophthalmic Solution

Company: Ocuphire Pharma, Inc.
Treatment for: Reversal of Pharmacologically Induced Mydriasis

Nyxol (phentolamine mesylate) is a nonselective alpha adrenergic antagonist ophthalmic formulation in development for the reversal of pharmacologically-induced mydriasis.

ATI-1501 (metronidazole)

Company: Appili Therapeutics Inc. and Saptalis Pharmaceuticals LLC
Treatment for: Bacterial Infection

ATI-1501 (metronidazole) is a liquid suspension formulation of the approved antibiotic metronidazole in development for the treatment of parasitic and anaerobic bacterial infections.

Reproxalap

Company: Aldeyra Therapeutics, Inc.
Treatment for: Dry Eye Disease

Reproxalap is a first-in-class small-molecule modulator of RASP (reactive aldehyde species) in development for the treatment of signs and symptoms of dry eye disease.

OX124 (naloxone)

Company: Orexo AB
Treatment for: Opioid Overdose

OX124 (naloxone) is a high-dose nasal formulation of the approved opioid antagonist naloxone in development as a rescue medication for opioid overdose.

Vamorolone

Company: Santhera Pharmaceuticals and ReveraGen BioPharma, Inc.
Treatment for: Duchenne Muscular Dystrophy

Vamorolone is a first-in-class steroidal anti-inflammatory in development for the treatment of Duchenne muscular dystrophy (DMD).

See also: Generic approvals, New drug approvals, Recent additions to Drugs.com, Alphabetical listing of all new drug applications, FDA approval process

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