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.

aducanumab

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).

Biogen Completes Submission of Biologics License Application to FDA for Aducanumab as a Treatment for Alzheimer’s Disease - July 8, 2020

pralsetinib

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.

Blueprint Medicines Announces Submission of New Drug Application to FDA for Pralsetinib for the Treatment of Advanced RET Mutant and RET Fusion-Positive Thyroid Cancers - July 1, 2020

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.

scPharmaceuticals Announces Furoscix NDA Resubmission - July 1, 2020

udenafil

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.

Mezzion Announces Submission of New Drug Application for its Orphan Drug Udenafil to Treat Patients who have undergone the Fontan Operation for Single Ventricle Heart Disease - June 30, 2020

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.

HTX-011 (bupivacaine and meloxicam)

Company: Heron Therapeutics, Inc.
Treatment for: Postoperative Pain

HTX-011 (bupivacaine and meloxicam) is an investigational fixed-dose combination of the local anesthetic bupivacaine and the nonsteroidal anti-inflammatory drug (NSAID) meloxicam in development for the management of postoperative pain.

casimersen

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.

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 - June 26, 2020

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.

Mayne Pharma Announces FDA Filing Acceptance of New Drug Application for E4/DRSP in the US - June 24, 2020

Contepo (fosfomycin)

Company: Nabriva Therapeutics plc
Treatment for: Urinary Tract Infection

Contepo (fosfomycin for injection) is an investigational epoxide antibiotic in development as a first-line treatment for complicated urinary tract infections (cUTIs), including acute pyelonephritis.

arimoclomol

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).

Orphazyme Initiates Rolling Submission of New Drug Application for Arimoclomol with US FDA in Niemann-Pick Disease Type C - May 29, 2020

Eysuvis (loteprednol etabonate)

Company: Kala Pharmaceuticals, Inc.
Treatment for: Dry Eye Disease

Eysuvis (loteprednol etabonate) is an ophthalmic corticosteroid formulation in development for the temporary relief of signs and symptoms of dry eye disease.

dasiglucagon

Company: Zealand Pharma A/S
Treatment for: Hypoglycemia

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

Zealand Pharma Announces FDA acceptance of New Drug Application for the Dasiglucagon HypoPal® Rescue Pen for Treatment of Severe Hypoglycemia - May 22, 2020

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.

Adamis Pharmaceuticals Resubmits Zimhi New Drug Application to FDA - May 20, 2020

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.

sutimlimab

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).

FDA Grants Priority Review of Sutimlimab, Potential First Approved Treatment of Hemolysis in Adult Patients with Cold Agglutinin Disease - May 14, 2020

setmelanotide

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.

Rhythm Pharmaceuticals Announces FDA Acceptance of New Drug Application for Setmelanotide for the Treatment of POMC and LEPR Deficiency Obesities - May 13, 2020

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.

U.S. Food and Drug Administration (FDA) Accepts for Priority Review Bristol Myers Squibb’s Application for CC-486 for Maintenance Treatment of Adult Patients in Remission with Acute Myeloid Leukemia - May 1, 2020

brincidofovir

Company: Chimerix, Inc.
Treatment for: Smallpox

Brincidofovir is a nucleotide analog broad-spectrum antiviral in development as a medical countermeasure for smallpox.

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