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: Novartis Pharmaceuticals Corporation
Treatment for: Acute Lymphoblastic Leukemia
CTL019 (tisagenlecleucel-T) is an investigational chimeric antigen receptor T cell (CAR-T) therapy in development for use in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).
Company: Puma Biotechnology, Inc.
Treatment for: Breast Cancer -- Adjuvant
Neratinib is a potent irreversible tyrosine kinase inhibitor in development for the extended adjuvant treatment of HER2-positive early stage breast cancer.
- Puma Biotechnology Announces FDA Advisory Committee to Review Neratinib for the Extended Adjuvant Treatment of HER2-Positive Early Stage Breast Cancer - April 17, 2017
- Puma Biotechnology Announces FDA Acceptance of NDA for PB272 (Neratinib) for Extended Adjuvant Treatment of HER2-Positive Early Stage Breast Cancer - September 20, 2016
- Puma Biotechnology Submits New Drug Application for PB272 (Neratinib) to FDA for Extended Adjuvant Treatment of HER2-Positive Early Stage Breast Cancer - July 21, 2016
Company: Rigel Pharmaceuticals, Inc.
Treatment for: Thrombocytopenia Autoimmune
Fostamatinib is an investigational oral spleen tyrosine kinase (SYK) inhibitor in development for the treatment of patients with chronic and persistent immune thrombocytopenia (ITP).
Company: Eli Lilly and Company and Incyte Corporation
Treatment for: Rheumatoid Arthritis
Baricitinib is an oral selective JAK1 and JAK2 inhibitor in development for the treatment of rheumatoid arthritis.
- FDA Issues Complete Response Letter for Baricitinib - April 14, 2017
- U.S. FDA Extends Review Period For Baricitinib, An Investigational Rheumatoid Arthritis Treatment - January 13, 2017
- Lilly and Incyte Announce Submission of NDA to FDA for Oral Once-Daily Baricitinib for Treatment of Moderate-to-Severe Rheumatoid Arthritis - January 19, 2016
Company: Icon Bioscience Inc.
Treatment for: Postoperative Ocular Inflammation
Dexycu (dexamethasone) is an investigational, long-acting, injectable corticosteroid formulation delivering dexamethasone into the anterior chamber of the eye for the treatment of inflammation associated with cataract surgery.
Zerviate (cetirizine) Ophthalmic Solution - formerly AC-170
Company: Nicox S.A.
Treatment for: Allergic Conjunctivitis
Zerviate (cetirizine) is a second generation antihistamine topical ophthalmic formulation in development for the treatment of ocular itching associated with allergic conjunctivitis.
- Nicox Announces PDUFA Date for Zerviate NDA - April 11, 2017
- Nicox Resubmits AC-170 (Zerviate) NDA to the U.S. FDA - March 9, 2017
- Nicox Receives Complete Response Letter from the FDA Related to GMP on NDA for AC-170 - October 10, 2016
- U.S. FDA grants Priority Review for Nicox’s AC-170 New Drug Application - June 21, 2016
- Nicox submits New Drug Application for AC-170 to U.S. FDA - April 19, 2016
Company: Novo Nordisk
Treatment for: Hemophilia B
Nonacog beta pegol is an investigational glycoPEGylated extended-half-life recombinant factor IX product in development for the treatment of hemophilia B.
Vyxeos (cytarabine and daunorubicin) Injection
Company: Jazz Pharmaceuticals plc
Treatment for: Acute Myeloid Leukemia
Vyxeos (cytarabine and daunorubicin liposome injection) is an investigational nano-scale liposome co-formulation of cytarabine and daunorubicin at a synergistic 5:1 molar ratio in development for the treatment of acute myeloid leukemia.
- Jazz Pharmaceuticals Completes Rolling Submission of New Drug Application for Vyxeos (CPX-351), an Investigational Treatment for Acute Myeloid Leukemia - April 3, 2017
- Jazz Pharmaceuticals Initiates Rolling NDA Submission for Vyxeos (CPX-351) Expects to Complete NDA Submission by Early 2017 - October 3, 2016
KTE-C19 (axicabtagene ciloleucel)
Company: Kite Pharma, Inc.
Treatment for: non-Hodgkin's Lymphoma
KTE-C19 (axicabtagene ciloleucel) is an investigational anti-CD19 CAR-T therapy in development for the treatment of patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant.
KTE-C19 therapy involves the engineering of a patient's T cells to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias. The T cells are then redirected to kill the cancer cells.
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).
- Aeterna Zentaris Intends to File NDA with Respect to Macrilen in Third Quarter of 2017 - March 30, 2017
- Aeterna Zentaris Announces Plans to Pursue FDA Registration of Macrilen - February 13, 2017
- Aeterna Zentaris Completes Patient Recruitment for Confirmatory Phase 3 Trial of Macrilen - October 26, 2016
- Aeterna Zentaris Announces Plans to Initiate Phase 3 Study of Macrilen in AGHD - April 13, 2015
- FDA Issues Complete Response Letter for Macrilen (macimorelin) NDA in Adult Growth Hormone Deficiency - November 6, 2014
- Aeterna Zentaris NDA for Macimorelin Acetate in AGHD Accepted for Filing by the FDA - January 6, 2014
- Aeterna Zentaris Submits New Drug Application to FDA for Macimorelin Acetate (AEZS-130) for Evaluation of AGHD - November 5, 2013
Solosec (secnidazole) Oral Granules
Company: Symbiomix Therapeutics
Treatment for: Bacterial Vaginosis
Solosec (secnidazole) is a potent, next-generation, investigational 5-nitroimidazole antibiotic with enhanced pharmacokinetic properties anticipated to be the first and only single-dose oral therapy approved for the treatment of bacterial vaginosis (BV).
Vesneo (latanoprostene bunod) Ophthalmic Solution
Company: Valeant Pharmaceuticals International, Inc.
Treatment for: Glaucoma (Open Angle), Intraocular Hypertension
Vesneo (latanoprostene bunod ophthalmic solution) is a nitric oxide donating prostaglandin receptor agonist in development for the treatment of patients with open angle glaucoma or ocular hypertension.
- Bausch + Lomb And Nicox Announce PDUFA Date For Novel Glaucoma Candidate Latanoprostene Bunod - March 20, 2017
- Bausch + Lomb And Nicox Resubmit US New Drug Application For Novel Glaucoma Candidate Latanoprostene Bunod - February 27, 2017
- Valeant Pharmaceuticals Receives FDA Complete Response Letter for Latanoprostene Bunod - July 22, 2016
- Bausch+Lomb and Nicox Announce FDA Acceptance of NDA for Novel Glaucoma Candidate Vesneo (latanoprostene bunod) - September 22, 2015
Company: Array BioPharma
Treatment for: NRAS-Mutant Melanoma
Binimetinib is a late-stage small molecule MEK inhibitor in development for the treatment of advanced NRAS-mutant melanoma.
- Array BioPharma Announces Withdrawal of NDA for Binimetinib Monotherapy for the Treatment of NRAS-Mutant Melanoma - March 19, 2017
- Array BioPharma Announces FDA Acceptance of Binimetinib NDA for Patients with Advanced NRAS-Mutant Melanoma - September 1, 2016
- Array BioPharma Submits Binimetinib New Drug Application to U.S. FDA - June 30, 2016
ZS-9 (sodium zirconium cyclosilicate)
Treatment for: Hyperkalemia
ZS-9 (sodium zirconium cyclosilicate) is a crystalline lattice potassium ion-trap in development for the treatment of hyperkalemia.
- AstraZeneca Receives Complete Response Letter from US FDA for ZS-9 (sodium zirconium cyclosilicate) for Hyperkalemia - March 17, 2017
- FDA Accepts for Review New Drug Application for Sodium Zirconium Cyclosilicate (ZS-9) for the Treatment of Hyperkalemia - October 18, 2016
- AstraZeneca Receives Complete Response Letter from FDA for Sodium Zirconium Cyclosilicate (ZS-9) for Hyperkalemia - May 27, 2016
- ZS Pharma Announces FDA Acceptance of ZS-9 New Drug Application - July 29, 2015
- ZS Pharma Submits NDA for ZS-9 for the Treatment of Hyperkalemia - May 26, 2015
Company: Radius Health, Inc.
Treatment for: Osteoporosis
Abaloparatide is a synthetic peptide analog of hPTHrP (human parathyroid hormone-related protein) in development for the treatment of postmenopausal women with osteoporosis.
- Radius Health Receives Notification of PDUFA Extension for Abaloparatide-SC - March 10, 2017
- Radius Announces FDA Acceptance for Filing of NDA for Abaloparatide-SC for the Treatment of Postmenopausal Women with Osteoporosis - May 31, 2016
- Radius Health Submits NDA to the U.S. FDA for Abaloparatide-SC for the Treatment of Postmenopausal Women with Osteoporosis - March 30, 2016
Company: Celgene Corporation
Treatment for: Acute Myeloid Leukemia
Enasidenib is a first-in-class, oral, targeted inhibitor of mutant isocitrate dehydrogenase 2 (IDH2) in development for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation.
Company: PTC Therapeutics, Inc.
Treatment for: Duchenne Muscular Dystrophy
Translarna (ataluren) is a protein restoration therapy in development for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
- PTC Therapeutics Announces FDA Acknowledgment of New Drug Application Filing for Translarna for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy - March 6, 2017
- PTC Therapeutics Provides Regulatory Update on Translarna (ataluren) for Nonsense Mutation Duchenne Muscular Dystrophy - October 17, 2016
- PTC Receives Refuse to File Letter from FDA for Translarna (ataluren) - February 23, 2016
- PTC Therapeutics Begins Rolling NDA Submission to the FDA for Translarna to Treat Duchenne Muscular Dystrophy - December 23, 2014
Treatment for: Diabetes Type 2
Ertugliflozin is a sodium-glucose cotransporter-2 (SGLT2) inhibitor in development to help improve glycemic control in adults with type 2 diabetes.