Treatment Watch

2591 eventsUpdated today

FDA drug approvals, PDUFA decisions, orphan designations, and breakthrough therapy designations for rare diseases — sourced daily from FDA.gov.

FDA data is sourced from FDA.gov and openFDA.
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All eventsFDA approval (873)PDUFA date (0)Orphan designation (0)Breakthrough therapy (0)Complete response letter (0)Upcoming only·Year:All2015201420132012201120102009200820072006

91 events · 2014

Dec 29
2014
label expansion
KALYDECO (IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Dec 19
2014
label expansion
ZYPREXA RELPREVV (OLANZAPINE PAMOATE)FDA label ↗
· H2-Pharma LLC· NDA022173
Dec 19
2014
label expansion
ZYPREXA INTRAMUSCULAR (OLANZAPINE)FDA label ↗
· Eli Lilly and Company· NDA021253
Dec 19
2014
label expansion
ZYPREXA ZYDIS (OLANZAPINE)FDA label ↗
· Eli Lilly and Company· NDA021086
Dec 18
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020844
Dec 18
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020505
Dec 15
2014
label expansion
VALPROATE SODIUM (VALPROATE SODIUM)
· Hikma Pharmaceuticals USA Inc.· ANDA078523
Dec 13
2014
label expansion
ISOTRETINOIN (ISOTRETINOIN)
· Mayne Pharma· ANDA076485
Dec 13
2014
label expansion
LAMOTRIGINE (LAMOTRIGINE)
Lennox-Gastaut syndrome· Sun Pharmaceutical Industries, Inc.· ANDA078525
Dec 12
2014
label expansion
LAMOTRIGINE (LAMOTRIGINE)
Lennox-Gastaut syndrome· Alembic Pharmaceuticals Limited· ANDA090607
Dec 12
2014
FDA approvalOrphan drug
Abilify (aripiprazole)

Treatment of pediatric patients with Tourette's

Tourette syndrome· Otsuka Pharmaceutical Development
Dec 12
2014
label expansion
LAMOTRIGINE (LAMOTRIGINE)
Lennox-Gastaut syndrome· Aurobindo Pharma Limited· ANDA078956
Dec 11
2014
label expansion
KALYDECO (IVACAFTOR)
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Dec 10
2014
label expansion
LAMOTRIGINE KIT (LAMOTRIGINE)
Lennox-Gastaut syndrome· OWP Pharmaceuticals, Inc.· ANDA078947
Dec 10
2014
label expansion
VALPROIC ACID (VALPROIC ACID)
Juvenile absence epilepsy· Bionpharma Inc.· ANDA073484
Dec 5
2014
label expansion
ABILIFY MAINTENA (ARIPIPRAZOLE)FDA label ↗
Tourette syndrome· Otsuka America Pharmaceutical, Inc.· NDA202971
Dec 2
2014
label expansion
CARDENE IV (NICARDIPINE HYDROCHLORIDE)
· Baxter Healthcare Corporation· NDA019734
Nov 25
2014
label expansion
BOSULIF (BOSUTINIB)FDA label ↗
Chronic myeloid leukemia· Pfizer Laboratories Div Pfizer Inc· NDA203341
Nov 20
2014
label expansion
DEPAKOTE SPRINKLES (DIVALPROEX SODIUM)FDA label ↗
· AbbVie Inc.· NDA019680
Nov 10
2014
label expansion
KALYDECO (IVACAFTOR)
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Nov 4
2014
label expansion
LOSARTAN POTASSIUM AND HYDROCHLOROTHIAZIDE (LOSARTAN POTASSIUM AND HYDROCHLOROTHIAZIDE)
· Macleods Pharmaceuticals Limited· ANDA202289
Oct 30
2014
label expansion
ZYPREXA RELPREVV (OLANZAPINE PAMOATE)FDA label ↗
· H2-Pharma LLC· NDA022173
Oct 23
2014
FDA approvalOrphan drug
OBIZUR (antihemophilic factor (recombinant), porcine sequence)

Treatment of bleeding episodes in adults with acquired hemophilia A.

Rare thrombotic disorder due to a coagulation factors defect· Takeda Development Center Americas, Inc.
Oct 21
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020844
Oct 21
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020505
Oct 20
2014
label expansion
ABSORICA (ISOTRETINOIN)
· Sun Pharmaceutical Industries, Inc.· NDA021951
Oct 17
2014
label expansion
AUBAGIO (TERIFLUNOMIDE)FDA label ↗
· Genzyme Corporation· NDA202992
Oct 15
2014
FDA approvalOrphan drug
Ofev (NINTEDANIB)FDA label ↗

Treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis· Boehringer Ingelheim Pharmaceuticals, Inc.· NDA205832
Oct 15
2014
FDA approvalOrphan drug
Pirfenidone (PIRFENIDONE)

Treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis· AvKARE· ANDA212569
Sep 30
2014
label expansion
ZYPREXA INTRAMUSCULAR (OLANZAPINE)
· Eli Lilly and Company· NDA021253
Sep 29
2014
label expansion
ABILIFY MAINTENA (ARIPIPRAZOLE)FDA label ↗
Tourette syndrome· Otsuka America Pharmaceutical, Inc.· NDA202971
Sep 26
2014
label expansion
PHENTERMINE AND TOPIRAMATE (PHENTERMINE AND TOPIRAMATE)FDA label ↗
· Prasco Laboratories· NDA022580
Sep 26
2014
label expansion
OZURDEX (DEXAMETHASONE)FDA label ↗
Multiple myeloma· Allergan, Inc.· NDA022315
Sep 26
2014
label expansion
MEKINIST (TRAMETINIB)
Non-small cell lung cancer· Novartis Pharmaceuticals Corporation· NDA204114
Sep 23
2014
FDA approvalOrphan drug
HUMIRA (adalimumab)

Reducing signs and symptoms and inducing and maintaining clinical remission in patients 6 years of age and older with moderately to severely active Crohn's disease who have had an inadequate response to corticosteroids or immunomodulators such as azathioprine, 6-mercaptopurine, or methotrexate.

Pediatric-onset Graves disease· AbbVie, Inc.
Sep 11
2014
label expansion
TRAZODONE HYDROCHLORIDE (TRAZODONE HYDROCHLORIDE)
· Apotex Corp· ANDA071196
Aug 25
2014
label expansion
TAFINLAR (DABRAFENIB)FDA label ↗
Malignant lymphoma with peripheral neuropathy· Novartis Pharmaceuticals Corporation· NDA202806
Aug 20
2014
label expansion
DEPAKOTE SPRINKLES (DIVALPROEX SODIUM)FDA label ↗
· AbbVie Inc.· NDA019680
Aug 19
2014
FDA approvalOrphan drug
Cerdelga (ELIGLUSTAT)

Long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

Fetal Gaucher disease· Genzyme Corporation
Aug 15
2014
label expansion
ABSORICA (ISOTRETINOIN)FDA label ↗
· Sun Pharmaceutical Industries, Inc.· NDA021951
Aug 15
2014
label expansion
ABILIFY MAINTENA (ARIPIPRAZOLE)
Tourette syndrome· Otsuka America Pharmaceutical, Inc.· NDA202971
Aug 13
2014
label expansion
MEKINIST (TRAMETINIB)
Non-small cell lung cancer· Novartis Pharmaceuticals Corporation· NDA204114
Aug 12
2014
label expansion
ESTRING (ESTRADIOL)FDA label ↗
· Pfizer Laboratories Div Pfizer Inc· NDA020472
Aug 11
2014
label expansion
CONCERTA (METHYLPHENIDATE HYDROCHLORIDE)
Isolated hyperchlorhidrosis· Janssen Pharmaceuticals, Inc.· NDA021121
Aug 11
2014
label expansion
ZYPREXA INTRAMUSCULAR (OLANZAPINE)
· Eli Lilly and Company· NDA021253
Aug 1
2014
label expansion
TYVASO (TREPROSTINIL)
Pulmonary arterial hypertension· United Therapeutics Corporation· NDA022387
Jul 30
2014
label expansion
ABSORICA (ISOTRETINOIN)FDA label ↗
· Sun Pharmaceutical Industries, Inc.· NDA021951
Jul 29
2014
label expansion
KALYDECO (IVACAFTOR)
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Jul 23
2014
FDA approvalOrphan drug
Zydelig (idelalisib)

Zydelig is indicated for the treatment of patients with relapsed follicular B-cell non-Hodgkin lymphoma (FL) who have received at least two prior systemic therapies.

Follicular lymphoma· Gilead Sciences, Inc.
Jul 23
2014
label expansion
CARDENE IV (NICARDIPINE HYDROCHLORIDE)FDA label ↗
· Baxter Healthcare Corporation· NDA019734
Jul 22
2014
FDA approvalOrphan drug
RYANODEX (dantrolene sodium suspension for injection)

Treatment of malignant hyperthermia in conjunction with appropriate supportive measures and for the prevention of malignant hyperthermia in patients at high risk.

Multiple pterygium-malignant hyperthermia syndrome· Eagle Pharmaceuticals, Inc.
Jul 3
2014
FDA approvalOrphan drug
Beleodaq (Belinostat)

Treatment of patients with relapsed or refractory peripheral T-cell lymphoma

Primary cutaneous peripheral T-cell lymphoma not otherwise specified· Acrotech Biopharma LLC
Jun 28
2014
label expansion
OZURDEX (DEXAMETHASONE)FDA label ↗
Multiple myeloma· Allergan, Inc.· NDA022315
Jun 24
2014
label expansion
VFEND (VORICONAZOLE)
· Roerig· NDA021267
Jun 20
2014
label expansion
DEPAKOTE SPRINKLES (DIVALPROEX SODIUM)FDA label ↗
· AbbVie Inc.· NDA019680
Jun 19
2014
label expansion
DEPAKOTE SPRINKLES (DIVALPROEX SODIUM)FDA label ↗
· AbbVie Inc.· NDA019680
Jun 13
2014
label expansion
KALYDECO (IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Jun 13
2014
FDA approvalOrphan drug
LYMPHOSEEK (technetium Tc 99m tilmanocept)

Guiding sentinel lymph node biopsy, using a hand-held gamma counter in patients with clinically node negative squamous cell carcinoma of the oral cavity.

Immunodeficiency with isotype or light chain deficiencies with normal number of B-cells· Cardinal Health 414, LLC
Jun 9
2014
label expansion
DEPAKOTE SPRINKLES (DIVALPROEX SODIUM)FDA label ↗
· AbbVie Inc.· NDA019680
Jun 9
2014
FDA approval
DULOXETINE HYDROCHLORIDE (DULOXETINE)
· Alembic Pharmaceuticals Limited· ANDA202949
Jun 9
2014
label expansion
VFEND (VORICONAZOLE)
· Roerig· NDA021267
Jun 6
2014
FDA approvalOrphan drug
Eloctate (antihemophilic factor (recombinant), Fc fusion protein)

Indicated in adults and children with Hemophilia A (congenital Factor VIII deficiency) for on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce the frequency of bleeding episodes.

Moderate hemophilia A· Bioverativ Therapeutics, Inc.
May 30
2014
label expansion
ABILIFY MAINTENA (ARIPIPRAZOLE)FDA label ↗
Tourette syndrome· Otsuka America Pharmaceutical, Inc.· NDA202971
May 20
2014
label expansion
TYVASO (TREPROSTINIL)FDA label ↗
Pulmonary arterial hypertension· United Therapeutics Corporation· NDA022387
May 16
2014
label expansion
ESTRING (ESTRADIOL)
· Pfizer Laboratories Div Pfizer Inc· NDA020472
Apr 30
2014
label expansion
CARDENE IV (NICARDIPINE HYDROCHLORIDE)
· Baxter Healthcare Corporation· NDA019734
Apr 28
2014
FDA approvalOrphan drug
Purixan (mercaptopurine oral solution)

Treatment of patients with acute lymphoblastic leukemia as part of a combination regimen.

Acute myeloblastic leukemia with maturation· Nova Laboratories Limited
Apr 23
2014
FDA approvalOrphan drug
SYLVANT (siltuximab)

Treatment of patients with multicentric Castleman's disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative.

Unicentric Castleman disease· Recordati Rare Diseases Inc.
Apr 17
2014
label expansion
KESIMPTA (OFATUMUMAB)FDA label ↗
T-cell prolymphocytic leukemia· Novartis Pharmaceuticals Corporation· BLA125326
Apr 8
2014
label expansion
KALYDECO (IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Apr 7
2014
label expansion
VFEND (VORICONAZOLE)FDA label ↗
· Roerig· NDA021267
Apr 4
2014
FDA approvalOrphan drug
Lipiodol (ethiodized oil injection)

Selective hepatic intra-arterial use for imaging tumors in adults with known hepatocellular carcinoma (HCC)

Pediatric hepatocellular carcinoma· Guerbet LLC
Apr 3
2014
label expansion
SABRIL (VIGABATRIN)FDA label ↗
Neuroendocrine tumor of stomach· Lundbeck Pharmaceuticals LLC· NDA020427
Apr 3
2014
label expansion
SABRIL (VIGABATRIN)FDA label ↗
Neuroendocrine tumor of stomach· Lundbeck Pharmaceuticals LLC· NDA022006
Mar 28
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020844
Mar 28
2014
FDA approvalOrphan drug
ALPROLIX (Coagulation factor IX (recombinant), Fc fusion protein)

Adults and children with Hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

Multisystem inflammatory syndrome in children and adults· Bioverativ Therapeutics, Inc.
Mar 28
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020505
Mar 19
2014
FDA approvalOrphan drug
IMPAVIDO (miltefosine)

Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due to Leishmania braziliensis.

Leishmaniasis· Knight Therapeutics (USA)
Mar 18
2014
label expansion
GLYCOPYRROLATE (GLYCOPYRROLATE)
Neurometabolic disorder due to serine deficiency· Hikma Pharmaceuticals USA Inc.· NDA017558
Mar 11
2014
FDA approval
TOPIRAMATE (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Upsher-Smith Laboratories, LLC· NDA205122
Mar 4
2014
label expansion
USTEKINUMAB (USTEKINUMAB)FDA label ↗
· Janssen Biotech, Inc.· BLA125261
Feb 24
2014
FDA approvalOrphan drug
MYALEPT (metreleptin)

Adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

AKT2-related familial partial lipodystrophy· Chiesi Farmaceutici S.p.A.
Feb 21
2014
label expansion
KALYDECO (IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA203188
Feb 18
2014
label expansion
ZYPREXA RELPREVV (OLANZAPINE PAMOATE)
· H2-Pharma LLC· NDA022173
Feb 18
2014
FDA approvalOrphan drug
NORTHERA (droxidopa)

Treatment of orthostatic dizziness, lightheadedness, or the "feeling that you are about to black out" in adult patients with symptomatic neurogenic orthostatic hypotension caused by primary autonomic failure (Parkinson's disease, multiple system atrophy, and pure autonomic failure), dopamine beta-hydroxylase deficiency, and non-diabetic autonomic neuropathy.

PrP systemic amyloidosis· Lundbeck LLC
Feb 7
2014
label expansion
OZURDEX (DEXAMETHASONE)FDA label ↗
Multiple myeloma· Allergan, Inc.· NDA022315
Feb 7
2014
FDA approval
AMMONIA N 13 (NH3N13)
· Washington University School of Medicine· ANDA204506
Jan 29
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020844
Jan 29
2014
label expansion
TOPAMAX (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Janssen Pharmaceuticals, Inc.· NDA020505
Jan 9
2014
label expansion
TAFINLAR (DABRAFENIB)FDA label ↗
Malignant lymphoma with peripheral neuropathy· Novartis Pharmaceuticals Corporation· NDA202806
Jan 8
2014
label expansion
MEKINIST (TRAMETINIB)FDA label ↗
Non-small cell lung cancer· Novartis Pharmaceuticals Corporation· NDA204114

Not medical advice — always consult your healthcare provider before making treatment decisions.

Data sourced from FDA.gov and openFDA, updated daily. Dates are subject to change. Always verify with FDA.gov for official decisions.