Treatment Watch

2715 eventsUpdated today

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

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FDA data is sourced from FDA.gov and openFDA.
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All eventsFDA approval (620)PDUFA date (0)Orphan designation (0)Breakthrough therapy (0)Complete response letter (0)Upcoming only·Year:All2026202520242023202220212020201920182017

34 events · FDA approval · 2019

Dec 20
2019
FDA approvalOrphan drug
DORC ILM-Blue (Brilliant Blue G Ophthalmic Solution 0.025%)

TissueBlue (Brilliant Blue G Ophthalmic Solution) 0.025% is indicated to selectively stain the internal limiting membrane (ILM).

Rare genetic ophthalmic disorder with cortical involvement· Dutch Ophthalmic Research Center International BV
Dec 13
2019
FDA approvalOrphan drug
Tiglutik (riluzole oral suspension)

TIGLUTIK is indicated for the treatment of amyotrophic lateral sclerosis (ALS).

Amyotrophic Lateral Sclerosis (ALS)· Italfarmaco SpA
Nov 22
2019
FDA approvalOrphan drug
EXSERVAN (riluzole oral film)

EXSERVAN is indicated for the treatment of amyotrophic lateral sclerosis (ALS).

Amyotrophic Lateral Sclerosis (ALS)· Aquestive Therapeutics
Nov 20
2019
FDA approvalOrphan drug
GIVLAARI (givosiran)

GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP).

Acute hepatic porphyria· Alnylam Pharmaceuticals, Inc.
Oct 21
2019
FDA approvalOrphan drug
Trikafta (elexacaftor/tezacaftor/ivacaftor)

Indicated for CF in patients aged 2+ with at least one F508del mutation.

Cystic fibrosis· Vertex Pharmaceuticals
Oct 8
2019
FDA approvalOrphan drug
SCENESSE® (afamelanotide)

SCENESSE® is indicated to increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP)

Hepatoerythropoietic porphyria· Clinuvel Inc.
Oct 3
2019
FDA approvalOrphan drug
Dexamethasone (DEXAMETHASONE)

HEMADY is indicated in combination with other anti-myeloma products for the treatment of adults with multiple myeloma

Multiple myeloma· Advanced Rx Pharmacy of Tennessee, LLC· ANDA201270
Sep 6
2019
FDA approvalOrphan drug
Ofev (NINTEDANIB)FDA label ↗

OFEV is indicated to slow the rate of decline in pulmonary function in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD).

Exposure-related interstitial lung disease· Boehringer Ingelheim Pharmaceuticals, Inc.· NDA205832
Aug 28
2019
FDA approvalOrphan drug
Harvoni (ledipasvir/sofosbuvir)

HARVONI is indicated for the treatment of adults and pediatric patients 3 years of age and older with chronic hepatitis C virus (HCV) genotype 1, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis, genotype 1 infection with decompensated cirrhosis, for use in combination with ribavirin, and genotype 1 or 4 infection who are liver transplant recipients without cirrhosis or with compensated cirrhosis, for use in combination with ribavirin

Adenovirus infection in immunocompromised patients· Gilead Sciences, Inc.
Aug 15
2019
FDA approvalOrphan drug
ROZLYTREK (entrectinib)

ROZLYTREK is indicated for the treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have either progressed following treatment or have no satisfactory alternative therapy

RELA fusion-positive ependymoma· Genentech, Inc.
Aug 14
2019
FDA approval
WAKIX (PITOLISANT HYDROCHLORIDE)FDA label ↗
· Harmony Biosciences, LLC· NDA211150
Aug 14
2019
FDA approvalOrphan drug
Pretomanid (Pretomanid)

Pretomanid tablet is indicated, as part of a combination regimen with bedaquiline and linezolid for the treatment of adults with pulmonary extensively drug resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB).

Primary genito-urinary tuberculosis· Global Alliance for TB Drug Development
Aug 9
2019
FDA approval
TRAMADOL HYDROCHLORIDE (TRAMADOL HYDROCHLORIDE)FDA label ↗
· Unichem Pharmaceuticals (USA), Inc.· ANDA211825
Aug 2
2019
FDA approvalOrphan drug
TURALIO (pexidartinib)

TURALIO is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery.

Tenosynovial giant cell tumor· Daiichi Sankyo, Inc.
Jul 19
2019
FDA approvalOrphan drug
OTEZLA® (apremilast)

OTEZLA is indicated for the treatment of adult patients with oral ulcers associated with Beh®et®s Disease.

Thomsen and Becker disease· Amgen Inc.
Jul 19
2019
FDA approval
PREGABALIN (PREGABALIN)
· Ascend Laboratories, LLC· ANDA207623
Jun 28
2019
FDA approvalOrphan drug
Thiola (Tiopronin)

THIOLA EC is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 20 kg and greater with severe homozygous cystinuria, who are not responsive to these measures alone.

Homocystinuria due to cystathionine beta-synthase deficiency· Mission Pharmacal Company
Jun 17
2019
FDA approvalOrphan drug
Sunosi (Solriamfetol)

SUNOSI is indicated to improve wakefulness in adult patients with excessive daytime sleepiness associated with narcolepsy

Narcolepsy· Axsome Malta, Ltd.
Jun 6
2019
FDA approvalOrphan drug
NUCALA (mepolizumab)

NUCALA prefilled autoinjector and single syringe device is indicated for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA).

Eosinophilic granulomatosis with polyangiitis· GlaxoSmithKline LLC
May 28
2019
FDA approvalOrphan drug
Lenalidomide (LENALIDOMIDE)

REVLIMID in combination with a rituximab product, is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL).

MALT lymphoma· Teva Pharmaceuticals, Inc.· ANDA201452
May 17
2019
FDA approvalOrphan drug
NAYZILAM (midazolam)

NAYZILAM is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient®s usual seizure pattern in patients with epilepsy 12 years of age and older.

Cytomegalovirus disease in patients with impaired cell mediated immunity deemed at risk· UCB, Inc.
May 15
2019
FDA approvalOrphan drug
VENCLEXTA (venetoclax)

VENCLEXTA is indicated for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

T-cell prolymphocytic leukemia· AbbVie, Inc
Apr 30
2019
FDA approvalOrphan drug
MAVYRET (glecaprevir and pibrentasvir)

MAVYRET is indicated for the treatment of adult and pediatric patients 12 years and older or weighing at least 45 kg with chronic hepatitis C virus (HCV) genotype 1, 2, 3, 4, 5 or 6 infection without cirrhosis or with compensated cirrhosis (Child-Pugh A). MAVYRET is also indicated for the treatment of adult and pediatric patients 12 years and older or weighing at least 45 kg with HCV genotype 1 infection, who previously have been treated with a regimen containing an HCV NS5A inhibitor or an NS3/4A protease inhibitor (PI), but not both.

Congenital varicella syndrome· AbbVie, Inc.
Apr 26
2019
FDA approval
BOSENTAN (BOSENTAN)FDA label ↗
Pulmonary arterial hypertension· Actavis Pharma, Inc.· ANDA207110
Apr 26
2019
FDA approval
BOSENTAN (BOSENTAN)FDA label ↗
Pulmonary arterial hypertension· Zydus Pharmaceuticals USA Inc.· ANDA207760
Apr 26
2019
FDA approval
BOSENTAN (BOSENTAN)FDA label ↗
Pulmonary arterial hypertension· Sun Pharmaceutical Industries, Inc.· ANDA209324
Apr 22
2019
FDA approvalOrphan drug
CORLANOR (ivabradine)

Corlanor is indicated for the treatment of stable symptomatic heart failure due to dilated cardiomyopathy (DCM) in pediatric patients aged 6 months and older, who are in sinus rhythm with an elevated heart rate.

Sensorineural deafness with dilated cardiomyopathy· Amgen, Inc.,
Apr 10
2019
FDA approval
AMBRISENTAN (AMBRISENTAN)FDA label ↗
Pulmonary arterial hypertension· Sigmapharm Laboratories, LLC· ANDA208354
Mar 29
2019
FDA approvalOrphan drug
Avaclyr (acyclovir)

AVACLYR is a sterile topical antiviral indicated for the treatment of acute herpetic keratitis (dendritic ulcers) in patients with herpes simplex (HSV-1 and HSV-2) virus.

Infective dermatitis associated with HTLV-1· Fera Pharmaceuticals
Mar 18
2019
FDA approvalOrphan drug
TECENTRIQ (atezolizumab)

TECENTRIQ®, in combination with carboplatin and etoposide, is indicated for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC)

Small cell lung cancer· Genentech, Inc.
Feb 22
2019
FDA approvalOrphan drug
LONSURF (trifluridine/tipiracil)

LONSURF is indicated for the treatment of adult patients with metastatic gastric or gastroesophageal junction adenocarcinoma previously treated with at least two prior lines of chemotherapy that included a fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy.

Adenocarcinoma of the oesophagus and oesophagogastric junction· Taiho Oncology, Inc.
Feb 13
2019
FDA approvalOrphan drug
Egaten (triclabendazole)

EGATEN® is indicated for the treatment of fascioliasis in patients 6 years of age and older.

Fasciolopsiasis· Novartis Pharmaceuticals Corporation
Feb 6
2019
FDA approvalOrphan drug
Cablivi (CAPLACIZUMAB)FDA label ↗

CABLIVI is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

Thrombotic thrombocytopenic purpura· Genzyme Corporation· BLA761112
Jan 14
2019
FDA approval
VIGABATRIN (VIGABATRIN)FDA label ↗
Neuroendocrine tumor of stomach· Actavis Pharma, Inc.· ANDA209822

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.