Treatment Watch

2715 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 (621)PDUFA date (0)Orphan designation (0)Breakthrough therapy (0)Complete response letter (0)Upcoming only·Year:All2026202520242023202220212020201920182017

53 events · FDA approval · 2018

Dec 21
2018
FDA approvalOrphan drug
ELZONRIS (Tagraxofusp-erzs)

ELZONRIS is a CD123-directed cytotoxin for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older.

Blastic plasmacytoid dendritic cell neoplasm· Stemline Therapeutics, Inc.
Dec 19
2018
FDA approvalOrphan drug
Envarsus XR (tacrolimus)

ENVARSUS XR is indicated for the prophylaxis of organ rejection in kidney transplant patients in combination with other immunosuppressants.

Rare disorder potentially indicated for kidney transplant· Veloxis Pharmaceuticals, Inc.
Nov 29
2018
FDA approvalOrphan drug
Astagraf XL (tacrolimus extended-release capsules)

prophylaxis of organ rejection in kidney transplant patients in combination with other immunosuppressants in adult and pediatric patients.

Recurrent hepatitis C virus induced liver disease in liver transplant recipients· Astellas Pharma Global Development, Inc.
Nov 28
2018
FDA approvalOrphan drug
XOSPATA (gilteritinib)

XOSPATA is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved test.

Acute megakaryoblastic leukemia· Astellas Pharma US, Inc.
Nov 28
2018
FDA approvalOrphan drug
Firdapse (AMIFAMPRIDINE PHOSPHATE)FDA label ↗

Treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults

Lambert-Eaton myasthenic syndrome· Catalyst Pharmaceuticals, Inc.· NDA208078
Nov 26
2018
FDA approvalOrphan drug
VITRAKVI (larotrectinib)

VITRAKVI® is indicated for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine 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 no satisfactory alternative treatments or that have progressed following treatment.

Embryonal tumor with multilayered rosettes· Bayer HealthCare Pharmaceuticals Inc.
Nov 21
2018
FDA approval
METHYLPHENIDATE HYDROCHLORIDE (METHYLPHENIDATE HYDROCHLORIDE)
Isolated hyperchlorhidrosis· Granules Pharmaceuticals Inc.· ANDA210992
Nov 21
2018
FDA approvalOrphan drug
DAURISMO (Glasdegib)

DAURISMO is indicated, in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.

Acute megakaryoblastic leukemia· Pfizer, Inc.
Nov 20
2018
FDA approval
VIGABATRIN (VIGABATRIN)FDA label ↗
Neuroendocrine tumor of stomach· Dr. Reddys Laboratories Inc.,· ANDA211481
Nov 16
2018
FDA approvalOrphan drug
Eltrombopag (ELTROMBOPAG OLAMINE)

PROMACTA is indicated in combination with standard immunosuppressive therapy for the first-line treatment of adult and pediatric patients 2 years and older with severe aplastic anemia.

Rare aplastic anemia· Novadoz Pharmaceuticals LLC· ANDA220250
Nov 9
2018
FDA approval
ANASTROZOLE (ANASTROZOLE TABLETS)
· Avet Pharmaceuticals Inc· ANDA206037
Nov 6
2018
FDA approvalOrphan drug
Empliciti (elotuzumab)

EMPLICITI is indicated in combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor.

Multiple myeloma· Bristol-Myers Squibb Co.
Nov 2
2018
FDA approvalOrphan drug
LORBRENA (Lorlatinib)

LORBRENA® is indicated for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease; or alectinib as the first ALK inhibitor therapy for metastatic disease; or ceritinib as the first ALK inhibitor therapy for metastatic disease.

Non-small cell lung cancer· Pfizer, Inc.
Oct 28
2018
FDA approval
BIJUVA (ESTRADIOL AND PROGESTERONE)FDA label ↗
· Mayne Pharma· NDA210132
Oct 16
2018
FDA approval
BUPROPION HYDROCHLORIDE SR (BUPROPION HYDROCHLORIDE)
· Slate Run Pharmaceuticals, LLC· ANDA211347
Oct 5
2018
FDA approvalOrphan drug
Tegsedi (inotersen)

Indicated for polyneuropathy of hATTR amyloidosis in adults

Wild type ATTR amyloidosis· Ionis/AstraZeneca
Oct 5
2018
FDA approvalOrphan drug
Revcovi (elapegademase-lvlr)

Treatment of Adenosine Deaminase-Severe Combined Immunodeficiency (ADA-SCID).

Short-limb skeletal dysplasia with severe combined immunodeficiency· Chiesi USA Inc.
Sep 28
2018
FDA approvalOrphan drug
Epidiolex (CANNABIDIOL)FDA label ↗

EPIDIOLEX is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients 2 years of age and older.

Lennox-Gastaut syndrome· Jazz Pharmaceuticals, Inc.· NDA210365
Sep 28
2018
FDA approvalOrphan drug
Arikayce (liposomal amikacin)

Treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy

Mendelian susceptibility to mycobacterial diseases· Insmed Incorporated
Sep 27
2018
FDA approvalOrphan drug
VIZIMPRO (dacomitinib)

For the first-line treatment of patients with metastatic non-small-cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R

Non-small cell lung cancer· Pfizer, Inc.
Sep 24
2018
FDA approvalOrphan drug
COPIKTRA (duvelisib)

Treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies

Precursor T-cell acute lymphoblastic leukemia· Secura Bio, Inc.
Sep 21
2018
FDA approvalOrphan drug
COAGADEX (Coagulation factor X (human))

COAGADEX is indicated in adults and children with hereditary Factor X deficiency for routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment and control of bleeding episodes; and perioperative management of bleeding in patients with mild and moderate hereditary Factor X deficiency.

Congenital factor XI deficiency· Bio Products Laboratory Limited
Sep 13
2018
FDA approvalOrphan drug
LUMOXITI (moxetumomab pasudotox-tdfk)

LUMOXITI is indicated for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who received at least two prior systemic therapies, including treatment with a purine nucleoside analog (PNA).

Classic hairy cell leukemia· AstraZeneca Pharmaceuticals LP
Aug 22
2018
FDA approvalOrphan drug
Oxervate (cenegermin-bkbj)

Treatment of neurotrophic keratitis

Neurotrophic keratopathy· Dompe S.p.A.
Aug 15
2018
FDA approvalOrphan drug
LENVIMA (lenvatinib)

LENVIMA is indicated for the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)

Adult hepatocellular carcinoma· Eisai Inc.
Aug 13
2018
FDA approval
FEXOFENADINE HYDROCHLORIDE (FEXOFENADINE HYDROCHLORIDE)
· Pioneer Life Sciences, LLC· ANDA210137
Aug 10
2018
FDA approvalOrphan drug
Onpattro (PATISIRAN)

ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Hereditary transthyretin-mediated amyloidosis· Alnylam Pharmaceuticals, Inc.
Aug 8
2018
FDA approvalOrphan drug
POTELIGEO (mogamulizumab)

POTELIGEO is indicated for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or S®zary syndrome (SS) after at least one prior systemic therapy.

Indolent primary cutaneous T-cell lymphoma· Kyowa Kirin Pharmaceutical Development Inc.
Aug 8
2018
FDA approval
POTELIGEO (MOGAMULIZUMAB-KPKC)FDA label ↗
· Kyowa Kirin, Inc.· BLA761051
Aug 7
2018
FDA approval
ORKAMBI (LUMACAFTOR AND IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA211358
Jul 30
2018
FDA approvalOrphan drug
Azedra Ultratrace (Iobenguane I 131)

AZEDRA is indicated for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.

Neuroendocrine tumor of the rectum· Progenics Pharmaceuticals, Inc.
Jul 27
2018
FDA approvalOrphan drug
Omegaven (fish oil triglycerides)

Omegaven is indicated as a source of calories and fatty acids in pediatric patients with parenteral nutrition-associated cholestasis (PNAC)

Parenteral nutrition-associated cholestasis· Fresenius Kabi USA, LLC
Jul 20
2018
FDA approval
GEMCITABINE (GEMCITABINE)
· Sagent Pharmaceuticals· ANDA209077
Jul 20
2018
FDA approvalOrphan drug
Krintafel (tafenoquine)

KRINTAFEL is indicated for the radical cure (prevention of relapse) of Plasmodium vivax malaria in patients aged 16 years and older who are receiving appropriate antimalarial therapy for acute P. vivax infection.

Spondyloepiphyseal dysplasia-brachydactyly-speech disorder syndrome· GlaxoSmithKline Intellectual Property Development Ltd. England
Jul 11
2018
FDA approval
DALFAMPRIDINE (DALFAMPRIDINE)
Multiple sclerosis· Accord Healthcare Inc.· ANDA206863
Jun 29
2018
FDA approvalOrphan drug
Signifor (pasireotide)

SIGNIFOR® LAR is indicated for the treatment of patients with Cushing®s disease for whom pituitary surgery is not an option or has not been curative.

Cushing disease· Novartis Pharmaceuticals Corporation
Jun 27
2018
FDA approval
BRAFTOVI (ENCORAFENIB)FDA label ↗
Non-small cell lung cancer· Array BioPharma Inc.· NDA210496
Jun 21
2018
FDA approvalOrphan drug
Ablysinol (dehydrated alcohol)

ABLYSINOL® is indicated to induce controlled cardiac septal infarction to improve exercise capacity in adults with symptomatic hypertrophic obstructive cardiomyopathy who are not candidates for surgical myectomy.

Familial isolated hypertrophic cardiomyopathy· Belcher Pharmaceuticals, LLC
Jun 14
2018
FDA approval
BUPROPION HYDROCHLORIDE XL (BUPROPION HYDROCHLORIDE)
· Slate Run Pharmaceuticals, LLC· ANDA210015
May 24
2018
FDA approval
PALYNZIQ (PEGVALIASE-PQPZ)FDA label ↗
Phenylketonuria· BioMarin Pharmaceutical Inc.· BLA761079
May 14
2018
FDA approval
METHYLPREDNISOLONE (METHYLPREDNISOLONE)
· Epic Pharma, LLC· ANDA204072
May 4
2018
FDA approvalOrphan drug
Tafinlar(r) Capsules a nd Mekinist(r) Tablets (dabrafenib and trametinib)

MEKINIST is indicated, in combination with dabrafenib, for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options

Cortical dysgenesis with pontocerebellar hypoplasia due to TUBB3 mutation· Novartis Pharmaceuticals Corporation
May 3
2018
FDA approvalOrphan drug
ANDEXXA (Coagulation factor Xa (recombinant), inactivated-zhzo)

Coagulation factor Xa (recombinant), inactivated-zhzo is indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding

Congenital vitamin K-dependent coagulation factors deficiency· Alexion Pharmaceuticals, Inc.
Apr 30
2018
FDA approvalOrphan drug
MEKINIST and TAFINLAR (trametinib and dabrafenib)

TAFINLAR is indicated, in combination with trametinib, for the adjuvant treatment of patients with melanoma with BRAF V600E or V600K mutations, as detected by an FDA-approved test, and involvement of lymph node(s), following complete resection

Melanoma· Novartis Pharmaceuticals Corporation
Apr 23
2018
FDA approvalOrphan drug
Jynarque (tolvaptan)

JYNARQUE is indicated to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD)

Autosomal dominant polycystic kidney disease· Otsuka Pharmaceutical Company, Ltd.
Apr 6
2018
FDA approvalOrphan drug
Rubraca (rucaparib)

For the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete or partial response to platinum-based chemotherapy.

Rare ovarian cancer· pharma&
Mar 15
2018
FDA approvalOrphan drug
Hizentra (Immune Globulin Subcutaneous (Human), 20% Liquid)

Indicated for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment.

Chronic inflammatory demyelinating polyneuropathy· CSL Behring
Mar 13
2018
FDA approval
VIGABATRIN (VIGABATRIN)FDA label ↗
Neuroendocrine tumor of stomach· Amneal Pharmaceuticals NY LLC· ANDA210155
Mar 6
2018
FDA approvalOrphan drug
Trogarzo (ibalizumab)

TROGARZO, in combination with other antiretroviral(s), is indicated for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen.

Acute disseminated encephalomyelitis without anti-MOG antibodies· Theratechnologies Inc.
Feb 14
2018
FDA approvalOrphan drug
Makena (hydroxyprogesterone caproate)

Makena auto-injector for subcutaneous injection is indicated to reduce the risk of preterm birth in women with singleton pregnancy who have a history of singleton spontaneous preterm birth.

Pancreatic arteriovenous malformation· AMAG Pharma USA, Inc.
Feb 12
2018
FDA approval
SYMDEKO (TEZACAFTOR AND IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA210491
Feb 12
2018
FDA approval
GLATOPA (GLATIRAMER ACETATE)FDA label ↗
Pediatric multiple sclerosis· Sandoz Inc· ANDA206921
Jan 12
2018
FDA approvalOrphan drug
Gilotrif (afatinib)

GILOTRIF is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have non-resistant epidermal growth factor receptor (EGFR) mutations as detected by an FDA-approved test.

Non-small cell lung cancer· Boehringer Ingelheim Pharmaceuticals, Inc.

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.