Treatment Watch
2715 eventsUpdated todayFDA drug approvals, PDUFA decisions, orphan designations, and breakthrough therapy designations for rare diseases — sourced daily from FDA.gov.
All eventsFDA approval (620)PDUFA date (0)Orphan designation (0)Breakthrough therapy (0)Complete response letter (0)Upcoming only·Year:All2026202520242023202220212020201920182017
103 events · 2018
Dec 27
2018
label expansion
Dec 27
2018
label expansion
Dec 21
2018
FDA approvalOrphan drug
Blastic plasmacytoid dendritic cell neoplasm· Stemline Therapeutics, Inc.
Dec 19
2018
FDA approvalOrphan drug
Rare disorder potentially indicated for kidney transplant· Veloxis Pharmaceuticals, Inc.
Nov 29
2018
FDA approvalOrphan drug
Recurrent hepatitis C virus induced liver disease in liver transplant recipients· Astellas Pharma Global Development, Inc.
Nov 28
2018
FDA approvalOrphan drug
Nov 26
2018
FDA approvalOrphan drug
Embryonal tumor with multilayered rosettes· Bayer HealthCare Pharmaceuticals Inc.
Nov 21
2018
FDA approval
Nov 20
2018
FDA approval
Nov 16
2018
FDA approvalOrphan drug
Oct 25
2018
label expansion
Oct 19
2018
label expansion
Oct 18
2018
label expansion
Oct 16
2018
FDA approval
· Slate Run Pharmaceuticals, LLC· ANDA211347
Oct 5
2018
FDA approvalOrphan drug
Short-limb skeletal dysplasia with severe combined immunodeficiency· Chiesi USA Inc.
Sep 28
2018
FDA approvalOrphan drug
Mendelian susceptibility to mycobacterial diseases· Insmed Incorporated
Sep 28
2018
FDA approvalOrphan drug
Aug 31
2018
Aug 21
2018
label expansion
Aug 21
2018
label expansion
Aug 15
2018
label expansion
Aug 15
2018
label expansion
Aug 13
2018
FDA approval
· Pioneer Life Sciences, LLC· ANDA210137
Aug 10
2018
FDA approvalOrphan drug
Hereditary transthyretin-mediated amyloidosis· Alnylam Pharmaceuticals, Inc.
Aug 8
2018
FDA approvalOrphan drug
Indolent primary cutaneous T-cell lymphoma· Kyowa Kirin Pharmaceutical Development Inc.
Aug 7
2018
FDA approval
Aug 7
2018
label expansion
Jul 30
2018
FDA approvalOrphan drug
Neuroendocrine tumor of the rectum· Progenics Pharmaceuticals, Inc.
Jul 27
2018
FDA approvalOrphan drug
Parenteral nutrition-associated cholestasis· Fresenius Kabi USA, LLC
Jul 27
2018
label expansion
· Baxter Healthcare Corporation· NDA019734
Jul 20
2018
FDA approvalOrphan drug
Spondyloepiphyseal dysplasia-brachydactyly-speech disorder syndrome· GlaxoSmithKline Intellectual Property Development Ltd. England
Jul 11
2018
Jul 5
2018
label expansion
Jun 29
2018
Jun 27
2018
FDA approval
Jun 21
2018
FDA approvalOrphan drug
Familial isolated hypertrophic cardiomyopathy· Belcher Pharmaceuticals, LLC
Jun 19
2018
label expansion
Jun 19
2018
label expansion
Jun 14
2018
FDA approval
· Slate Run Pharmaceuticals, LLC· ANDA210015
May 24
2018
FDA approval
May 15
2018
May 4
2018
FDA approvalOrphan drug
Cortical dysgenesis with pontocerebellar hypoplasia due to TUBB3 mutation· Novartis Pharmaceuticals Corporation
May 4
2018
label expansion
May 4
2018
label expansion
May 3
2018
FDA approvalOrphan drug
Congenital vitamin K-dependent coagulation factors deficiency· Alexion Pharmaceuticals, Inc.
May 2
2018
May 1
2018
label expansion
Apr 30
2018
label expansion
Apr 30
2018
label expansion
Apr 23
2018
FDA approvalOrphan drug
Autosomal dominant polycystic kidney disease· Otsuka Pharmaceutical Company, Ltd.
Apr 23
2018
Apr 20
2018
label expansion
Apr 10
2018
label expansion
Mar 27
2018
Mar 23
2018
label expansion
· Prasco Laboratories· NDA022580
Mar 13
2018
FDA approval
Mar 9
2018
label expansion
Mar 7
2018
label expansion
· Aurobindo Pharma Limited· ANDA078539
Mar 6
2018
FDA approvalOrphan drug
Acute disseminated encephalomyelitis without anti-MOG antibodies· Theratechnologies Inc.
Mar 1
2018
label expansion
Feb 12
2018
FDA approval
Feb 12
2018
FDA approval
Jan 29
2018
label expansion
Jan 25
2018
label expansion
Jan 12
2018
FDA approvalOrphan drug
Non-small cell lung cancer· Boehringer Ingelheim Pharmaceuticals, Inc.
Jan 10
2018
label expansion
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.