Trade Name (Agent): IMGN632
Sponsor: ImmunoGen, Inc
Date of BT Designation Disclosure: 10/05/2020
Indication: Treatment of relapsed or refractory blastic plasmacytoid dendritic cell neoplasm
Category: Cancer
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): IMGN632
Sponsor: ImmunoGen, Inc
Date of BT Designation Disclosure: 10/05/2020
Indication: Treatment of relapsed or refractory blastic plasmacytoid dendritic cell neoplasm
Category: Cancer
Trade Name (Agent): Farxiga® (dapagliflozin)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 10/02/2020
Approval Date: 4/30/2021
Indication: Patients with chronic kidney disease (CKD), with and without type 2 diabetes (T2D)
Category: Other
Trade Name (Agent): BI 1482694
Sponsor: Boehringer Ingelheim
Date of BT Designation Disclosure: 12/18/2015
Indication: Advanced/pre-treated EGFR mutation-positive NSCLC
Category: Cancer
Trade Name (Agent): Pembrolizumab (Keytruda)
Sponsor: Merck
Date of BT Designation Disclosure: 11/02/2015
Indication: Microsatellite Instability-High (MSI-H) metastatic colorectal cancer (mCRC)
Category: Cancer
Trade Name (Agent): Inotuzumab Ozogamicin
Sponsor: Pfizer
Date of BT Designation Disclosure: 10/19/2015
Indication: ALL
Category: Cancer
Trade Name (Agent): RBX2660
Sponsor: Rebiotix, Inc.
Date of BT Designation Disclosure: 10/12/2015
Indication: Recurrent Clostridium difficile (C diff) infection
Category: Infectious Disease
Trade Name (Agent): BMS-663068
Sponsor: BMS
Date of BT Designation Disclosure: 7/21/2015
Indication: HIV-1 in heavily treatment-experienced adult patients
Category: Infectious Disease
Trade Name (Agent): DX-2930
Sponsor: Dyax
Date of BT Designation Disclosure: 7/07/2015
Indication: Hereditary angioedema (HAE)
Category: Rare Inherited Disorders
Trade Name (Agent): VTS 270
Sponsor: Vtesse
Date of BT Designation Disclosure: 1/06/2016
Indication: Niemann-Pick Type C1 Disease (NPC)
Category: Rare Inherited Disorders
Trade Name (Agent): Lynparza (olaparib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 1/27/2016
Indication: Metastatic Castration Resistant Prostate Cancer
Category: Cancer
Trade Name (Agent): Venetoclax
Sponsor: AbbVie Inc., Roche
Date of BT Designation Disclosure: 1/28/2016
Indication: AML combination therapy
Category: Cancer
Trade Name (Agent): Rapastinel (GLYX-13)
Sponsor: Allergan
Date of BT Designation Disclosure: 1/31/2016
Indication: Major Depressive Disorder (MDD)
Category: Other
Trade Name (Agent): Affinity enhanced T-cell therapy
Sponsor: Adaptimmune
Date of BT Designation Disclosure: 2/09/2016
Indication: NY-ESO in synovial sarcoma
Category: Cancer
Trade Name (Agent): Durvalumab (MEDI4736)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 2/17/2016
Indication: PD-L1+ inoperable/metastatic urothelial bladder cancer
Category: Cancer
Trade Name (Agent): PKC412 (midostaurin)
Sponsor: Novartis
Date of BT Designation Disclosure: 2/19/2016
Indication: Newly-diagnosed FLT3-mutated AML
Category: Cancer
Trade Name (Agent): NI-0501
Sponsor: Novimmune
Date of BT Designation Disclosure: 3/16/2016
Indication: Primary Hemophagocytic Lymphohistiocytosis (HLH)
Category: Rare Inherited Disorders
Trade Name (Agent): SER-109
Sponsor: Seres
Date of BT Designation Disclosure: 6/12/2015
Indication: Clostridium difficile infection (CDI)
Category: Infectious Disease
Trade Name (Agent): ACTEMRA (tocilizumab)
Sponsor: Genentech/Roche
Date of BT Designation Disclosure: 6/10/2015
Approval Date: 3/04/2021
Indication: Systemic scerlosis
Category: Other
Trade Name (Agent): MYDICAR
Sponsor: Celladon
Date of BT Designation Disclosure: 4/10/2014
Indication: Heart failure
Category: Cardiovascular
Trade Name (Agent): Rociletinib (CO-1686)
Sponsor: Clovis Oncology
Date of BT Designation Disclosure: 5/19/2014
Indication: NSCLC
Category: Cancer
Trade Name (Agent): CRS-207 and GVAX
Sponsor: Aduro
Date of BT Designation Disclosure: 7/21/2014
Indication: Pancreatic cancer
Category: Cancer
Trade Name (Agent): AP26113
Sponsor: Ariad Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/02/2014
Indication: ALK-positive NSCLC
Category: Cancer
Trade Name (Agent): SPK-RPE65
Sponsor: Spark Therapeutics
Date of BT Designation Disclosure: 11/06/2014
Indication: Inherited retinal dystrophy (IRD)
Category: Rare Inherited Disorders
Trade Name (Agent): JCAR015
Sponsor: Juno Therapeutics
Date of BT Designation Disclosure: 11/24/2014
Indication: ALL
Category: Cancer
Trade Name (Agent): Ixazomib
Sponsor: Takeda
Date of BT Designation Disclosure: 12/02/2014
Indication: Systemic light-chain (AL) amyloidosis
Category: Other
Trade Name (Agent): Obeticholic acid (OCA)
Sponsor: Intercept
Date of BT Designation Disclosure: 1/29/2015
Indication: Nonalcoholic steatohepatitis (NASH)
Category: Other
Trade Name (Agent): Olipudase alfa
Sponsor: Sanofi/Genzyme
Date of BT Designation Disclosure: 6/04/2015
Indication: Niemann-Pick disease Type B
Category: Rare Inherited Disorders
Trade Name (Agent): Viaskin Peanut
Sponsor: DBV
Date of BT Designation Disclosure: 4/09/2015
Indication: Peanut allergy in children
Category: Other
Trade Name (Agent): Rucaparib
Sponsor: Clovis
Date of BT Designation Disclosure: 4/06/2015
Indication: Ovarian cancer
Category: Cancer
Trade Name (Agent): EBV-CTL
Sponsor: Atara and MSKCC
Date of BT Designation Disclosure: 3/02/2015
Indication: EBV-associated lymphoproliferative disease
Category: Cancer
Trade Name (Agent): Rindopepimut (Rintega)
Sponsor: Celldex
Date of BT Designation Disclosure: 2/23/2015
Indication: Glioblastoma (GBM)
Category: Cancer
Trade Name (Agent): MPDL3280A
Sponsor: Genentech/Roche
Date of BT Designation Disclosure: 2/01/2015
Indication: Non-small cell lung cancer
Category: Cancer
Trade Name (Agent): Ivacaftor (Kalydeco)
Sponsor: Vertex
Date of BT Designation Disclosure: 12/15/2012
Indication: CF combination therapy
Category: Rare Inherited Disorders
Trade Name (Agent): PVS-RIPO
Sponsor: Duke Medicine
Date of BT Designation Disclosure: 5/15/2016
Indication: Recurrent glioblastoma multiforme (GBM)
Category: Cancer
Trade Name (Agent): SHP621 (budesonide oral suspension, or BOS)
Sponsor: Shire
Date of BT Designation Disclosure: 6/13/2016
Indication: Eosinophilic esophagitis (EoE)
Category: Rare Inherited Disorders
Trade Name (Agent): Ivacaftor (Kalydeco)
Sponsor: Vertex
Date of BT Designation Disclosure: 2014
Approval Date: 3/17/2015
Indication: CF monotherapy, patients 2 years and older with one of the following CFTR gene mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H
Category: Rare Inherited Disorders
Trade Name (Agent): Ivacaftor (Kalydeco)
Sponsor: Vertex
Date of BT Designation Disclosure: 2014
Approval Date: 12/29/2014
Indication: CF monotherapy, patients 6 years or older with R117H mutationin CFTR gene
Category: Rare Inherited Disorders
Trade Name (Agent): SPK-9001
Sponsor: Pfizer/Spark
Date of BT Designation Disclosure: 7/21/2016
Indication: Hemophilia B.
Category: Rare Inherited Disorders
Trade Name (Agent): V920
Sponsor: Merck
Date of BT Designation Disclosure: 7/25/2016
Indication: Ebola Zaire
Category: Infectious Disease
Trade Name (Agent): Pracinostat
Sponsor: MEI Pharma
Date of BT Designation Disclosure: 8/01/2016
Indication: In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy
Category: Cancer
Trade Name (Agent): Esketamine
Sponsor: Janssen
Date of BT Designation Disclosure: 8/16/2016
Approval Date: 7/31/2020
Indication: Major depressive disorder with imminent risk for suicide
Category: Other
Trade Name (Agent): SL-401
Sponsor: Stemline Therapeutics
Date of BT Designation Disclosure: 8/23/2016
Indication: Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123)
Category: Rare Inherited Disorders
Trade Name (Agent): Daclatasvir
Sponsor: BMS
Date of BT Designation Disclosure: 4/25/2013
Indication: Hepatitis C combination therapy
Category: Infectious Disease
Trade Name (Agent): SD101
Sponsor: Scioderm
Date of BT Designation Disclosure: 4/29/2013
Indication: Epidermolysis bullosa
Category: Other
Trade Name (Agent): LIVMARLI (maralixibat)
Sponsor: Shire
Date of BT Designation Disclosure: 6/13/2016
Approval Date: 9/29/2021
Indication: Progressive familial intrahepatic cholestasis type 2 (PFIC2)
Category: Rare Inherited Disorders
Trade Name (Agent): Daclatasvir/asunaprevir combination
Sponsor: BMS
Date of BT Designation Disclosure: 2/24/2014
Indication: Hepatitis C
Category: Infectious Disease
Trade Name (Agent): Esketamine
Sponsor: J&J
Date of BT Designation Disclosure: 1/21/2014
Indication: Treatment resistant depression
Category: Other
Trade Name (Agent): Dabrafenib
Sponsor: GlaxoSmithKline
Date of BT Designation Disclosure: 1/13/2014
Indication: NSCLC w/ BRAF mutation
Category: Cancer
Trade Name (Agent): cPMP (ALXN1011)
Sponsor: Alexion Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/24/2013
Indication: Molybdenum cofactor deficiency (MoCD) type A
Category: Rare Inherited Disorders
Trade Name (Agent): Grazoprevir/Elbasvir
Sponsor: Merck
Date of BT Designation Disclosure: 10/22/2013
Indication: Hepatitis C
Category: Infectious Disease
Trade Name (Agent): Volasertib
Sponsor: Boehringer Ingelheim
Date of BT Designation Disclosure: 9/17/2013
Indication: AML combination therapy
Category: Cancer
Trade Name (Agent): Entinostat
Sponsor: Syndax
Date of BT Designation Disclosure: 9/11/2013
Indication: ER-positive metastatic breast cancer combination
Category: Cancer
Trade Name (Agent): BYM338 (Bimagrumab)
Sponsor: Novartis
Date of BT Designation Disclosure: 8/20/2013
Indication: Sporadic inclusion body myositis (sIBM)
Category: Other
Trade Name (Agent): Drisapersen
Sponsor: GlaxoSmithKline/Prosensa
Date of BT Designation Disclosure: 6/27/2013
Indication: Duchenne muscular dystrophy (DMD)
Category: Rare Inherited Disorders
Trade Name (Agent): Serelaxin
Sponsor: Novartis
Date of BT Designation Disclosure: 6/21/2013
Indication: Acute heart failure
Category: Cardiovascular
Trade Name (Agent): Alecensa (alectinib)
Sponsor: Roche
Date of BT Designation Disclosure: 10/04/2016
Indication: Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor
Category: Cancer
Trade Name (Agent): Elbit Imaging
Sponsor: Gamida Cell
Date of BT Designation Disclosure: 10/10/2016
Indication: Nicord
Category: Cancer
Trade Name (Agent): Acalabrutinib
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/01/2017
Indication: Mantle cell lymphoma for patients who have received at least one prior line of therapy
Category: Cancer
Trade Name (Agent): Venetoclax
Sponsor: AbbVie Inc., Roche
Date of BT Designation Disclosure: 8/01/2017
Indication: Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy
Category: Cancer
Trade Name (Agent): Methylenedioxymethamphetamine (MDMA)
Sponsor: MAPS
Date of BT Designation Disclosure: 8/28/2017
Indication: PTSD
Category: Other
Trade Name (Agent): EB-101
Sponsor: Abeona Therapeutics Inc
Date of BT Designation Disclosure: 8/30/2017
Indication: Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Category: Other
Trade Name (Agent): DAS181
Sponsor: Ansun BioPharma
Date of BT Designation Disclosure: 10/10/2017
Indication: Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients
Category: Infectious Disease
Trade Name (Agent): Valoctocogene Roxaparvovec
Sponsor: BioMarin
Date of BT Designation Disclosure: 10/26/2017
Indication: Hemophilia A
Category: Rare Inherited Disorders
Trade Name (Agent): Monjuvi
Sponsor: MorpoSys
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 7/31/2020
Indication: Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation
Category: Cancer
Trade Name (Agent): Avelumab in combination with Inlyta (axitinib)
Sponsor: EMD Serono and Pfizer
Date of BT Designation Disclosure: 12/21/2017
Indication: Treatment-naïve patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Lenvima (lenvatinib)
Sponsor: Merck and Eisai
Date of BT Designation Disclosure: 1/09/2018
Approval Date: 8/10/2021
Indication: In combination with pembrolizumab for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
Category: Cancer
Trade Name (Agent): Upadacitinib (ABT-494)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 1/09/2018
Indication: Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy
Category: Other
Trade Name (Agent): Livtencity (Maribavir)
Sponsor: Takeda Pharmaceutical Company Ltd.
Date of BT Designation Disclosure: 1/08/2018
Approval Date: 11/23/2021
Indication: Treatment of adults and pediatric patients 12 years of age and older and weighing at least 35 kg with posttransplant cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet.
Category: Infectious Disease
Trade Name (Agent): Imcivree (setmelanotide)
Sponsor: Rhythm Pharmaceutical, Inc.
Date of BT Designation Disclosure: 1/7/2016
Approval Date: 11/27/2020
Indication: Pro-opiomelanocortin (POMC) deficiency obesity and obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway (the MC4 pathway)
Category: Rare Inherited Disorders
Trade Name (Agent): Tonmya (TNX-102 SL)
Sponsor: Tonix Pharmaceuticals
Date of BT Designation Disclosure: 12/19/2016
Indication: PTSD
Category: Other
Trade Name (Agent): ABT-530
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 9/30/2016
Indication: genotype 1 HCV patients who have failed to respond to a previous therapy of direct acting antivirals (or DAAs)
Category: Infectious Disease
Trade Name (Agent): Neridronic acid
Sponsor: Grünenthal and Abiogen Pharma
Date of BT Designation Disclosure: 12/16/2016
Indication: Complex regional pain syndrome (CRPS)
Category: Rare Inherited Disorders
Trade Name (Agent): AMT-060
Sponsor: uniQure
Date of BT Designation Disclosure: 1/20/2017
Indication: Hemophilia B
Category: Rare Inherited Disorders
Trade Name (Agent): Toca 511 & Toca FC
Sponsor: Tocagen
Date of BT Designation Disclosure: 2/23/2017
Indication: Recurrent high grade glioma
Category: Cancer
Trade Name (Agent): Evkeeza (evinacumab-dgnb)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 4/07/2017
Approval Date: 2/11/2021
Indication: Homozygous familial hypercholesterolemia (HOFH)
Category: Rare Inherited Disorders
Trade Name (Agent): Rethymic (RVT-802)
Sponsor: Enzyvant
Date of BT Designation Disclosure: 4/14/2017
Approval Date: 10/8/2021
Indication: Treatment of pediatric patients with congenital athymia.
Category: Rare Inherited Disorders
Trade Name (Agent): Vonapanitase
Sponsor: Proteon Therapeutics, Inc.
Date of BT Designation Disclosure: 5/10/2017
Indication: Increasing arteriovenous fistula secondary patency (i.e., survival of the fistula without abandonment) and use for hemodialysis in patients on or expected to initiate hemodialysis
Category: Other
Trade Name (Agent): GMI-1271
Sponsor: GlycoMimetics
Date of BT Designation Disclosure: 5/17/2017
Indication: Adult Relapsed/Refractory Acute Myeloid Leukemia
Category: Cancer
Trade Name (Agent): Balovaptan
Sponsor: Roche
Date of BT Designation Disclosure: 1/29/2018
Indication: Autism spectrum disorder (ASD)
Category: Rare Inherited Disorders
Trade Name (Agent): Burtomab
Sponsor: Y-mAbs Therapeutics, Inc.
Date of BT Designation Disclosure: 6/07/2017
Indication: Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis
Category: Cancer
Trade Name (Agent): OMS721
Sponsor: Omeros
Date of BT Designation Disclosure: 6/13/2017
Indication: Immunoglobulin A (IgA) nephropathy
Category: Other
Trade Name (Agent): PTI-428
Sponsor:
Date of BT Designation Disclosure: 3/16/2018
Indication: Cystic Fibrosis for patients with two copies of the F508del mutation — one from their mother and one from their father — who are receiving Vertex’s Orkambi (lumacaftor/ivacaftor)
Category: Rare Inherited Disorders
Trade Name (Agent): tab-cel
Sponsor:
Date of BT Designation Disclosure: 2/15/2015
Indication: Patients with EBV+ PTLD following allogeneic hematopoietic stem cell transplant
Category: Other
Trade Name (Agent): bb2121
Sponsor:
Date of BT Designation Disclosure: 11/04/2017
Indication: Patients with relapsed or refractory multiple myeloma
Category: Cancer
Trade Name (Agent): ALIS (amikacin liposome inhalation suspension)
Sponsor:
Date of BT Designation Disclosure: 3/29/2018
Indication: Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex
Category: Other
Trade Name (Agent): Pedmark (sodium thiosulfate)
Sponsor:
Date of BT Designation Disclosure: 3/27/2018
Indication: Prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB)
Category: Other
Trade Name (Agent): SPK-8011
Sponsor:
Date of BT Designation Disclosure: 2/15/2018
Indication: Patients with hemophilia A.
Category: Rare Inherited Disorders
Trade Name (Agent): Ketamine
Sponsor:
Date of BT Designation Disclosure: 4/16/2018
Indication: Patients with depression
Category: Other
Trade Name (Agent): Ribaxamase (SYN-004)
Sponsor:
Date of BT Designation Disclosure: 5/11/2017
Indication: prevention of Clostridium difficile infection
Category: Infectious Disease
Trade Name (Agent): OMS721
Sponsor:
Date of BT Designation Disclosure: 4/26/2018
Indication: Patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patents who have persistent TMA despite modification of immunosupressive therapy
Category: Other
Trade Name (Agent): Trumenba (Meningococcal Group B Vaccine)
Sponsor:
Date of BT Designation Disclosure: 4/23/2018
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 1 through 9 years of age
Category:
Trade Name (Agent): Kymriah (tisagenlecleucel)
Sponsor:
Date of BT Designation Disclosure: 4/18/2017
Approval Date: 5/1/2018
Indication: Treatment of adults with relapsed/refractory diffuse large B-cell lymphoma (DLBCL).
Category: Cancer
Trade Name (Agent): Oxlumo (lumasiran)
Sponsor:
Date of BT Designation Disclosure: 3/12/2018
Approval Date: 11/23/2020
Indication: Primary Hyperoxaluria Type 1 (PH1)
Category: Infectious Disease
Trade Name (Agent): Trastuzumab Deruxtecan (DS-8201)
Sponsor:
Date of BT Designation Disclosure: 8/29/2017
Indication: HER2-positive, locally advanced, or metastatic breast cancer who have been treated with trastuzumab (Herceptin) and pertuzumab (Perjeta) and have disease progression after ado-trastuzumab emtansine (T-DM1; Kadcyla)
Category: Cancer
Trade Name (Agent): Balovaptan (RG7314)
Sponsor:
Date of BT Designation Disclosure: 1/28/2018
Indication: A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD)
Category: Other
Trade Name (Agent): Kymriah (CTL019)
Sponsor:
Date of BT Designation Disclosure: 4/18/2017
Indication: Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies
Category: Cancer
Trade Name (Agent): Korsuva (difelikefalin)
Sponsor:
Date of BT Designation Disclosure: 6/22/2017
Approval Date: 8/23/2021
Indication: Treatment of moderate-to-severe pruritus associated with chronic kidney disease (CKD-aP) in adults undergoing hemodialysis (HD).
Category: Other
Trade Name (Agent): ZX008 (fenfluramine)
Sponsor: Zogenix, Inc.
Date of BT Designation Disclosure: 2/06/2018
Indication: Patients who have seizures associated with Dravet syndrome
Category: Other
Trade Name (Agent): SAGE-217
Sponsor:
Date of BT Designation Disclosure: 2/07/2018
Indication: Patients with major depressive disorder (MDD)
Category: Other
Trade Name (Agent): PF-04965842
Sponsor:
Date of BT Designation Disclosure: 2/14/2018
Indication: Patients with moderate-to-severe atopic dermatitis (AD)
Category: Other