The Breakthrough therapies chart is a list of all publicly announced breakthrough therapy designations since the program’s inception in 2012. The chart is updated daily and includes information about each individual agent or trade name of a drug or treatment, the sponsor company, the date of the designation’s public disclosure, the individual agent’s indication, or the broad disease category in which the designation was granted. The interactive search and sort feature lets you filter your results and download the table at any time into an excel format. Click here to learn more about the history of Breakthrough therapies. As of August 31, 2021, the FDA has approved 251* breakthrough therapy designated products and lists that there have been 1182 total requests for the designation with 458** designations granted.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 7/01/2017
Approval Date: 12/19/2018
Indication: Adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC)
Category: Cancer
Trade Name (Agent): Elzonris (tagraxofusp)
Sponsor: Stemline Therapeutics, Inc.
Date of BT Designation Disclosure: 6/15/2018
Approval Date: 12/21/2018
Indication: Adults and children aged 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN)
Category: Cancer
Trade Name (Agent): Kisquali Femara Co-Pack (letrozole and ribociclib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 1/10/2018
Approval Date: 2/13/2019
Indication: Treatment of pre/perimenopausal women with HR-positive, HER2- negative advanced or metastatic breast cancer
Category: Cancer
Trade Name (Agent): Spravato (esketamine)
Sponsor: Janssen Pharmaceuticals Inc.
Date of BT Designation Disclosure: 11/15/2013
Approval Date: 3/05/2019
Indication: Patients with treatment-resistant depression
Category: Other
Trade Name (Agent): Dupixent (Dupilumab)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/01/2016
Approval Date: 3/11/2019
Indication: Patients 12 to less than 18 years of age with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable
Category: Other
Trade Name (Agent): Libtayo (cemiplimab-rwlc)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 9/08/2017
Approval Date: 9/28/2018
Indication: Patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation
Category: Cancer
Trade Name (Agent): Vitrakvi (larotrectinib)
Sponsor: Bayer Healthcare Pharmaceuticals Inc.
Date of BT Designation Disclosure: 7/13/2016
Approval Date: 11/26/2018
Indication: Adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion who have no satisfactory alternative treatments or that have progressed following treatment
Category: Cancer
Trade Name (Agent): Brukinsa (zanubrutinib)
Sponsor: BeiGene USA, Inc.
Date of BT Designation Disclosure: 1/16/2019
Approval Date: 11/14/2019
Indication: Patients with mantle cell lymphoma (MCL) who have received at least one prior therapy
Category: Cancer
Trade Name (Agent): Balversa (erdafitinib)
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 3/15/2018
Approval Date: 4/12/2019
Indication: Adult patients with locally advanced or metastatic urothelial carcinoma (mUC) that has susceptible FGFR3 or FGFR2 genetic alterations and progressed during at least on line of platinum-containing chemotherapy
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharpe & Dohme Corp.
Date of BT Designation Disclosure: 12/10/2017
Approval Date: 4/19/2019
Indication: In combination with axitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Vyndaquel (tafamidis meglumine)
Sponsor: Foldrx Pharmaceuticals, Inc. Sub. Pfizer, Inc.
Date of BT Designation Disclosure: 5/24/2018
Approval Date: 5/03/2019
Indication: Patients with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular related hospitalization
Category: Other
Trade Name (Agent): Kadcyla (ado-trastuzumab emtansine)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 2/05/2019
Approval Date: 5/03/2019
Indication: Adjuvant treatment of patients with HER2-positive early breast cancer who have residual disease after neoadjuvant taxane and trastuzumab-based treatment
Category: Cancer
Trade Name (Agent): Bavencio (avelumab)
Sponsor: EMD Serono Inc.
Date of BT Designation Disclosure: 12/22/2017
Approval Date: 5/14/2019
Indication: In combination with Inlyta (axitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Venclexta (venetoclax)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 3/15/2019
Approval Date: 5/15/2019
Indication: In combination with obinutuzumab for treatment of previously untreated patients with chronic lymphocytic leukemia (1L CLL) or small lymphocytic lymphoma (SLL)
Category: Cancer
Trade Name (Agent): Emgality (galcanezumab)
Sponsor: Eli Lilly & Company
Date of BT Designation Disclosure: 11/15/2018
Approval Date: 6/04/2019
Indication: Episodic cluster headache in adults
Category: Rare Inherited Disorders
Trade Name (Agent): Polivy (polatuzumab vedotin-piiq)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 12/10/2017
Approval Date: 6/10/2019
Indication: In combination with bendamustine and a rituximab product for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, after at least two prior therapies
Category: Cancer
Trade Name (Agent): Turalio (pexidartinib)
Sponsor: Daiichi Sankyo
Date of BT Designation Disclosure: 10/30/2015
Approval Date: 8/02/2019
Indication: Adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery
Category: Cancer
Trade Name (Agent): Rozlytrek (entrectinib)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 5/15/2017
Approval Date: 8/15/2019
Indication: Adult and pediatric patients 12 years of age and older with solid tumors that have a NTRK gene fusion without a known acquired resistance mutation, are metastatic, and have progressed following treatment or have no alternative treatment option
Category: Cancer
Trade Name (Agent): Promacta (eltrombopag)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 1/04/2018
Approval Date: 11/16/2018
Indication: First-line treatment for adults and pediatric patients two years and older with SAA in combination with standard immunosuppressive therapy (IST)
Category: Rare Inherited Disorders
Trade Name (Agent): Ibrance (palbociclib)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 2017
Approval Date: 4/04/2019
Indication: In combination with an aromatase inhibitor, or in combination with fulvestrant, for treatment of male patients with advanced or metastatic breast cancer
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 8/01/2018
Approval Date: 9/17/2019
Indication: In combination with lenvatinib for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for surgery or radiation
Category: Cancer
Trade Name (Agent): Adakveo (crizanlizumab-tmca)
Sponsor: Novartis Pharmaceuticals Corporation
Date of BT Designation Disclosure: 1/08/2019
Approval Date: 11/15/2019
Indication: To reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease
Category: Rare Inherited Disorders
Trade Name (Agent): Calquence (acalabrutinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/14/2019
Approval Date: 11/21/2019
Indication: Adult patients with untreated chronic lymphocytic leukemia (CLL)
Category: Cancer
Trade Name (Agent): Calquence (acalabrutinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/14/2019
Approval Date: 11/21/2019
Indication: Adult patients with relapsed or refractory chronic lymphocytic leukemia
Category: Cancer
Trade Name (Agent): Padcev (enfortumab vedotin-ejfv)
Sponsor: Astellas Pharma US, Inc.
Date of BT Designation Disclosure: 3/26/2018
Approval Date: 12/18/2019
Indication: Patients with locally advanced or metastatic urothelial cancer who have previously received a PD-1 or PD-L1 inhibitor, and a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting
Category: Cancer
Trade Name (Agent): omidubicel
Sponsor: Gamida Cell Ltd.
Date of BT Designation Disclosure: 10/11/2016
Indication: Patients with high-risk blood cancers who do not have a suitable matched donor
Category: Cancer
Trade Name (Agent): Tibosovo (ivosidenib)
Sponsor: Servier Pharmaceuticals
Date of BT Designation Disclosure: 3/27/2019
Approval Date: 5/25/2022
Indication: In combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.
Category: Cancer
Trade Name (Agent): Cosela (trilaciclib)
Sponsor: Cosela to G1 Therapeutics, Inc.
Date of BT Designation Disclosure: 8/07/2019
Approval Date: 2/12/2021
Indication: Reduce the frequency of chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage (when the cancer has spread beyond the lungs) small cell lung cancer.
Category: Cancer
Trade Name (Agent): B38M (JNJ‐4528)
Sponsor: Legend Biotech + Janssen Global Services
Date of BT Designation Disclosure: 12/06/2019
Indication: Patients with previously treated multiple myeloma
Category: Cancer
Trade Name (Agent): Enhertu (fam-trastuzumab deruxtecan-nxki)
Sponsor: Daiichi Sankyo Co.
Date of BT Designation Disclosure: 5/08/2019
Approval Date: 12/20/2019
Indication: Adults with unresectable (unable to be removed with surgery) or metastatic (when cancer cells spread to other parts of the body) HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting
Category: Cancer
Trade Name (Agent): Oxbryta (voxelotor)
Sponsor: Global Blood Therapeutics, Inc.
Date of BT Designation Disclosure: 1/09/2018
Approval Date: 11/25/2019
Indication: Sickle cell disease (SCD) in patients 12 years and older
Category: Rare Inherited Disorders
Trade Name (Agent): Lenvima (lenvatinib)
Sponsor: Eisai Co.
Date of BT Designation Disclosure: 8/01/2018
Approval Date: 9/17/2019
Indication: In combination with pembrolizumab for the treatment of patients with previously advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for surgery or radiation
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 4/18/2016
Approval Date: 3/14/2017
Indication: Patients with hematological malignancies: Hodgkin Lymphoma
Category: Cancer
Trade Name (Agent): Kisqali (ribociclib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 8/03/2016
Approval Date: 3/13/2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer as initial endocrine-based therapy
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 6/27/2016
Approval Date: 2/02/2017
Indication: Patients with locally advanced or metastatic urothelial carcinoma in patients who have disease progression during or following platinum-containing chemotherapy; have disease progression within 12 months neo- or neoadjuvant treatment with platinum chemo
Category: Cancer
Trade Name (Agent): Rubraca (rucaparib)
Sponsor: Clovis Oncology Inc.
Date of BT Designation Disclosure: 4/06/2016
Approval Date: 12/19/2016
Indication: Patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more chemotherapies
Category: Cancer
Trade Name (Agent): Bavencio (avelumab)
Sponsor: EMD Serono Inc.
Date of BT Designation Disclosure: 11/18/2015
Approval Date: 3/23/2017
Indication: Adult and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC)
Category: Cancer
Trade Name (Agent): Zejula (niraparib)
Sponsor: Tesaro Inc.
Date of BT Designation Disclosure: 10/14/2016
Approval Date: 3/27/2017
Indication: Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy
Category: Cancer
Trade Name (Agent): Dupixent (dupilumab)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 11/20/2014
Approval Date: 3/28/2017
Indication: Adult patients with moderate-to-severe atopic dermatitis (AD) whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable
Category: Other
Trade Name (Agent): Ocrevus (ocrelizumab)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 2/17/2016
Approval Date: 3/28/2017
Indication: Adult patients with primary progressive forms of multiple sclerosis
Category: Rare Inherited Disorders
Trade Name (Agent): Ibrance (palbociclib)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 2017
Approval Date: 3/31/2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, in combination with an aromatase inhibitor as initial endocrine-based therapy
Category: Cancer
Trade Name (Agent): Brineura (cerliponase alfa)
Sponsor: Biomarin Pharmaceutical Inc.
Date of BT Designation Disclosure: 8/27/2015
Approval Date: 4/27/2017
Indication: Pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency
Category: Rare Inherited Disorders
Trade Name (Agent): Besponsa (inotuzumab ozogamicin)
Sponsor: Wyeth Pharmaceuticals LLC
Date of BT Designation Disclosure: 2017
Approval Date: 8/17/2017
Indication: Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)
Category: Cancer
Trade Name (Agent): Darzalex (daratumumab)
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 7/26/2016
Approval Date: 11/21/2016
Indication: Patients with multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone
Category: Cancer
Trade Name (Agent): Ingrezza (valbenazine)
Sponsor: Neurocrine Biosciences Inc.
Date of BT Designation Disclosure: 10/30/2014
Approval Date: 4/11/2017
Indication: Patients with tardive dyskinesia
Category: Other
Trade Name (Agent): Alunbrig (brigatinib)
Sponsor: Ariad Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/01/2014
Approval Date: 4/28/2017
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive mestastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib
Category: Cancer
Trade Name (Agent): Kisqali Femara Co-Pack (letrozole and ribociclib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 8/02/2016
Approval Date: 5/04/2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer
Category: Cancer
Trade Name (Agent): Imfinzi (durvalumab)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 2/16/2016
Approval Date: 5/01/2017
Indication: Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neo- or adjuvant treatment with platinum-containing chemo
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 2017
Approval Date: 5/23/2017
Indication: Adult and pediatric patients with unresectable or metastatic MSI-H or mismatch repair deficient solid tumors that have progressed following prior treatment or metastatic, MSI-H or mistmatch repair colorectal cancer that has progressed following treatment
Category: Cancer
Trade Name (Agent): Zykadia (ceritinib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 2017
Approval Date: 5/26/2017
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA approved test
Category: Cancer
Trade Name (Agent): Mekinist (trametinib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 2017
Approval Date: 6/22/2017
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test
Category: Cancer
Trade Name (Agent): Tafinlar (dabrafenib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 7/24/2015
Approval Date: 6/22/2017
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test
Category: Cancer
Trade Name (Agent): Imbruvica (ibrutinib)
Sponsor: Pharmacyclics LLC
Date of BT Designation Disclosure: 6/29/2016
Approval Date: 8/02/2017
Indication: Adult patients with chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy
Category: Infectious Disease
Trade Name (Agent): VYXEOS (cytarabine and daunorubicin)
Sponsor: Celator Pharmaceuticals Inc.
Date of BT Designation Disclosure: 5/20/2016
Approval Date: 8/03/2017
Indication: Adult patients with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)
Category: Cancer
Trade Name (Agent): Mavyret (glecaprevir & pibrentasvir)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 9/30/2016
Approval Date: 8/03/2017
Indication: Patients with chronic hepatitis C virus (HCV) genotype (GT) 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis; and also for patients with HCV GT1 infection previously treated w/ HCV NS5A or NS3/4A inhibitor but not both
Category: Infectious Disease
Trade Name (Agent): Rydapt (midostaurin)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 1/29/2016
Approval Date: 4/28/2017
Indication: Adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive, as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation
Category: Cancer
Trade Name (Agent): Actemra (tocilizumab)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 10/05/2016
Approval Date: 5/22/2017
Indication: Adult patients with giant cell arteritis (GCA)
Category: Rare Inherited Disorders
Trade Name (Agent): Trogarzo (ibalizumab)
Sponsor: Theratechnologies Inc.
Date of BT Designation Disclosure: 2/27/2015
Approval Date: 3/06/2018
Indication: 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
Category: Infectious Disease
Trade Name (Agent): Verzenio (abemaciclib)
Sponsor: Eli Lilly and Co.
Date of BT Designation Disclosure: 10/08/2015
Approval Date: 9/28/2017
Indication: Patients with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting
Category: Cancer
Trade Name (Agent): Calquence (acalabrutinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/01/2017
Approval Date: 10/31/2017
Indication: Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy
Category: Cancer
Trade Name (Agent): Alecensa (alectinib)
Sponsor: Hoffman La Roche Inc.
Date of BT Designation Disclosure: 9/23/2013
Approval Date: 11/06/2017
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive, metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test
Category: Cancer
Trade Name (Agent): Zelboraf (vemurafenib)
Sponsor: Hoffmann La Roche Inc.
Date of BT Designation Disclosure: 8/07/2017
Approval Date: 11/06/2017
Indication: Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation
Category: Other
Trade Name (Agent): Prevymis (letermovir)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 2/27/2017
Approval Date: 11/08/2017
Indication: Prophylaxis of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT)
Category: Infectious Disease
Trade Name (Agent): Adcetris (brentuximab vedotin)
Sponsor: SEATTLE GENETICS INC
Date of BT Designation Disclosure: 10/02/2017
Approval Date: 11/09/2017
Indication: Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy
Category: Cancer
Trade Name (Agent): Hemlibra (emicizumab-kxwh)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 9/04/2015
Approval Date: 11/16/2017
Indication: Prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A with factor VIII inhibitors
Category: Rare Inherited Disorders
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 10/17/2017
Approval Date: 12/20/2017
Indication: Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection
Category: Cancer
Trade Name (Agent): Symdeko (tezacaftor and ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 1/28/2014
Approval Date: 2/12/2018
Indication: Patients with cystic fibrosis aged 12 year and older who are homozygous for the F508del mutation who have at least one mutation in the CTFR gene that is responsive to Symdeko based on in vitro data or clinical evidence
Category: Rare Inherited Disorders
Trade Name (Agent): Imfinzi (durvalumab)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 7/31/2017
Approval Date: 2/16/2018
Indication: Patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy
Category: Cancer
Trade Name (Agent): Austedo (deutetrabenazine)
Sponsor: Teva Branded Pharmaceutical Products R and D Inc.
Date of BT Designation Disclosure: 11/09/2015
Approval Date: 8/30/2017
Indication: Patients with tardive dyskinesia
Category: Other
Trade Name (Agent): Adcetris (brentuximab vedotin)
Sponsor: SEATTLE GENETICS INC
Date of BT Designation Disclosure: 10/15/2017
Approval Date: 3/20/2018
Indication: Treatment for adult patients with previously untreated Stage III or IV classical Hodgkin Lymphoma, in combination with chemotherapy
Category: Cancer
Trade Name (Agent): Venclexta (venetoclax)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 2018
Approval Date: 6/08/2018
Indication: Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma, with or without 17p deletion, who have received at least one prior therapy
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2/16/2018
Approval Date: 4/16/2018
Indication: In combination with ipilimumab, for treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC
Category: Cancer
Trade Name (Agent): Yervoy (ipilimumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2/16/2018
Approval Date: 4/16/2018
Indication: In combination with nivolumab, for treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Crysvita (burosumab)
Sponsor: Kyowa Kirin Inc.
Date of BT Designation Disclosure: 6/28/2016
Approval Date: 4/17/2018
Indication: Patients aged 1 year and older with x-linked hypophosphatemia (XLH)
Category: Rare Inherited Disorders
Trade Name (Agent): Tagrisso (osimertinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 10/09/2017
Approval Date: 4/18/2018
Indication: First-line treatment of patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions of L858R substitution mutations, as detected by an FDA approved test
Category: Cancer
Trade Name (Agent): Tafinlar (dabrafenib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 4/30/2018
Indication: In combination with trametinib for the adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection
Category: Cancer
Trade Name (Agent): Mekinist (trametinib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 4/30/2018
Indication: In combination with dabrafenib for the adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 2017
Approval Date: 5/18/2017
Indication: Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or neo-adjuvant treatment with platinum-containing chemotherapy
Category: Cancer
Trade Name (Agent): Mekinist (trametinib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 5/04/2018
Indication: 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
Category: Cancer
Trade Name (Agent): Tafinlar (dabrafenib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 5/04/2018
Indication: In combination with trametinib 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
Category: Cancer
Trade Name (Agent): Gilenya (fingolimod)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 12/18/2017
Approval Date: 5/11/2018
Indication: Patients with relapsing multiple sclerosis (MS) to include pediatric patients 10 years of age and above
Category: Rare Inherited Disorders
Trade Name (Agent): Rituximab (Rituxan)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 3/24/2017
Approval Date: 6/07/2018
Indication: Patients with moderate to severe pemphigus vulgaris (PV)
Category: Rare Inherited Disorders
Trade Name (Agent): Orkambi (lumacaftor and ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 1/29/2014
Approval Date: 7/02/2015
Indication: Patients 12 years and older with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene
Category: Rare Inherited Disorders
Trade Name (Agent): Eylea (aflibercept)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 9/16/2014
Approval Date: 3/25/2015
Indication: Treatment of diabetic retinopathy (DR) in patients with diabetic macular edema (DME)
Category: Other
Trade Name (Agent): Kalydeco (ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 12/15/2012
Approval Date: 3/17/2015
Indication: Patients 2 years and older with cystic fibrosis and have that one of the following CFTR gene mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H
Category: Rare Inherited Disorders
Trade Name (Agent): Lucentis (ranibizumab)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 12/15/2014
Approval Date: 2/06/2015
Indication: Treatment of Diabetic Retinopathy (DR) in patients with Diabetic Macula Edema (DME)
Category: Other
Trade Name (Agent): Xuriden (uridine triacetate)
Sponsor: Wellstat Therapeutics Corp.
Date of BT Designation Disclosure: 4/30/2014
Approval Date: 9/04/2015
Indication: Patients with hereditary orotic aciduria
Category: Rare Inherited Disorders
Trade Name (Agent): Blincyto (blinatumomab)
Sponsor: Amgen Inc.
Date of BT Designation Disclosure: 7/01/2014
Approval Date: 12/03/2014
Indication: Patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)
Category: Cancer
Trade Name (Agent): Viekira Pak (ombitasvir, paritaprevir, ritonavir)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 5/06/2013
Approval Date: 12/19/2014
Indication: Patients with genotype 1 chronic hepatitis C virus (HCV) infection including those with compensated cirrhosis
Category: Infectious Disease
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2/15/2013
Approval Date: 12/22/2014
Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor
Category: Cancer
Trade Name (Agent): Technivie (ombitasvir, paritaprevir, ritonavir)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 6/30/2014
Approval Date: 7/24/2015
Indication: Patients with genotype 4 chronic hepatitis C viruse (HCV) infection without cirrhosis
Category: Infectious Disease
Trade Name (Agent): Esbriet (pirfenidone)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 7/17/2014
Approval Date: 10/15/2014
Indication: Patients with idiopathic pulmonary fibrosis (IPF)
Category: Other
Trade Name (Agent): Kalydeco (ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 2014
Approval Date: 12/29/2014
Indication: Patients age 6 years and older with cystic fibrosis who have the R117H mutation in the CF transmembrane conductance regulator (CTFR gene)
Category: Rare Inherited Disorders
Trade Name (Agent): Cabotetyx (cabozantinib)
Sponsor: Exelixis Inc.
Date of BT Designation Disclosure: 8/24/2015
Approval Date: 4/25/2016
Indication: Patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy
Category: Cancer
Trade Name (Agent): Venclexta (venetoclax)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 5/06/2015
Approval Date: 4/11/2016
Indication: Patients with chronic lymphocytic leukemia (CLL) with 17p deletion who have received at least one prior therapy
Category: Cancer
Trade Name (Agent): Ilaris (canakinumab)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 4/27/2016
Approval Date: 9/23/2016
Indication: Patients with Hyperimmunoglobulin D Syndrome (HIDS)
Category: Rare Inherited Disorders
Trade Name (Agent): Alecensa (alectinib)
Sponsor: Hoffman La Roche Inc.
Date of BT Designation Disclosure: 6/15/2013
Approval Date: 12/11/2015
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), who have progressed on or are intolerant to crizotinib
Category: Cancer
Trade Name (Agent): Kanuma (sebelipase alfa)
Sponsor: Alexion Pharmaceuticals Inc.
Date of BT Designation Disclosure: 5/20/2013
Approval Date: 12/08/2015
Indication: Patients with a diagnosis of lysosomal acid lipase (LAL) deficiency
Category: Rare Inherited Disorders
Trade Name (Agent): Harvoni (sofosbuvir/ledipasvir)
Sponsor: Gilead Sciences Inc.
Date of BT Designation Disclosure: 7/25/2013
Approval Date: 10/10/2014
Indication: Patients with chronic hepatitis C, genotype 1 infection
Category: Infectious Disease
Trade Name (Agent): Ofev (nintedanib)
Sponsor: Boehringer Ingelheim, Boehringer Ingelheim Pharmaceuticals Inc.
Date of BT Designation Disclosure: 7/16/2014
Approval Date: 10/15/2014
Indication: Patients with idiopathic pulmonary fibrosis (IPF)
Category: Other
