Skip to content

Breakthrough Therapies

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.

  • Filter by Sponsor(s)

  • Category

  • FDA Status

  • Reset

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