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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 218* breakthrough therapy designated products and lists that there have been 1182 total requests for the designation with 458** designations granted.

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Trade Name (Agent)

Sponsor

Date of BT Designation Disclosure

Approval Date

Indication

Category

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): 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): Firdapse (amifampridine)

Sponsor: Catalyst Pharmaceuticals Inc.

Date of BT Designation Disclosure: 8/27/2013

Approval Date: 11/28/2018

Indication: Adult patients with Lambert–Eaton myasthenic syndrome (LEMS)

Category: Other

Trade Name (Agent): Gamifant (emapalumab-Izsg)

Sponsor: Swedish Orphan Biovitrum AB (Publ.)

Date of BT Designation Disclosure: 2018

Approval Date: 11/20/2018

Indication: Adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy

Category: Rare Inherited Disorders

Trade Name (Agent): Lorbrena (lorlatinib)

Sponsor: Pfizer Inc.

Date of BT Designation Disclosure: 4/27/2017

Approval Date: 11/02/2018

Indication: Patients with ALK-positive metastatic NSCLC whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease or alectinib or ceritinib

Category: Cancer

Trade Name (Agent): Adcetris (brentuximab vedotin)

Sponsor: Seattle Genetics Inc.

Date of BT Designation Disclosure: 11/15/2018

Approval Date: 11/16/2018

Indication: Adult patients with previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL) in combination with cyclophosphamide, doxorubicin, and prednisone

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): 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): 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): Imfinzi (durvalumab)

Sponsor: AstraZeneca UK Ltd

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): 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): 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): 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): 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): 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): 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): 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): Calquence (acalabrutinib)

Sponsor: AstraZeneca UK Ltd.

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): 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): 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): 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): 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): 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): 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): 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): 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): Tagrisso (osimertinib)

Sponsor: AstraZeneca Pharmaceuticals LP

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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): Imfinzi (durvalumab)

Sponsor: AstraZeneca UK Ltd.

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): 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): 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): 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): 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): Empliciti (elotuzumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 5/19/2014

Approval Date: 11/30/2015

Indication: Patients with multiple myeloma who have received one to three prior therapies

Category: Cancer

Trade Name (Agent): Opdivo (nivolumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 9/16/2015

Approval Date: 11/23/2015

Indication: Patients with advanced renal cell carcinoma who have received prior antiangiogenic therapy

Category: Cancer

Trade Name (Agent): Darzalex (daratumumab)

Sponsor: Janssen Biotech Inc.

Date of BT Designation Disclosure: 5/01/2013

Approval Date: 11/16/2015

Indication: Patients with multiple myeloma who have received at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or are double refractory to a proteasome inhibitor and an immunomodulatory agent

Category: Cancer

Trade Name (Agent): Tagrisso (osimertinib) 

Sponsor: AstraZeneca Pharmaceuticals LP

Date of BT Designation Disclosure: 4/24/2014

Approval Date: 11/13/2015

Indication: Patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive-non-small-cell lung cancer (NSCLC), as detected by and FDA approved test who have progressed on or after EGFR TKI therapy

Category: Cancer

Trade Name (Agent): Strensiq (asfotase alfa)

Sponsor: Alexion Pharmaceuticals Inc.

Date of BT Designation Disclosure: 5/28/2013

Approval Date: 10/23/2015

Indication: Patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP)

Category: Rare Inherited Disorders

Trade Name (Agent): Praxbind (idarucizumab)

Sponsor: Boehringer Ingelheim Pharmaceuticals Inc.

Date of BT Designation Disclosure: 6/26/2014

Approval Date: 10/16/2015

Indication: Patients treated with Pradaxa when reversal of the anticoagulant effects of dabigatran is needed for emergency surgery/urgent procedures and in life-threatening or uncontrolled bleeding

Category: Other

Trade Name (Agent): Nuplazid (pimavanserin)

Sponsor: Acadia Pharmaceuticals Inc.

Date of BT Designation Disclosure: 9/02/2014

Approval Date: 4/29/2016

Indication: Patients with hallucinations and delusions associated with Parkinson's disease psychosis

Category: Other

Trade Name (Agent): Lenvima (lenvatinib mesylatel)

Sponsor: Eisai Inc.

Date of BT Designation Disclosure: 7/29/2015

Approval Date: 5/13/2016

Indication: Patients with renal cell carcinoma (RCC): in combination with evorlimus, for patients with advanced RCC following one prior anti-angiogenic therapy

Category: Cancer

Trade Name (Agent): Zepatier (grazoprevir and elbasvir)

Sponsor: Merck Sharp & Dohme Corp.

Date of BT Designation Disclosure: 4/08/2015

Approval Date: 1/28/2016

Indication: Adult patients with chronic hepatitis C virus (HCV) genotypes 1 and 4 infections

Category: Infectious Disease

Trade Name (Agent): Opdivo (nivolumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 4/25/2016

Approval Date: 11/10/2016

Indication: Patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum based therapy

Category: Cancer

Trade Name (Agent): Lartruvo (olaratumab)

Sponsor: Eli Lilly and Company

Date of BT Designation Disclosure: 6/08/2015

Approval Date: 10/19/2016

Indication: Patients with soft tissue sarcoma (STS) with a histologic subtype, in combination with doxorubicin, for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery

Category: Cancer

Trade Name (Agent): Tecentriq (atezolizumab)

Sponsor: Genentech Inc.

Date of BT Designation Disclosure: 5/31/2014

Approval Date: 10/18/2016

Indication: Patients with metastatic non-small cell lung cancer who have disease progression during or following platinum-containing chemotherapy

Category: Cancer

Trade Name (Agent): Orkambi (lumacaftor and ivacaftor)

Sponsor: Vertex Pharmaceuticals Inc.

Date of BT Designation Disclosure: 2016

Approval Date: 9/28/2016

Indication: Patients ages 6-11 with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene

Category: Rare Inherited Disorders

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 Familial Mediterranean Fever (FMF)

Category: Rare Inherited Disorders

Trade Name (Agent): Darzalex (daratumumab)

Sponsor: Janssen Biotech Inc.

Date of BT Designation Disclosure: 2016

Approval Date: 11/21/2016

Indication: Patients with multiple myeloma who have received at least one prior therapy, in combination with lenalidomide and dexamethasone

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 Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS)

Category: Rare Inherited Disorders

Trade Name (Agent): Epclusa (sofosbuvir and velpatasvir)

Sponsor: Gilead Sciences Inc.

Date of BT Designation Disclosure: 10/28/2015

Approval Date: 6/28/2016

Indication: Adult patients with chronic hepatitis C virus (HCV) genotypes 1,2,3,4,5, or 6 infection: -without cirrhosis or with compensated cirrhosis -with decompensated cirrhosis for use in combination with ribavirin

Category: Infectious Disease

Trade Name (Agent): Tecentriq (atezolizumab)

Sponsor: Genentech Inc.

Date of BT Designation Disclosure: 5/22/2014

Approval Date: 5/18/2016

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 adjuvant treatment with platinum-containing chemotherapy

Category: Cancer

Trade Name (Agent): Opdivo (nivolumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 5/14/2014

Approval Date: 5/17/2016

Indication: Patients with Hodgkin Lymphoma

Category: Cancer

Trade Name (Agent): Keytruda (pembrolizumab)

Sponsor: Merck Sharp & Dohme Corp.

Date of BT Designation Disclosure: 10/27/2014

Approval Date: 10/02/2015

Indication: Patients with metastatic, PD-L1 positive, non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy

Category: Cancer

Trade Name (Agent): Sovaldi (sofosbuvir)

Sponsor: Gilead Sciences Inc.

Date of BT Designation Disclosure: 10/25/2013

Approval Date: 12/06/2013

Indication: Patients with chronic hepatitis C infection

Category: Infectious Disease

Trade Name (Agent): Keytruda (pembrolizumab)

Sponsor: Merck Sharp & Dohme Corp

Date of BT Designation Disclosure: 2014

Approval Date: 9/04/2014

Indication: Patients with unresectable or metastatic melanoma & disease progression following ipilimumab and, if BRAF V600 mutation positibr, a BRAF inhibitor

Category: Cancer

Trade Name (Agent): Promacta (eltrombopag)

Sponsor: Novartis Pharmaceuticals Corp.

Date of BT Designation Disclosure: 2/03/2014

Approval Date: 8/26/2014

Indication: Cytopenias in patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy 

Category: Other

Trade Name (Agent): Imbruvica (ibrutinib)

Sponsor: Pharmacyclics LLC

Date of BT Designation Disclosure: 4/08/2013

Approval Date: 7/28/2014

Indication: Patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy and CLL with 17p deletion

Category: Cancer

Trade Name (Agent): Zydelig (idelalisib)

Sponsor: Gilead Sciences Inc.

Date of BT Designation Disclosure: 11/18/2013

Approval Date: 7/23/2014

Indication: Patients with relapsed chronic lymphocytic leukemia, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities

Category: Cancer

Trade Name (Agent): Arzerra (ofatumumab)

Sponsor: Novartis Pharmaceuticals Corp.

Date of BT Designation Disclosure: 9/13/2013

Approval Date: 4/17/2014

Indication: Previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate

Category: Cancer

Trade Name (Agent): Gazyva (obinutuzumab)

Sponsor: Genentech Inc.

Date of BT Designation Disclosure: 5/15/2013

Approval Date: 11/01/2013

Indication: Patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil

Category: Cancer

Trade Name (Agent): Braftovi (encorafenib)

Sponsor: Array BioPharma Inc.

Date of BT Designation Disclosure: 2019

Approval Date: 4/08/2020

Indication: In combination with cetuximab for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, detected by an FDA-approved test, after prior therapy

Category: Cancer

Trade Name (Agent): Exebacase

Sponsor: ContraFect Corporation

Date of BT Designation Disclosure: 2/24/2020

Indication: Patients with MRSA bacteremia, including right-sided endocarditis, when used in conjunction with standard of care antibiotics

Category: Infectious Disease

Trade Name (Agent): Abrocitinib

Sponsor: Pfizer Inc.

Date of BT Designation Disclosure: 2/15/2018

Indication: Patients with atopic dermatitis

Category: Other

Trade Name (Agent): Keytruda (pembrolizumab)

Sponsor: Merck Sharpe & Dohme Corp.

Date of BT Designation Disclosure: 7/15/2019

Indication: Patients with hepatocellular carcinoma

Category: Cancer

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 Pharmaceuticals Inc.

Date of BT Designation Disclosure: 7/16/2014

Approval Date: 10/15/2014

Indication: Patients with idiopathic pulmonary fibrosis (IPF)

Category: Other

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): 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): 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