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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 1148 total requests for the designation with 445** designations granted.
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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: 4/24/2013

Approval Date: 12/18/2015

Indication: Patients with unresectable or metastatic melanoma

Category: Cancer

Trade Name (Agent): Ibrance (palbociclib)

Sponsor: Pfizer Inc.

Date of BT Designation Disclosure: 2016

Approval Date: 2/19/2016

Indication: Patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy

Category: Cancer

Trade Name (Agent): Gavreto (Pralsentinib)

Sponsor: Blueprint Medicines Corporation

Date of BT Designation Disclosure: 2/26/2019

Approval Date: 9/04/2020

Indication: Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy

Category: Cancer

Trade Name (Agent): LN-145

Sponsor: Iovance Biotherapeutics, Inc.

Date of BT Designation Disclosure: 5/22/2019

Indication: Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy

Category: Cancer

Trade Name (Agent): Zolgensma (onasemnogene abeparvovec-xioi)

Sponsor: AveXis

Date of BT Designation Disclosure: 7/20/2016

Approval Date: 5/24/2019

Indication: Spinal muscular atrophy (SMA) Type 1 in children less than two years old

Category: Other

Trade Name (Agent): enfortumab vedotin

Sponsor: Astellas Pharma US, Inc. and Seattle Genetics, Inc.

Date of BT Designation Disclosure: 3/26/2018

Indication: Patients with locally advanced or metastatic urothelial carcinoma

Category: Cancer

Trade Name (Agent): LJPC-0118

Sponsor: La Jolla Pharmaceutical Company

Date of BT Designation Disclosure: 4/24/2019

Indication: Patients with severe malaria

Category: Infectious Disease

Trade Name (Agent): elafibranor

Sponsor: Genfit

Date of BT Designation Disclosure: 4/19/2019

Indication: Patients with primary biliary cholangitis (PBC)

Category: Other

Trade Name (Agent): SER-109

Sponsor: Seres Therapeutics, Inc.

Date of BT Designation Disclosure: 4/29/2019

Indication: Recurrent C. difficile infection

Category: Rare Inherited Disorders

Trade Name (Agent): PB2452

Sponsor: PhaseBio Pharmaceuticals

Date of BT Designation Disclosure: 4/08/2019

Indication: Reversal agent for the antiplatelet drug ticagrelor

Category: Other

Trade Name (Agent): AXS-05

Sponsor: Axsome Therapeutics, Inc.

Date of BT Designation Disclosure: 3/27/2019

Indication: Major Depressive Disorder (MDD)

Category: Rare Inherited Disorders

Trade Name (Agent): Oliceridine (TRV130)

Sponsor: Trevena, Inc.

Date of BT Designation Disclosure: 2/22/2016

Indication: Moderate-to-Severe Acute Pain

Category: Other

Trade Name (Agent): Aliqopa (copanlisib)

Sponsor: Bayer

Date of BT Designation Disclosure: 5/30/2019

Indication: Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies

Category: Cancer

Trade Name (Agent): Jakafi (Ruxolitinib)

Sponsor: Incyte

Date of BT Designation Disclosure: 6/23/2016

Approval Date: 5/28/2019

Indication: Patients aged 12 years and older with steroid-refractory acute graft versus-host disease (GVHD)

Category: Other

Trade Name (Agent): Givlaari (givosiran)

Sponsor: Alnylam

Date of BT Designation Disclosure: 5/30/2017

Approval Date: 11/20/2019

Indication: Adult patients with acute hepatic porphyria (AHP)

Category: Rare Inherited Disorders

Trade Name (Agent): Trikafta (elexacaftor/ivacaftor/tezacaftor)

Sponsor: Vertex Pharmaceuticals Inc.

Date of BT Designation Disclosure: 5/01/2018

Approval Date: 10/21/2019

Indication: Patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene

Category: Rare Inherited Disorders

Trade Name (Agent): Zejula (niraparib)

Sponsor: Janssen Pharmaceutica

Date of BT Designation Disclosure: 10/03/2019

Indication: Patients with BRCA1/2-mutated metastatic castration-resistant prostate cancer (CRPC) who have previously received taxane chemotherapy and androgen receptor (AR)–targeted therapy

Category: Cancer

Trade Name (Agent): Cotellic (cobimetinib)

Sponsor: Memorial Sloan Kettering Cancer Center

Date of BT Designation Disclosure: 10/03/2019

Indication: Adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation

Category: Rare Inherited Disorders

Trade Name (Agent): Tepotinib (Tepmetko)

Sponsor: Merck

Date of BT Designation Disclosure: 9/11/2019

Approval Date: 2/03/2021

Indication: Patients with metastatic non-small cell lung cancer (NSCLC) harboring a MET exon 14 skipping alteration who have progressed following platinum-based chemotherapy

Category: Cancer

Trade Name (Agent): Nirogacestat

Sponsor: SpringWorks Therapeutics, Inc.

Date of BT Designation Disclosure: 8/29/2019

Indication: Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis

Category: Cancer

Trade Name (Agent): NKTR-214 (bempegaldesleukin) with Opdivo (nivolumab)

Sponsor: Nektar Therapeutics

Date of BT Designation Disclosure: 8/23/2019

Indication: Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery

Category: Cancer

Trade Name (Agent): Peginterferon Lambda

Sponsor: Eiger Pharmaceuticals

Date of BT Designation Disclosure: 8/20/2019

Indication: Hepatitis delta virus (HDV) infection

Category: Infectious Disease

Trade Name (Agent): DCR-PHXC

Sponsor: Dicerna Pharmaceuticals

Date of BT Designation Disclosure: 7/15/2019

Indication: Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene)

Category: Other

Trade Name (Agent): Avexitide

Sponsor: Eiger BioPharmaceuticals, Inc.

Date of BT Designation Disclosure: 6/17/2019

Indication: Post-bariatric hypoglycemia (PBH)

Category: Other

Trade Name (Agent): seladelpar

Sponsor: CymaBay Therapeutics

Date of BT Designation Disclosure: 2/20/2019

Indication: Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA)

Category: Other

Trade Name (Agent): Braftovi (encorafenib) + Mektovi (binimetinib) + Erbitux (cetuximab)

Sponsor: Array BioPharma and Eli Lilly and Co.

Date of BT Designation Disclosure: 8/07/2018

Indication: Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting

Category: Cancer

Trade Name (Agent): Rezurock (belumosudil)

Sponsor: Kadmon Holdings, Inc.

Date of BT Designation Disclosure: 10/17/2018

Approval Date: 7/16/2021

Indication: Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy

Category: Other

Trade Name (Agent): Hemlibra (emicizumab-kxwh)

Sponsor: Genentech Inc.

Date of BT Designation Disclosure: 4/17/2018

Approval Date: 10/04/2018

Indication: Patients with hemophilia A without factor VIII inhibitors

Category: Rare Inherited Disorders

Trade Name (Agent): Rubraca (rucaparib)

Sponsor: Clovis Oncology

Date of BT Designation Disclosure: 10/03/2018

Indication: BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer

Category: Cancer

Trade Name (Agent): Luxturna (voretigene neparvovec-rzyl)

Sponsor: Spark Therapeutics Inc.

Date of BT Designation Disclosure: 9/24/2014

Approval Date: 12/19/2017

Indication: Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy

Category: Rare Inherited Disorders

Trade Name (Agent): Prevnar 20 (PF-06482077)

Sponsor: Pfizer, Inc.

Date of BT Designation Disclosure: 9/20/2018

Approval Date: 6/08/2021

Indication: Prevention of pneumonia and invasive disease caused by Streptococcus pneumoniae serotypes

Category: Infectious Disease

Trade Name (Agent): CA-008

Sponsor: Concentric Analgesics, Inc.

Date of BT Designation Disclosure: 9/11/2018

Indication: Patients needing long-lasting, non-opioid pain relief

Category: Other

Trade Name (Agent): Tezepelumab

Sponsor: AstraZeneca Plc & Amgen Inc.

Date of BT Designation Disclosure: 9/07/2018

Indication: Patients with severe asthma, without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers

Category: Other

Trade Name (Agent): PF-06651600

Sponsor: Pfizer Inc.

Date of BT Designation Disclosure: 9/06/2018

Indication: Patients with alopecia areata

Category: Other

Trade Name (Agent): LOXO-292

Sponsor: Loxo Oncology, Inc.

Date of BT Designation Disclosure: 9/05/2018

Indication: Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer

Category: Cancer

Trade Name (Agent): Danyelza (naxitamab-gqgk)

Sponsor: Y-mAbs Therapeutics, Inc.

Date of BT Designation Disclosure: 8/23/2018

Approval Date: 11/25/2020

Indication: In combination with GM-CSF for patients with high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow

Category: Cancer

Trade Name (Agent): Poteligeo (mogamulizumab-kpkc)

Sponsor: Kyowa Kirin, Inc.

Date of BT Designation Disclosure: 10/25/2017

Approval Date: 8/08/2018

Indication: Adult patients Sézary syndrome (SS) after at least one prior systemic therapy

Category: Cancer

Trade Name (Agent): LOXO-292

Sponsor: Loxo Oncology, Inc.

Date of BT Designation Disclosure: 10/16/2018

Indication: Patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment, and have no acceptable alternative treatment options

Category: Cancer

Trade Name (Agent): Myrcludex

Sponsor: MYR Pharma

Date of BT Designation Disclosure: 10/23/2018

Indication: Chronic hepatitis delta (HDV) infection

Category: Infectious Disease

Trade Name (Agent): Psilocybin therapy

Sponsor: COMPASS Pathways

Date of BT Designation Disclosure: 10/23/2018

Indication: Treatment-resistant Depression

Category: Other

Trade Name (Agent): AT-GAA (ATB200 + AT2221)

Sponsor: Amicus Therapeutics

Date of BT Designation Disclosure: 2/26/2019

Indication: Patients with late onset Pompe disease

Category: Rare Inherited Disorders

Trade Name (Agent): CP101

Sponsor: Finch Therapeutics Group, Inc.

Date of BT Designation Disclosure: 2/09/2019

Indication: Patients with recurrent Clostridium difficile infection

Category: Infectious Disease

Trade Name (Agent): UKONIQ (umbralisib)

Sponsor: TG Therapeutics

Date of BT Designation Disclosure: 1/28/2019

Approval Date: 2/05/2021

Indication: Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy

Category: Cancer

Trade Name (Agent): Vaxneuvance (V114)

Sponsor: Merck

Date of BT Designation Disclosure: 1/28/2019

Approval Date: 7/19/2021

Indication: Prevention of invasive pneumococcal disease in adults 18 years and older caused by 15 serotypes.

Category: Infectious Disease

Trade Name (Agent): MEDI8897

Sponsor: AstraZeneca

Date of BT Designation Disclosure: 2/06/2019

Indication: Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV)

Category: Other

Trade Name (Agent): lonafarnib

Sponsor: Eiger BioPharmaceuticals

Date of BT Designation Disclosure: 11/19/2018

Approval Date: 11/20/2020

Indication: Patients with Hutchinson-Gilford progeria syndrome (HGPS_

Category: Rare Inherited Disorders

Trade Name (Agent): Olipudase Alfa

Sponsor: Sanofi/Genzyme

Date of BT Designation Disclosure: 11/29/2018

Indication: Niemann-Pick disease Type B (NPB)

Category: Rare Inherited Disorders

Trade Name (Agent): Andexxa (andexanet alfa)

Sponsor: Portola Pharmaceuticals, Inc.

Date of BT Designation Disclosure: 11/25/2013

Approval Date: 5/03/2018

Indication: patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding

Category: Cardiovascular

Trade Name (Agent): Xolair (omalizumab)

Sponsor: Genentech, Inc.

Date of BT Designation Disclosure: 8/13/2018

Approval Date: 9/28/2018

Indication: Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies

Category: Other

Trade Name (Agent): NGX-101

Sponsor: NeuroRx

Date of BT Designation Disclosure: 11/13/2018

Indication: Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy

Category: Other

Trade Name (Agent): Promacta (eltrombopag)

Sponsor: Novartis Pharmaceuticals Corp.

Date of BT Designation Disclosure: 11/16/2018

Indication: Low platelet counts in people exposed to radiation

Category: Other

Trade Name (Agent): Breyanzi (Lisocabtagene maraleucel)

Sponsor: Juno Therapeutics/Celgene

Date of BT Designation Disclosure: 12/20/2016

Approval Date: 2/05/2021

Indication: Relapsed/refractory (r/r) aggressive large B-cell NHL, including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma

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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): 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): Enhertu (fam-trastuzumab deruxtecan-nxki)

Sponsor: Daiichi Sankyo

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): 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): 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): Tibosovo (ivosidenib) and azacitidine

Sponsor: Agios Pharmaceuticals

Date of BT Designation Disclosure: 3/27/2019

Indication: newly diagnosed patients with IDH1-mutant acute myeloid leukemia (AML) ≥75 years old or are ineligible for intensive induction chemotherapy

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): 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): Calquence (acalabrutinib)

Sponsor: AstraZeneca UK Ltd

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): Calquence (acalabrutinib)

Sponsor: AstraZeneca UK Ltd

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): 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): 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): Zulresso (Brexanolone)

Sponsor: Sage Therapeutics Inc.

Date of BT Designation Disclosure: 9/06/2016

Approval Date: 3/19/2019

Indication: Postpartum depression (PPD)

Category: Other

Trade Name (Agent): Venclexta (venetoclax)

Sponsor: Abbvie Inc.

Date of BT Designation Disclosure: 10/25/2017

Approval Date: 11/21/2018

Indication: In combination with azacitidine, decitabine, or low-dose cytarabine for adults aged 75 and older with treatment-naive acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy

Category: Cancer

Trade Name (Agent): Oxervate (cenegermin-bkbj ophthalmic solution)

Sponsor: Dompe Farmaceutici SpA

Date of BT Designation Disclosure: 11/30/2017

Approval Date: 8/22/2018

Indication: Patients with neurotrophic keratitis (NK)

Category: Other

Trade Name (Agent): Onpattro (patisiran)

Sponsor: Alnylam Pharmaceuticals Inc.

Date of BT Designation Disclosure: 2/02/2018

Approval Date: 8/10/2018

Indication: Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults

Category: Rare Inherited Disorders

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

Sponsor: Vertex Pharmaceuticals Inc.

Date of BT Designation Disclosure: 3/12/2018

Approval Date: 8/07/2018

Indication: Treatment of cystic fibrosis (CF) in patients 2 years and older, homozygous for the F508del-CFTR mutation in the CFTR gene

Category: Rare Inherited Disorders

Trade Name (Agent): Poteligeo (mogamulizumab-kpkc)

Sponsor: Kyowa Kirin, Inc.

Date of BT Designation Disclosure: 8/25/2017

Approval Date: 8/08/2018

Indication: Adult patients with relapsed or refractory mycosis fungoides (MF) or Sezary syndrome after at least one prior systemic therapy

Category: Cancer

Trade Name (Agent): Azedra (iobenguane I 131)

Sponsor: Progenics Pharmaceuticals Inc.

Date of BT Designation Disclosure: 7/28/2015

Approval Date: 7/30/2018

Indication: Adult and pediatric patients (12 years and older) with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy

Category: Cancer

Trade Name (Agent): Krintafel (tafenoquine)

Sponsor: GlaxoSmithKline Intellectual Property Development LTD England

Date of BT Designation Disclosure: 12/18/2013

Approval Date: 7/20/2018

Indication: For the radical cure (prevention of relapse) of plasmodium vivax malaria in patients aged 16 years and older who are receiving appropriate antimalarial therapy for acute P. vivax

Category: Infectious Disease

Trade Name (Agent): Kisqali (ribociclib)

Sponsor: Novartis Pharmaceuticals Corp.

Date of BT Designation Disclosure: 6/20/2018

Approval Date: 7/18/2018

Indication: In combination with an aromatase inhibitor for pre/perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy

Category: Cancer

Trade Name (Agent): Yervoy (ipilimumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 2/10/2018

Approval Date: 7/10/2018

Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan

Category: Cancer

Trade Name (Agent): Opdivo (nivolumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 2/10/2018

Approval Date: 6/10/2018

Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan

Category: Cancer

Trade Name (Agent): Keytruda (pembrolizumab)

Sponsor: Merck Sharp & Dohme Corp.

Date of BT Designation Disclosure: 2017

Approval Date: 6/13/2018

Indication: Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy

Category: Cancer

Trade Name (Agent): Takhzyro (lanadelumab-flyo)

Sponsor: Dyax Corp.

Date of BT Designation Disclosure: 7/02/2015

Approval Date: 8/23/2018

Indication: Prevention of attacks of hereditary angioedema (HAE) in people 12 years of age and older

Category: Rare Inherited Disorders

Trade Name (Agent): Arikayce (amikacin liposome inhalation suspension)

Sponsor: Insmed Inc.

Date of BT Designation Disclosure: 6/17/2014

Approval Date: 9/28/2018

Indication: Treatment in a limited population of patients with Mycobacterium avium complex (MAC) lung disease who do not respond to conventional treatment

Category: Infectious Disease

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): 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): 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): 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): 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