FDA Breakthrough Therapy Designation List 2016-2017

FDA Approvals list

With new FDA Breakthrough Therapy Designations constantly being granted, we keep an up-to-date list of every approval. Check back for all the latest news and information.

(Last Updated: 2 February 2017)

30 January 2017 – uniQure, AMT-060

An FDA Breakthrough Therapy Designation has been granted to human gene therapy leaders uniQure for AMT-060, its investigatory gene therapy in patients with severe haemophilia B.

The designation is based on results from the ongoing, does-tanging Phase I-II study that show sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up.

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20 December 2016 – Juno Therapeutics / Celgene, JCAR017

An FDA Breakthrough Therapy Designation has been granted for Juno Therapeutics/ Celegine for their drug JCAR017 for the treatment for relapsed/ refractory (r/r) aggressive large B-cell NHL, including diffue large B-Cell lymphoma (DLBCL), not other specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-Cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma.

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19 December 2016- Tonix Pharmaceuticals, TNX-102 SL

Tonix Pharmaceuticals has announced that the FDA has granted a Breakthrough Therapy Designation to TNX-102 SL for the treatment of post traumatic stress disorder (PTSD).

The benefits of Breakthrough Therapy designation include the eligibility for priority review of the New Drug Application (NDA) within six months instead of ten months and rolling submission of portions of the NDA, in addition to an organisational commitment involving FDA’s senior managers contributing significant guidance.

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16 December 2016 – Grünenthal and Abiogen Pharma, Neridronic acid 

Grünenthal and Abiogen Pharma have been awarded a Breakthrough Therapy Designation for their Neridronic acid to treat complex regional pain syndrome (CRPS).

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11 October 2016 – Seattle Genetics, Adcetris (brentuximab vedotin) 

The BTD has been awarded to Seattle Genetics for the drug Adcetris for the treatment of patients with CD30-expressing mycosis fungoides (MF) and primary cutanieous anaplastic large cell lymphoma (pcALCL) who requite systemic therapy and have received one prior systemic therapy.

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10 October 2016 – Gamida Cell, Nicord 

Gamida Cell has received a BTD from the FDA for Nicord due to improvement in absorption of neutrophils blood cells in bone marrow transplant for patients with high risk hematological malignancies (blood cancers).

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5 October 2016 – Roche, Actemra/ RoActemra (tocilizumab) 

The second of the month for Roche grants Actemra / RoActemra a Breakthrough Designation therapy for the treatment of giant cell arteritis

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4 October 2016 – Roche, Alecensa (Alectinib)

Roche’s latest Breakthrough Therapy Designation has been granted for Alecensa to treat Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor.

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30 September 2016 – Abbvie ,Glecaprevir & Pebrentasvir

The FDA BTD comes for Abbvie’s Glecaprevir & Pibrentasvir. Rgimen for patients with Hepatitis C virus infection that have previously failed direct acting antiviral therapy.

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7 September 2016 – Merck, Pembrolizumab (Keytruda)

Merck’s new Breakthrough Therapy Designation comes for their new drug Pembrolizumab to treat first-line treatment of patients with advanced non-small cell lung cancer (NSCLC) whose tumours express PD-L1.

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6 September 2016 – Sage Therapeutics, SAGE-547 

Sage Therapeutics a clinical stage biopharmaceutical company developing novel medicines to treat life-altering central nervous system (CNS) disorders is granted a Breakthrough Therapy Designation for SAGE-547 for patients with Postpartum depression (PPD).

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23 August 2016 – Stemline Therapeutics, SL 401

Stemline Therapeutics have received a BTD for their drug SL 401 for the treatment of blasatic plamacytoid dendric cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123).

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16 August 2016 – Janssen, Esketamine

Esketamine Recieves Breakthrough Therapy Designation from U.S FDA Aministration for major despressive disorder with imminent risk for suicide, and for patients with treatment resistant depression.

Esketamine was first awarded a Breakthrough Therapy Designation in November 2013.

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3 August 2016 – Novartis, Ribociclib (LEE011)

Novartis have announced that they have been granted Breakthrough Therapy Designation for LEE011/Ribociclib in combination with letrozole for the treatment of hormone receptor positive, human epidermal growth factor receptor 2-negative (HR+/HER2-) advanced or metastatic breast cancer. LEE011 is a selective cyclin dependent kinase (CDK4/6) inhibitor.

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1 August 2016 – MEI Pharma, Pracinostat

MEI Pharma’s pracinostat receives breakthrough therapy designation from FDA for treatment in combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy.

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27 July 2016 – Johnson & Johnson, Genmab – Darzalex (Daratumumab)

Johnson & Johnson’s Darzalex has received its second FDA Breakthrough Therapy Designation for Darzalex in combination with lenalidomide (an immunomodulatory agent) and dexamethasone, or bortezomib (a proeasome inhibitor and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy.

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23 June 2016 – Incyte Corporation, Ruxolitinib (Jakafi®)

Incyte Corporation have been granted FDA Breakthrough Therapy Designation for the treatment of patients with acute graft-versus-host disease (GVHD). There are currently no approved treatments for patients with acute GVHD.

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13 June 2016 – BRIEF-Shire, SHP621 & SHP625

SHP621 & SHP625 are investigational products for rare gastrointestinal conditions. SHP621 for eosinophilic esophagitis and SHP625 for progressive familial intrahepatic cholestasis type 2.

“Receiving Breakthrough Therapy Designation on two pipeline products this past week reflects the potential of our strong and innovative pipeline of more than 60 programs,” said Flemming Ornskov, M.D., MPH, and CEO, Shire.

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31 May 2016 – Intercept,  Ocaliva (Obeticholic acid)

Ocaliva was approved by the FDA for treatment of primary biliary cholangitis, previously known as primary biliary cirrhosis (PBC), in combination with ursodeoxycholic (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

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16 May 2016 -Recominant Poliovirus Therapy

Duke’s Poliovirus therapy is an immunotherapy developed in the laboratory of Matthias Gromeier, a professor in the departments of Neurosurgery, Molecular Genetics and Microbiology, and Medicine at Duke University School of Medicine.

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27 April 2016 – Novartis, Ilaris (Canakunumab)

Novartis received three FDA Breakthrough Therapy Designations for Ilaris to treat rare types of Periodic Fever Syndromes (Hereditary Periodic Fevers).

Division Head of Novartis Pharmaceuticals David Epstein said: “This is an important day for patients, including many children, who are affected by these serious and debilitating syndromes that have no or limited treatment options.”

Ilaris is being reviewed for the treatment of Tumor Necrosis Factor-Receptor Associated periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS/Mevalonate Kinase Deficiency (MKD), and in Familial Mediterranean Fever (FMF) patients who are not adequately controlled with colchicine.

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25 April 2016 – Bristol-Myers Squibb (BMY), Opdivo (Nivolumab)

BMS has announced that the FDA has granted Breakthrough Therapy Designation to Opdivo for the potential indication of recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) after platinum based therapy.

The BTD was granted based on the results of CheckMate -141, a Phase III, open label, randomised trial evaluating Opdivo V’s investigator’s choice of therapy in patients with recurrent or metastatic SCCHN with tumor progression within six months of platinum therapies in the adjuvant, primary, recurrent or metastatic setting.

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18 April 2016 – Merck, Pembrolizumab

Pembrolizumab has been designated a breakthrough therapy for the treatment of relapsed or refractory classical Hodgkin lymphoma.

The BTD was based on the results and data from an ongoing phase Ib KEYNOTE-013 study and phase II KEYNOTE -087 study. Pembrolizumab has also received the designation for advanced melanoma, advanced non-small cell lung cancer and advanced colorectal cancer.

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16 March 2016 – Novimmune, NI-0501

Novimmune’s NI-0501 has been granted  FDA Breakthrough Therapy Designation for the treatment of patients with Primary Hemophagocytic Lymphohistiocytosis (HLH).

The Swiss biotech’s NI-0501 is the first treatment developed for HLH, and is a fully human, anti-interferon-gamma monoclonal antibody discovered and developed by Novimmune.

The FDA BTD was granted on the strength of clinical data from a Phase II study in children with primary HLH.

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22 February 2016  – Oliceridine (TRV130)

Trevena, Inc. has announced that the FDA have granted a Breakthrough Therapy Designation to intravenous Oliceridine (TRV130). The company’s lead product candidate is for the management of moderate-to-severe acute pain.

The product has gone through two successful Phase II studies and is now in Phase III development.

ATHENA-1 safety and tolerability study is still ongoing, with pivotal studies likely to begin in Q2 of 2016.

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19 February 2016 – Novartis PKC412

Novartis’ PKC412 (midostaurin) is an investigational treatment for adult patients with newly diagnosed AML (acute myeloid leukaemia) who are FLT3 mutation positive (as detected by an FDA approved test) plus being eligible to receive standard induction and consolidation chemotherapy.

The designation was based on the positive results from the Phase III RATIFY (CALGB 10603) clinical trial that was carried out in partnership with the Alliance for Clinical Trials in Oncology.

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 17 February 2016 – Ocrevus™ (Ocrelizumab)

Announced by Roche, Ocrevus™ was granted FDA Breakthrough Therapy Designation for the treatment of people with PPMS (Primary Progressive Multiple Sclerosis).

This designation is particularly important as there are currently no approved treatments for PPMS, a form of MS that is debilitating and is characterised by the progressively steady worsening of symptoms and is typically without distinct relapses or periods of remission.

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 17 February 2016 – Durvalumab (MEDI4736)

On the same day that Roche was granted designation for Ocrevus™ (Ocrelizumab), AstraZeneca and MedImmune announced that its global biologics and development arm had gained its own FDA Breakthrough Therapy Designation. It was awarded to Durvalumab (MED14736) for the treatment of patients with PD-L1 positive inoperable or metastatic urothelial bladder cancer whose tumour has progressed during or after one standard platinum-based regimen.

The investigatory human monoclonal antibody is directed against programmed death ligand-1 (PD-L1).

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 9 February 2016 – Affinity enhanced T-cell therapy targeting NY-ESO in synovial sarcoma for HLA-A*201, HLA-A*205 or HLA-A*206 allele-positive patients

Adaptimmune Therapeutics PLC announced the FDA had given BTD to their affinity enhanced T-Cell therapy targeting NY-ESO in synovial sarcoma for HLA-A* 201, HLA-A* 205 or HLA-A* 206 allele-positive patients. These patients will have inoperable metastatic synovial sarcoma, have received prior chemotherapy and have a tumour that expresses the NY-ESO-1 tumour antigen.

The Breakthrough Therapy Designation was won off the back of results of a phase I/II trial in patients with unresectable, metastatic or recurrent synovial sarcoma who have received prior chemotherapy.

The patients were treated with lymphodepleting chemotherapy followed by immunotherapy with T-Cells engineered to recognise an HLA-A2 restricted NY-ESO-1 peptide.

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8 February 2016- Sacituzumab Govitecan

Immunomedics investigational antibody-drug conjugate Sacituzumab Govitecan (IMMU-132) being given Breakthrough Therapy Designation will come as good news for patients with triple-negative breast cancer who have failed at least two prior therapies for metastatic disease.

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31 January 2016 – Rapastinel (GLYX-13)

Allergan’s Rapastinel (GLYX-13) BTD for adjunctive treatment of Major Depressive Disorder (MDD) follows the successful Fast Track Designation for the drug granted by the FDA in 2014.

Speaking about the designation, David Nicholson, Executive VP and President of Global R&D brands at Allergan said:

“Rapastinel is the first Allergan medicine to be granted Breakthrough Therapy designation by the FDA, underscoring our commitment to innovative research and development that addresses significant unmet medical needs. Breakthrough Therapy designation will allow us to work more closely with the FDA to bring this important therapy to patients as rapidly as possible.”

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 27 January 2016 – Lynparza™ (olaparib)

The first of AstraZenaca’s FDA Breakthrough Therapy Designations in 2016, the company announced that Lynparza™ (Olaparib) was approved on 27 January.

The oral poly ADP-ribose polymerase (PARP) inhibitor Lynparza™ (olaparib) is for the monotherapy treatment of BRCA1/2 or ATM gene mutated metastatic Castration Resistant Prostate Cancer (mCRPC), in patients who have received a prior taxane-based chemotherapy and at least one newer hormonal agent (abiraterone or enzalutamide).

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20 January 2016 – Venetoclax in combination with Riuximab

AbbVie’s Venetoclax in combination with Riuxiab is an inhibitor for the B-Cell lymphoma-2 (BCL-2) protein for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL). It was awarded Breakthrough Therapy Designation on 20 January, and has been developed by AbbVie in partnership with Genetech and Roche.

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 7 January 2016 – Setmelanotide

Rhythm, a biopharmaceutical company developing peptide therapeutics for rare genetic deficiencies that result in life-threatening metabolic disorders, announced on 7 January that the FDA granted Breakthrough Therapy designation to setmelanotide.

The company’s novel melanocortin-4 receptor (MC4R) agonist is used for the treatment of pro-opiomelanocortin (POMC) deficiency obesity. Setmelanotide is in Phase 2 clinical trials for the treatment of rare genetic disorders of obesity caused by MC4 pathway deficiencies.

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6 January 2016 – VTS-270

The first FDA Breakthrough Therapy Designation of 2016 was for Vtesse’s VTS-270. VTS-270 is a modified form of cyclodextrin that has been in pre-clinical and clinical Niemann-Pick Disease Type C (NPC) studies at a number of academic laboratories and also at the NIH.

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