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1
SGEN
Cancer - ALCANZA trial for relapsed CD30-positive cutaneous T-cell lymphoma
Approved
2017-11-09 00:00:00
Approval announced November 9, 2017.
1
TGTX
Chronic Lymphocytic Leukemia (CLL) and small lymphocytic lymphoma (SLL)
Phase 3
2021-09-20 00:00:00
Phase 3 results showed that 421 patients were randomized to the U2 (n=210) or O+Chl (n=211) arms; 57% of patients were treatment-naïve and 43% had relapsed/refractory (R/R) CLL. At a median follow-up of 36.7 months, U2 significantly prolonged independent review committee (IRC) assessed progression-free survival (PFS) vs O+Chl (median 31.9 months vs 17.9 months; hazard ratio 0.546 (p<0.0001)). September 20, 2021
1
GMAB
Non-Hodgkin Lymphoma (NHL)
Phase 1/2
2021-12-14 00:00:00
Phase 1/2 data reported that 25% of patients reported Grade 4 Neutropenia, with a 73% CMR and a 27% PMR, noted December 14, 2021.
0
AGIO
Sickle cell disease
Phase 2
2023-06-26 00:00:00
Phase 2 portion met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms, noted June 26, 2023
0
HRTX
Postoperative nausea and vomiting (PONV)
Approved
2022-09-16 00:00:00
Approved September 16, 2022.
1
ZYME
Gastric/gastroesophageal junction adenocarcinoma (G/GEJC)
Phase 1/2
2023-10-20 00:00:00
Phase 1b/2 data presented at ESMO reported that the confirmed objective response rate by investigator (INV) was 75.8% and median progression-free survival was 16.7 months, noted October 20, 2023.
1
VIR
Mild to moderate COVID-19
Phase 3
2021-03-03 00:00:00
Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.
0
ABBV
Biosimilar candidate to Avastin (bevacizumab)
Approved
2017-09-14 00:00:00
Approved September 14, 2017.
1
MRK
Type 2 diabetes
Approved
2017-12-20 00:00:00
Approval announced December 20, 2017.
1
AVXL
Parkinson’s Disease Dementia (PDD)
Phase 2
2023-03-30 00:00:00
Phase 2 study data met primary and key secondary endpoints, noted March 30, 2023.
0
IMVIQ
Metastatic Bladder and Micro-Satellite Instability High (MSI-H) Solid Tumors
Phase 2
2019-09-30 00:00:00
Phase 2 data at ESMO 2019 noted 2/23 PRs.
0
CTMX
Solid tumors / Diffuse large B-cell lymphoma (DLBCL), squamous non-small cell lung cancer (sqNSCLC), head and neck squamous cell carcinoma, and esophageal/gastro-esophageal junction (E/GEJ) cancer
Phase 2
2021-12-20 00:00:00
Phase 2 initial data reported an objective response rate of 18.8% and disease control rate of 87.5% in squamous non-small cell lung cancer. In advanced head and neck squamous cell carcinoma with objective response rate of 4.0 percent and disease control rate of 56.0 percent, noted December 20, 2021.
0
LEGN
Multiple Myeloma
Phase 3
2023-06-05 00:00:00
Phase 3 results shared at ASCO demonstrated a statistically significant improvement in progression-free survival, with a hazard ratio of 0.26, noted June 5, 2023
1
VRDN
Active Thyroid Eye Disease (TED)
Phase 1/2
2023-07-10 00:00:00
Phase 1/2 preliminary data demonstrated clinically meaningful and rapid improvement in signs and symptoms of chronic TED at week 6 after receiving two infusions of VRDN-001 10 mg/kg or 3 mg/kg, noted July 10, 2023.
1
TNXP
Fibromyalgia
Phase 3
2023-06-15 00:00:00
Phase 3 met the primary endpoint on its RELIEF trial, noted June 15, 2023
0
REGN
Non-small cell lung cancer (NSCLC)
Phase 2
2023-10-31 00:00:00
Phase 2 preliminary efficacy data reported a 100% Disease Control Rate (DCR), noted October 31, 2023.
0
GNPX
Non-small cell lung cancer (NSCLC)
Phase 1
2023-06-03 00:00:00
Phase 1 data shared at ASCO reported that treatment was well tolerated, and there were no dose limiting toxicities, noted June 3, 2023.
1
MRK
Hospital-Acquired and Ventilator-Associated Bacterial Pneumonia (HABP/VABP)
Approved
2020-06-04 00:00:00
FDA approval announced June 4, 2020.
1
SNY
Eosinophilic esophagitis (EoE) in 1-11 year olds
Phase 3
2022-07-14 00:00:00
Phase 3 data reported that trial met its primary endpoint, noted July 14, 2022.
0
PCSA
Gastroparesis
Phase 2a
2023-05-06 00:00:00
Phase 2a data reported at DDW noted that treatment improved both gastroparesis symptom scores and gastric emptying rate, noted May 6, 2023.
1
NWBO
Glioblastoma multiforme (GBM)
Phase 3
2022-11-17 00:00:00
Phase 3 data reported that both median survival and "Long Tail" of extended survival were increased in both newly diagnosed and recurrent glioblastoma, noted November 17, 2022.
0
PTCT
Huntington's disease
Phase 2
2023-06-28 00:00:00
Phase 2 trial initiated March 30, 2022. Phase 2 trial data reported that treatment was well tolerated, with no treatment related serious adverse events and no adverse events leading to discontinuation, noted June 28, 2023.
1
DBVT
Peanut allergy - ages 1 to 3 years
Phase 3
2023-02-24 00:00:00
Phase 3 data due presented at AAAAI reported that data demonstrate that treatment with Viaskin Peanut 250 μg for up to 36 months in peanut-allergic children was generally safe and well tolerated, noted February 24, 2023.
1
IMUX
COVID-19
Phase 2
2021-02-17 00:00:00
Phase 2 top-line data released February 17, 2021 - low rates of COVID-19 prevented the primary endpoint from being evaluable.
1
MRK
Resectable Stage II, IIIA or IIIB Non-Small Cell Lung Cancer (NSCLC)
Approved
2023-10-16 00:00:00
Approved on October 16, 2023.
1
FBIO
Postoperative pain following bunionectomy surgery
CRL
2022-02-15 00:00:00
FDA AdCom Meeting resulted in a 8-14 vote against the question that the application showed that benefits outweighs the risks, noted February 15, 2022.
1
AEZS
Endometrial cancer
Phase 3
2017-05-01 00:00:00
Phase 3 trial did not meet primary endpoint - May 1, 2017.
1
AMGN
Biosimilar candidate to Avastin (bevacizumab)
Approved
2017-09-14 00:00:00
Approved September 14, 2017.
1
ALGS
Nonalcoholic steatohepatitis (NASH)
Phase 1
2023-03-29 00:00:00
Phase 1 data presented at the Hepatology Conference demonstrated a favorable safety and pharmacokinetic profile in healthy volunteers and subjects with hyperlipidemia, March 29, 2023.
1
ENTA
COVID-19
Phase 2
2023-05-08 00:00:00
Phase 2 trial met primary endpoint, noted May 8, 2023.
0
TXMD
Contraception
Approved
2018-08-10 00:00:00
FDA Approval announced August 10, 2018.
1
QURE
Huntington's disease
Phase 1/2
2023-06-21 00:00:00
Phase 1/2 early clinical data demonstrate trends consistent with a potential clinical benefit of AMT-130 at both doses, noted June 21, 2023.
1
FRLN
Gaucher disease
Phase 1/2
2023-10-25 00:00:00
Phase 1/2 results shared at the ESGCT reported no infusion reactions and no serious adverse events, noted October 25, 2023.
0
BPTH
Solid tumors
Phase 1
2023-07-17 00:00:00
Phase 1/1b first cohort completed, noted July 17, 2023
0
RHHBY
Castration-resistant prostate cancer
Phase 3
2020-06-19 00:00:00
Phase 3 data met one of two co-primary endpoints - June 19, 2020.
0
BGNE
Squamous non-small cell lung cancer (NSCLC)
Phase 3
2020-01-21 00:00:00
Phase 3 trial met primary endpoint of PFS at interim analyses.
0
KNSA
Chronic Pruritus
Phase 2
2020-05-11 00:00:00
Phase 2 interim data released May 11, 2020.
0
MRK
Facioscapulohumeral muscular dystrophy
Phase 2
2019-09-16 00:00:00
Phase 2 data did not achieve statistically significant improvements in functional endpoints relative to placebo - September 16, 2019.
1
MLYS
Hypertension
Phase 2
2023-11-02 00:00:00
Phase 2 data to be presented ASN further supports obesity-associated dysregulated aldosterone as an endotype predictive of enhanced response, noted November 2, 2023.
1
FDMT
X-linked retinitis pigmentosa (XLRP)
Phase 1/2
2021-10-10 00:00:00
Phase 1/2 initial results showed that dosing was well tolerated in all patients treated to-date, with no dose-limiting toxicities, no serious adverse events and no chronic inflammation, noted October 10, 2021
1
KPTI
Quadruple Refractory Multiple Myeloma
Approved
2019-07-03 00:00:00
FDA Approval announced July 3, 2019. Continued approval may be contingent upon data from its Phase 3 Boston trial (data due late-2019/early 2020).
1
DNLI
Healthy Volunteers
Phase 1
2021-10-06 00:00:00
Phase 1 data noted that treatment was well tolerated for up to 14 days of dosing, with robust distribution in the central nervous system (CNS) and predictable dose-related increases in exposure with a PK profile supporting once daily dosing, noted October 6, 2021.
1
BGNE
Refractory chronic lymphocytic leukemia (CLL)
Phase 3
2023-06-15 00:00:00
Phase 3 results presnted at ICML In a subgroup analysis showed improved PFS over ibrutinib in patients in China, consistent with the global study population, noted June 15, 2023.
1
PALI
Reduction in adhesions following abdominal and pelvic surgery
Phase 2
2023-08-09 00:00:00
Phase 2 study did not achieve primary efficacy endpoint of reducing adhesions in LB1148 treated patients compared to placebo treated patients post bowel resection surgery, noted August 9, 2023.
1
NCNA
Pancreatic cancer
Phase 3
2019-08-20 00:00:00
Phase 3 enrolment suspended due to futility - August 20, 2019.
0
INCY
Polycythemia Vera
Approved
2014-12-04 00:00:00
Approved December 4, 2014.
1
NBIX
Essential Tremor
Phase 2
2022-08-04 00:00:00
Phase 2 trial did not meet primary endpoints, noted August 4, 2022.
1
PFE
Ulcerative colitis
Phase 3
2022-03-29 00:00:00
Phase 3 data met the co-primary endpoints of clinical remission at both weeks 12 and 52 and all key secondary endpoints, noted March 29, 2022.
0
MRK
Triple negative breast cancer (TNBC)
Approved
2021-07-27 00:00:00
FDA approval announced July 27, 2021.
1
GILD
Pre-exposure prophylaxis
Approved
2019-10-03 00:00:00
FDA Approval announced October 3, 2019.
1
DCPH
Gastrointestinal Stromal Tumors (GIST) - fourth-line
Approved
2020-05-15 00:00:00
FDA Approval announced May 15, 2020.
1
NVIV
Complete Thoracic AIS A Spinal Cord Injury
Phase 3
2023-03-09 00:00:00
Pivotal trial did not meet the primary endpoint, noted March 9, 2023.
1
VERA
Kidney transplant
Phase 2
2022-06-04 00:00:00
Phase 2 data results showed that the antiviral effect was higher in the dosed group than the placebo group. Further, the treatment was well tolerated, with a comparable frequency of adverse events and serious adverse events between groups
1
MRK
First-Line Nonsquamous Non-small cell lung cancer (NSCLC)
Approved
2018-06-05 00:00:00
FDA approval (label expansion) announced June 5, 2018.
1
EGRX
Malignant hyperthermia - cancer
Approved
2014-07-23 00:00:00
Approved July 23, 2014 under priority review.
1
PFE
Gastric cancer
Phase 3
2019-11-08 00:00:00
Phase 3 endpoint not met - November 8, 2019.
0
INDP
Advanced/metastatic solid tumors
Phase 1
2023-10-31 00:00:00
Phase 1 data abstract to be presented at SITC on November 4, 2023 noted that each of the first cohort participants experienced transient activation of biomarkers associated with innate and/or adaptive immune responses, and generally expected transient adverse events, noted October 31, 2024.
1
ACXP
C. difficile Infection
Phase 2b
2023-11-02 00:00:00
Phase 2a and 2b data reported an observed Clinical Cure rate in the combined Phase 2 trials in patients with CDI was 96%, noted November 2, 2023.
1
SAGE
Postpartum Depression - moderate
Approved
2019-03-19 00:00:00
FDA approval announced March 19, 2018.
1
TCRX
Hematologic malignancies
Phase 1
2023-11-02 00:00:00
Phase 1 data from abstract reported that the safety analysis found expected post-HCT adverse events similar in treatment and control arms. Incidence of graft-versus-host disease (GvHD) was similar in control (3 events) and treatment arms, noted November 2, 2023.
1
NVS
Wet macular degeneration
Phase 3
2023-08-15 00:00:00
Phase 3 trial met its primary endpoint, noted August 15, 2023.
0
ABBV
Follicular lymphoma (FL)
Phase 1/2
2023-06-28 00:00:00
Phase 1/2 FL cohort data reported an overall response rate (ORR) of 82% and that the median duration of response (DOR) was not reached, noted June 28, 2023.
1
ADMP
Emergency treatment of anaphylaxis
Approved
2017-06-15 00:00:00
CRL issued March 27 2015. CRL issued again June 6, 2016. Approval announced June 15, 2017 following third submission.
1
TALS
COVID-19 in living donor kidney transplant (LDKT) patients
Phase 2
2022-06-07 00:00:00
Additional Phase 2 data presented at ATC reported that none of these 18 patients lost chimerism as a result of their COVID-19 vaccination, noted June 7, 2022.
1
FWBI
COVID-19-related gastrointestinal (GI) infections
Phase 2
2022-04-29 00:00:00
Phase 2 top-line data reported that trial did not meet its primary endpoint, noted April 29, 2022.
1
ALXO
Acute myeloid leukemia (AML)
Phase 1
2022-12-12 00:00:00
Additional Phase 1 data reported that treatment is well-tolerated with 4 patients achieving a response, noted December 12, 2022.
1
ATHX
Acute Respiratory Distress Syndrome
Phase 1/2
2022-02-02 00:00:00
Phase 1/2 data reported a median 18.5 ventilator-free days compared to 6.5 days for placebo. 25% of the treatment group reported mortality compared to 40% in placebo, noted February 2, 2022.
1
VNDA
Jet Lag Disorder
CRL
2019-08-19 00:00:00
CRL issued August 19, 2019.
0
TFFP
Invasive Pulmonary Aspergillosis (IPA)
Phase 2
2022-11-01 00:00:00
Phase 2 trial data showed that second patient treated to clinically benefit from administration in compassionate use program, noted November 1, 2022.
1
INVA
Chronic obstructive pulmonary disease (COPD)
Approved
2017-09-18 00:00:00
Approval announced September 18, 2017.
1
ICPT
NASH patients with compensated cirrhosis
Phase 3
2023-08-02 00:00:00
NASH Programs discontinued, noted August 2, 2023.
-1
NVTA
Early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE)
Phase 3
2023-10-02 00:00:00
Phase 3 results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients, noted October 2, 2023.
1
ARVN
ER+ positive / HER2- negative breast cancer
Phase 1b
2021-12-13 00:00:00
Phase 1 dose escalation data reported that treatment was well tolerated with no DLTs at total daily doses up to 700mg, noted December 13, 2021.
1
ABBV
B-cell Lymphoma (DLBCL)
Approved
2023-05-22 00:00:00
Approved May 19, 2023.
1
GILD
Hepatitis B virus (HBV) infection in pediatric patients 12 years of age and older
Approved
2022-11-02 00:00:00
Approved November 2, 2022.
1
AZN
Metastatic non-small cell lung cancer (NSCLC)
Phase 1b
2023-06-05 00:00:00
Phase 1b presented at ASCO demonstrated objective response rates of 57% and 50%, respectively, with a disease control rate of 91% across cohorts, noted June 6, 2023
1
BMY
Solid tumors
Phase 1
2021-10-07 00:00:00
Phase 1 updated data noted a confirmed responses for a cORR of 36% out of the 11 MET TKI-naïve NSCLC patients, cORR of 33% for the nine MET TKI-naïve GC/GEJ patients, and among the 12 patients with advanced other solid tumors, one patient with MET amplified colorectal cancer achieved a confirmed response, noted October 7, 2021.
1
ITOS
Solid tumors
Phase 1/2
2021-06-04 00:00:00
Phase 1/2 single agent data at ASCO June 4, 2021 noted 5/21 patients with stable disease in including two partial responses.
0
PTGX
Crohn's disease
Phase 2
2021-11-03 00:00:00
Phase 2 trial and candidate development has been stopped, noted November 3, 2021.
-1
VSTM
Low-grade serous ovarian cancer (LGSOC)
Phase 2
2023-05-25 00:00:00
Additional Phase 2 data from abstract reported a confirmed objective response rates (ORR) by blinded independent central review of 45%, noted May 25, 2023.
0
TAK
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
NDA Filing
2023-06-20 00:00:00
Phase 3 full result findings showed a clinically significant reduction in relapse rate as well as a delayed time to relapse compared to placebo when used as a maintenance therapy, noted June 20, 2023. NDA under current review by the FDA.
1
SNPX
Alzheimer's disease
Phase 2b
2023-09-09 00:00:00
Phase 2b data presented at IBRO reported that nearly all pre-specified secondary endpoints were achieved with statistical significance in the server cohort, noted September 9, 2023.
1
AZN
Monotherapy for expanded use in women with HR+, HER2- advanced breast cancer
Approved
2017-08-28 00:00:00
Expanded approval announced August 28, 2017.
1
BXRX
Acute pain following bunionectomy surgery
Approved
2020-02-20 00:00:00
FDA Approval announced February 20, 2020.
1
ATAI
Treatment Resistant Depression (TRD)
Phase 2b
2021-11-15 00:00:00
Phase 2b trial met primary endpoint noted November 2021.
0
REGN
Stage II to IV resectable cutaneous squamous cell carcinoma (CSCC)
Phase 2
2023-10-23 00:00:00
Phase 2 data from ESMO 23 reported a 89% event-free survival rate at one year, and no disease recurrence among patients who achieved a pCR (n=40), noted October 23, 2023.
1
ABBV
Multiple Myeloma
Phase 1/2
2021-12-13 00:00:00
Phase 1/2 safety and preliminary efficacy data reported that treatment of patients with t(11;14) RRMM using VenDd and those with RRMM with VenDvd demonstrated a tolerable safety profile and an overall response rate (ORR) of 95.8% and 91.7%, respectively, noted December 13, 2021.
1
ALNY
Hypercholesterolemia
Approved
2021-12-22 00:00:00
Approved December 22, 2021.
1
GSK
Ovarian cancer
Approved
2019-10-23 00:00:00
FDA Approval announced October 23, 2019.
1
HRMY
Excessive daytime sleepiness associated with Prader-Willi Syndrome (PWS)
Phase 2
2023-07-20 00:00:00
EoP2 meeting completed, noted July 20, 2023
1
MEIP
Relapsed/refractory Follicular Lymphoma (FL) Non Hodgkin Lymphoma
Phase 2
2022-06-04 00:00:00
Phase 2 top-line data presented at ASCO 22 reported a 70.3% objective response rate and that 35.2% achieved a complete response. 87.5% of responses achieved in first two cycles of therapy, 75% of CRs in first four cycles, 9.9% of patients discontinued the trial due to drug related adverse events, follow-up time for response was 8.4 months, and 83% reported grade 3 AE of special interest in cycles 1-3.
1
KROS
Pulmonary arterial hypertension (PAH) and disorders associated with bone loss
Phase 2
2023-05-22 00:00:00
Phase 2 biomarker reported that treatment led to changes in serum proteins involved in structural remodeling pathways and inflammation consistent with that seen in preclinical studies, noted May 22, 2023.
1
STOK
Dravet Syndrome
Phase 1/2
2023-07-25 00:00:00
Phase 1/2a new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001. The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population, noted July 25, 2024
1
GRTS
Colorectal Cancer / Gastric Cancer
Phase 1/2
2021-09-17 00:00:00
Phase 1/2 data showed a 44% molecular response rate (4/9) and extended median overall survival >17 months, September 17, 2021.
1
CAPR
COVID-19
Phase 2
2022-03-28 00:00:00
Phase 2 met safety primary endpoint, with an overall mortality of 20% and 5 deaths in the treatment group, noted March 28, 2022.
1
LUMO
Pediatric Growth Hormone Deficiency (PGHD)
Phase 2
2023-06-21 00:00:00
Phase 2 interim data shared at KOL demonstrate a durable response to LUM-201 from 6 to 12 months, noted June 21, 2023.
1
IMVT
Autoimmune diseases
Phase 1
2023-09-26 00:00:00
Phase 1 data reported no dose-related changes in serum albumin or LDL-C, and the mean total IgG reduction from baseline in this MAD cohort was 63%, noted September 26, 2023.
1
ZYNE
Fragile X syndrome
Phase 2/3
2022-05-13 00:00:00
Phase 2/3 long-term data reported that sustained improvement in ABC-CFXS Social Avoidance from baseline, noted May 13, 2022.
1
ETON
Methanol poisoning
CRL
2023-06-28 00:00:00
CRL Announced on June 28, 2023.
0
BMY
COVID-19
Phase 3
2022-06-02 00:00:00
Phase 3 data reported that the risk of death was lower for participants who received Orencia at 11%, versus 15% for those who received placebo, and the odds of dying were 37.4% lower, noted June 2, 2022.
0