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int64
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1
RLFTF
Urea cycle disorder (UCD)
Approved
2022-12-23 00:00:00
Approved December 23, 2022.
1
GSK
HIV
Approved
2020-07-02 00:00:00
FDA Approval announced July 2, 2020.
1
PFE
Metastatic colorectal cancer
Approved
2021-09-28 00:00:00
Approved September 28, 2021.
1
OBSV
Severe adenomyosis
Phase 3
2021-10-20 00:00:00
Pivotal trial final results noted that uterine volume was reduced by 55% at 12 weeks and 32% at 24 weeks. Pelvic pain was reduced at 12 and 24 weeks with initial signs of reduction after 4 weeks, noted October 20, 2021.
1
ATNXQ
Metastatic breast cancer
Phase 3
2021-11-22 00:00:00
Phase 3 post hoc analysis showed a median survival rate of 18.9 months in patients treated with Oral Paclitaxel vs 10.1 months in those treated with IV Paclitaxel, with a hazard ratio of 0.59, noted November 22, 2021.
1
EWTX
Duchenne Muscular Dystrophy (DMD)
Approved
2023-10-26 00:00:00
Approved October 26, 2023.
1
AVDL
Reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery
Approved
2013-06-03 00:00:00
Approved June 3, 2013.
1
RHHBY
Relapsed/Refractory Multiple Myeloma (RRMM)
Phase 1
2022-12-12 00:00:00
Phase 1 1 year data reported that the best overall response (BOR) achieved was: stringent complete response (sCR) in 7 patients, CR in 3 patients, very good partial response (VGPR) in 5 patients, and PR in 1 patient, noted December 12, 2022.
1
AMGN
Heart failure with reduced ejection fraction (HFrEF)
CRL
2023-02-28 00:00:00
CRL issued February 28, 2023.
0
GILD
COVID-19 in people with mild, moderate and severe hepatic impairment
Approved
2023-08-24 00:00:00
Approved August 24, 2023.
1
LLY
Migraine
Approved
2019-10-11 00:00:00
FDA Approval announced October 11, 2019.
1
ABBV
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
Approved
2018-06-11 00:00:00
Approval announced June 11, 2018.
1
VALN
Booster shot COVID-19 vaccine
Phase 1/2
2022-01-19 00:00:00
Additional data reported that all 30 samples (100%) presented neutralizing antibodies against the ancestral virus and Delta variant, and 26 samples (87%) presented neutralizing antibodies against the Omicron variant, noted January 19, 2022.
1
BHVN
Focal Epilepsy
Phase 1
2023-09-05 00:00:00
Examination of EEG in healthy subjects administered single doses confirmed central nervous system (CNS) activity consistent with effects observed with other antiseizure medications, noted September 5, 2023.
1
CNTX
Breast Cancer - 2nd-line
Phase 2
2022-12-08 00:00:00
Phase 2 data reported a 4-month PFS rate of 44%, noted December 8, 2022.
0
IMCR
Uveal melanoma
Approved
2022-01-26 00:00:00
Approved January 26, 2022.
1
RHHBY
Hepatocellular Carcinoma (HCC), Solid tumors
Phase 1/2
2023-04-17 00:00:00
Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.
1
DCTH
Liver-dominant ocular melanoma
Approved
2023-08-14 00:00:00
FDA Approved on August 14, 2023.
1
SAVA
Alzheimer’s disease
Phase 2b
2021-09-22 00:00:00
Phase 2b open label data showed ADAS-Cog11 scores improved an average of 3.2 points from baseline, 68% (n=50) improved on ADAS-Cog at 12 months, an average of 6.8 points. 20% declined less than 5 points on ADAS-Cog at 12 months, an average of 2.5 points, noted September 22, 2021.
1
IONS
Hereditary Angioedema (HAE)
Phase 2
2023-02-21 00:00:00
Phase 2 open-label study data reinforce the treatment's potential to be a best-in-class prophylactic treatment for patients living with hereditary angioedema, noted February 21, 2023.
1
PFE
Metastatic hormone sensitive prostate cancer
Phase 3
2021-09-17 00:00:00
Phase 3 data showed a reduced risk of death by 34% compared to placebo plus ADT. The median OS was not reached in either treatment group, September 17, 2021.
1
VTRS
Mydriasis (RM)
Approved
2023-09-27 00:00:00
FDA Approval on September 27, 2023.
1
NKTR
Solid Tumors
Phase 1b
2022-09-10 00:00:00
Phase 1b data presented at ESMO demonstrated preliminary clinical activity similar to that observed with cabozantinib in Phase 1 across a range of solid tumors and dose levels, with a manageable safety profile, noted September 10, 2022.
1
SYBX
Homocystinuria (HCU)
Phase 1
2023-03-12 00:00:00
Phase 1 data presented at SIMD reported that no serious adverse events or deaths related to SYNB1618 or SYNB1934, noted March 19, 2023.
1
EBS
Anthrax Vaccine
Approved
2023-07-21 00:00:00
Approved July 21, 2023.
1
REGN
Prurigo nodularis (PN)
Approved
2022-10-27 00:00:00
Approved in 3Q 2022.
1
FULC
Facioscapulohumeral muscular dystrophy (FSHD)
Phase 3
2023-09-08 00:00:00
Phase 3 enrollment completed, noted September 8, 2023
0
TCRT
Solid tumors
Phase 1/2
2023-08-14 00:00:00
Program wind down due to prioritization. Phase 1/2 interim data reported a 83% disease control rate, noted August 14, 2023.
0
HALO
Various blood cancers
Approved
2017-06-22 00:00:00
Approval announced June 22, 2017.
1
AZN
Acute ischaemic stroke
Approved
2020-11-06 00:00:00
FDA approval announced November 6, 2020.
1
EXEL
Hepatocellular Carcinoma
Phase 3
2022-03-14 00:00:00
Phase 3 final OS data reported no improvement, noted March 14, 2022.
1
RHHBY
HER2-positive breast cancer - residual invasive disease
Approved
2019-05-06 00:00:00
FDA approval announced May 6, 2019.
1
ALNY
Primary Hyperoxaluria Type 1 (PH1)
Approved
2020-11-24 00:00:00
FDA approval announced November 24, 2020.
1
AZN
Hormone Receptor Positive Breast Cancer, Triple Negative Breast Cancer and Non-small Cell Lung Cancer
Phase 1
2022-12-09 00:00:00
Phase 1 data reported an objective response rate (ORR) of 27% with all patients achieving a partial response and 56% with stable disease, noted December 9, 2022.
1
AVTX
Non-Eosinophilic Asthma (NEA)
Phase 2
2023-06-26 00:00:00
Phase 2 did not meet the primary endpoint measured by reduction in asthma related events compared to placebo, noted June 26, 2023.
1
CRIS
Acute Myeloid Leukemia / Myelodysplastic Syndromes (MDS)
Phase 1/2
2023-07-06 00:00:00
FDA removed partial hold, noted July 6, 2023
0
SAGE
Major Depressive Disorder (MDD)
Phase 3
2022-02-16 00:00:00
Phase 3 trial met its primary and key secondary endpoints, comparing a 50 mg co-initiated with standard of care antidepressant vs. standard of care co-initiated with placebo. At the Day 3 primary endpoint, dosing with a standard of care antidepressant showed a statistically significant reduction in depressive symptoms.
1
CORT
Nonalcoholic steatohepatitis (NASH)
Phase 1b
2023-07-17 00:00:00
Phase 1b data demonstrated that miricorilant effectively reduced liver fat, improved liver health and key metabolic and lipid measures and is well-tolerate, noted July 17, 2023.
1
LIAN
Ulcerative Colitis
Phase 2
2021-10-04 00:00:00
Phase 2 data displayed clinical remission in 36.1% of the 880 mg group and 35.5% of the 440 mg group during the blinded maintenance phase. Both dose cohorts had 10% of patients report an AE of ulcerative colitis worsening, noted October 4, 2021.
0
GTHX
Triple-negative breast cancer (TNBC)
Phase 2
2021-11-12 00:00:00
Phase 2 trial data demonstrated that patients who received trilaciclib prior to GCb showed increased T cell function as measured by greater production of inflammatory cytokines, noted November 12, 2021.
1
ACIU
Neurodegenerative diseases (NDD)
Phase 1
2023-07-18 00:00:00
Updated biomarker data reported that ACI-15916shows significantly improved specific binding to pathological a-syn aggregates in brain sections from different a-synucleinopathy cases, noted July 18, 2023.
1
AXSM
Treatment resistant depression
Phase 2
2021-08-09 00:00:00
Phase 2 trial met primary and key secondary endpoints - August 9, 2021.
0
LGND
Relapsed Multiple Myeloma
Approved
2018-01-17 00:00:00
sNDA approved January 17, 2018.
1
BMY
Small cell lung cancer (SCLC)
Approved
2018-08-17 00:00:00
FDA approval announced August 17, 2018.
1
JNJ
Tablet reduction, non-metastatic castration-resistant prostate cancer (nmCRPC)
Approved
2023-04-03 00:00:00
Approved April 3, 2023.
1
EVAX
Metastatic Melanoma
Phase 2b
2023-10-31 00:00:00
Phase 2b data presented at SITC reported that a pronounced and ongoing tumor reduction was observed in a patient with progressive disease, noted October 31, 2023.
1
IPHA
COVID-19
Phase 2
2021-07-06 00:00:00
Phase 2 trial did not meet primary endpoints - July 6, 2021.
1
BCTX
Breast cancer
Phase 1/2
2023-02-23 00:00:00
Additional Phase 1/2 data reported that 9 of 11 (82%) patients remain alive from 2021/2022 dosing and 7 of 11 (64%) patients showed either disease control or progression-free survival, noted February 23, 2023.
1
FRLN
Hemophilia B
Phase 1/2
2021-12-13 00:00:00
Phase 1/2 long term data reported a dose-dependent increase in FIX activity and FIX expression was sustained in nine of 10 patients, noted December 13, 2021.
0
IFRX
ANCA-associated Vasculitis (AAV)
Phase 2
2021-11-15 00:00:00
Phase 2 data released November 15, 2021. Clinical response as well as clinical remission were achieved in comparably high rates in all three arms: Clinical response at week 16 in evaluable patients was observed in 16 out of 18 (88.9%) patients in the treatment group receiving vilobelimab alone; in 22 out of 23 (95.7%) patients receiving SDGC; and in 10 out of 13 (76.9%) patients in the vilobelimab + RDGC group.
1
ALZN
Mild to Moderate Dementia of the Alzheimer's Type
Phase 1/2
2022-03-28 00:00:00
Phase 1 results reported that treatment was safe and well-tolerated and salicylate exposures were within safe limits, noted March 28, 2022.
1
JAZZ
Excessive sleepiness associated with narcolepsy
Approved
2019-03-20 00:00:00
FDA Approval announced March 20, 2019.
1
BMY
Ulcerative colitis (UC)
Phase 2
2023-10-26 00:00:00
Phase 2 trial was unsuccessful, noted October 26, 2023.
0
INCY
Triple-negative breast cancer
Phase 2
2021-12-10 00:00:00
Phase 2 data reported that treatment is tolerated, noted December 10, 2021.
1
JNJ
Multiple myeloma
Approved
2020-05-01 00:00:00
FDA Approval announced May 1, 2020.
1
PULM
Allergic bronchopulmonary aspergillosis in patients with asthma.
Phase 1
2018-07-17 00:00:00
Phase 1 Part 3 data released July 17, 2018 - safe and well tolerated
1
RXDX
Crohn’s Disease
Phase 2a
2023-03-03 00:00:00
Phase 2a primary and secondary results reported efficacy in biologic-experienced subjects consistent with overall population and consistent across subgroups and irrespective of presence of ADA, noted March 3, 2023.
0
NERV
Schizophrenia
Phase 3
2021-09-30 00:00:00
Phase 3 results met all key pharmacokinetic (PK) objectives and the data demonstrate bioequivalence across the various formulations - noted September 30, 2021.
1
REGN
IgA nephropathy
Phase 2
2022-08-29 00:00:00
Phase 2 new results demonstrated an equivalent 36% reduction in 24-hour urine total protein and a higher proportion, 32 vs 13%, of patients treated in comparison to placebo, respectively, achieving greater than or equal to 50% reduction in 24-hour UPCR, noted August 29, 2022.
1
BLUE
Sickle cell disease
PDUFA
2023-06-21 00:00:00
PDUFA Date assigned for December 20, 2023.
0
AZN
Relapsed/refractory chronic lymphocytic leukaemia
Phase 3
2021-12-12 00:00:00
Phase 3 updated results reported a reduced risk of disease progression or death by 71% versus IdR/BR as assessed by investigators at three years, noted December 12, 2021.
1
JAZZ
Small cell lung cancer (SCLC)
Approved
2020-06-15 00:00:00
FDA Approval announced June 15, 2020.
1
AZN
Eosinophilic Esophagitis
Phase 3
2022-10-25 00:00:00
Phase 3 data reported that trial did not meet one of the two dual-primary endpoints, noted October 25, 2022.
1
ORIC
Tumors - EGFR/HER2 Exon 20 Inhibitor
Phase 1b
2023-10-23 00:00:00
Phase 1b initial data presented at ESMO reported responses observed in 2 of 3 EGFR exon 20 patients previously treated with amivantamab (67% ORR), including a confirmed complete response, note October 23, 2023.
1
RVNC
Plantar fasciitis
Phase 2
2020-11-09 00:00:00
Phase 2 data did not meet primary endpoint - November 9, 2020.
1
CLNN
Amyotrophic Lateral Sclerosis
Phase 2
2023-08-29 00:00:00
Phase 2 OLE data reported a significant median survival benefit of 19.3 months using the rank-preserving structural failure time model (RPSFTM) and a significant 52% decreased risk of ALS clinical worsening events, noted August 29, 2023.
1
RGNX
Duchenne Muscular Dystrophy (DMD)
Phase 1/2
2023-10-03 00:00:00
Phase 1/2 additional data shared at the World Muscle Society Congress reported that in the patient aged 4.4 years old, RGX-202 microdystrophin expression was measured to be 38.8% compared to control, and in the patient aged 10.6 years old, RGX-202 microdystrophin expression was measured to be 11.1% compared to control, noted October 3, 2023.
0
SGEN
Cervical Cancer
Phase 3
2023-10-23 00:00:00
Additional data presented at ESMO that the overall survival (OS) was statistically significantly prolonged with TIVDAK, demonstrating a 30% reduction in the risk of death compared with chemotherapy, noted October 22, 2023.
1
MRK
Neurofibromatosis type 1 plexiform neurofibromas
Approved
2020-04-13 00:00:00
FDA Approval announced April 13, 2020.
1
LLY
Ulcerative Colitis
CRL
2023-04-13 00:00:00
CRL announced April 13, 2023.
0
RIGL
Acute myeloid leukemia
Approved
2022-12-01 00:00:00
Approved December 1, 2022.
1
ALEC
Alzheimer’s disease
Phase 1
2021-11-10 00:00:00
Phase 1 data demonstrated target engagement of CD33 in both blood and central nervous system (CNS) compartments at well tolerated doses in healthy volunteers, noted November 10, 2021.
1
RDHL
Gastroenteritis
Phase 3
2017-06-14 00:00:00
Phase 3 top-line data released June 14, 2017 - primary endpoint met.
0
AMRX
Avastin biosimilar
Approved
2022-04-13 00:00:00
Approved April 13, 2022.
1
BGNE
Non-squamous non-small cell lung cancer (NSCLC)
Phase 3
2020-09-17 00:00:00
Phase 3 primary endpoint met - PFS 9.7 months in Arm A compared to 7.6 months in the chemotherapy alone Arm B (p=0.0044; stratified hazard ratio [HR]=0.64.
0
PFE
Lyme disease
Phase 2
2023-09-07 00:00:00
Phase 2 data showed that participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, noted September 7, 2023.
1
VRTX
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
CRL
2016-02-05 00:00:00
CRL issued February 5, 2016.
0
ORGS
Acute pancreatitis
Approved
2018-08-22 00:00:00
Approved August 22, 2018.
1
BLCO
Dry Eye Disease Associated With Meibomian Gland Dysfunction
Approved
2023-06-29 00:00:00
Approved on May 18, 2023.
1
ALBO
Progressive familial intrahepatic cholestasis (PFIC)
Approved
2021-07-20 00:00:00
FDA approval announced July 20, 2021.
1
LPTX
Esophagogastric adenocarcinoma
Phase 1/2
2019-08-06 00:00:00
Phase 1/2 data August 6, 2019 noted 50% ORR.
0
MRNA
COVID-19 vaccine
Approved
2022-01-31 00:00:00
Full approval January 31, 2022.
1
MRK
Newly Diagnosed Classical Hodgkin Lymphoma (cHL)
Phase 2
2022-12-12 00:00:00
Phase 2 data reported that after a median follow-up of 15.5 months, 22 patients had objective response (ORR, 73% [95% CI, 54-88]; complete response, 9 [30%]; partial response, 13 [43%]), noted December 12, 2022.
0
HOOK
Cytomegalovirus (CMV)
Phase 2
2021-11-09 00:00:00
Phase 2 safety, immunogenicity, and efficacy data reported 86% seroconversion and 100% CD8+ T cell responses, 41% reduction in CMV viremia, and 41% reduction in the use of antiviral therapy, noted November 9, 2021.
1
SAGE
SRSE
Phase 3
2017-09-12 00:00:00
Phase 3 data released September 12, 2017 - primary endpoint not met.
0
RGNX
Mucopolysaccharidosis Type I (MPS I)
Phase 1/2
2023-08-30 00:00:00
Phase 1/2 initial interim analysis reported that cognitive function was higher than the age equivalent scores in the available natural history, noted August 30, 2023.
0
ACAD
Adjunctive treatment of schizophrenia
Phase 3
2019-07-22 00:00:00
Phase 3 data did not meet primary endpoint - July 22, 2019.
1
PRVB
Crohn's disease
Phase 2
2019-10-22 00:00:00
Phase 2a data did not meet primary endpoint - October 22, 2019.
1
OCUL
Post-surgical ocular inflammation and pain
Approved
2019-06-21 00:00:00
FDA Approval June 21, 2019.
1
MRK
Stage IB, II or IIIA non-small cell lung cancer (NSCLC)
Approved
2023-01-27 00:00:00
Approved January 27, 2023.
1
MRK
Esophageal Cancer
Approved
2019-07-31 00:00:00
FDA Approval announced July 31, 2019.
1
ARQT
Scalp psoriasis
Phase 2
2021-09-30 00:00:00
Phase 2 study met its primary endpoint with a Scalp-Investigator Global Assessment success rate of 59.1% patients compared to a vehicle rate of 11.4% (P<0.0001). Of these, 34.3% of patients on roflumilast foam achieved a status of clear - noted September 30, 2021.
1
FATE
Relapsed/refractory Multiple Myeloma
Phase 1
2022-12-12 00:00:00
Phase 1 data presented at ASH reported that one patient in Regimen A achieved a partial response, and one patient achieving a minor response in regimen B, noted December 12, 2022.
1
ABBV
Myelofibrosis
Phase 2
2022-12-12 00:00:00
Phase 2 cohort 3 data reported that 35% (9/26) of patients achieved ≥ 1 grade improvement at any time during treatment with a median time-to-improvement of 12.3 weeks, noted December 12, 2022.
1
SNY
Atopic dermatitis
Approved
2017-03-28 00:00:00
Approved under priority review - March 28, 2017.
1
CRIS
Basal Cell Carcinoma
Approved
2012-01-30 00:00:00
Approved January 30, 2012.
1
RAIN
MDM2-amplified advanced solid tumors
Phase 2
2023-05-30 00:00:00
Phase 2 enrollment suspended, noted May 30, 2023.
0
DYN
Myotonic Dystrophy Type 1 (DM1)
Phase 1/2
2023-09-20 00:00:00
Phase 1/2 initiated Septmember 6, 2022. Phase 1/2 Orphan Drug Designation was granted on September 20, 2023.
0
BLCM
HER2+ Solid Tumors
Phase 1/2
2021-12-06 00:00:00
Phase 1/2 initial data reported no dose-limiting toxicities with other Grade 3+ treatment-emergent adverse events being neutropenia, leukopenia, and anemia, noted December 6, 2021.
1
ADIL
Alcohol use disorder
Phase 3
2023-07-11 00:00:00
FDA reviewed the safety data from the ONWARD trial and did not express any concerns with the data, noted July 11, 2023.
0