Submissions Review Committee C1 (Aggregate)

Best Pharmaceutical Product (18) Export data |Download All

Company	Product	Indication		Linda B. BUCK, PhD	Susan DESMOND HELLMANN	Laurie GLIMCHER	Cato T. LAURENCIN	Steven LESTER	Michael ROSENBLATT	Phillip A. SHARP	Marc TESSIER LAVIGNE	Total	Aggregated Vote	Vote 1 Most favorable 5 Least favorable 1=most favorable; 5=least favorable
Genentech, A Member of the Roche Group	PHESGO	PHESGO treats HER2-positive breast cancer: before surgery, after surgery, and metastatic, used with chemotherapy in various stages of the disease.	View Online
Eisai Inc. and Biogen Inc.	LEQEMBI®	LEQEMBI® treats mild Alzheimer’s disease, starting in clinical trials for those with mild cognitive impairment or dementia.	View Online
Merck & Co. Inc.	WINREVAIR™(sotatercept-csrk) for injection, 45mg, 60mg	WINREVAIR™ treats pulmonary arterial hypertension in adults (WHO Group 1) to enhance exercise capacity, improve functional class, and reduce clinical worsening risk.	View Online
Genentech, A Member of the Roche Group	VABYSMO	To treat neovascular (wet) aged-related macular degeneration and diabetic macular edema	View Online
Sanofi-Aventis U.S. LLC	Tzield®	Tzield® is a CD3-directed antibody indicated to delay the onset of Stage 3 type 1 diabetes (T1D) in adults and pediatric patients aged 8 years and older with Stage 2 T1D.	View Online
Incyte Corporation	Opzelura® (ruxolitinib) cream 1.5%	Opzelura® treats mild to moderate atopic dermatitis and nonsegmental vitiligo in adults and children aged 12 and older.	View Online
Innoviva Specialty Therapeutics, Inc.	Xacduro	XACDURO® treats hospital-acquired and ventilator-associated bacterial pneumonia in adults caused by susceptible Acinetobacter baumannii-calcoaceticus complex isolates.	View Online
Eli Lilly and Company	Kisunla™ (donanemab-azbt)	Kisunla is an FDA-approved treatment for adults with early symptomatic Alzheimer’s disease (AD), which includes mild cognitive impairment (MCI) or mild dementia stage of disease.	View Online
Biogen, Sage Therapeutics	ZURZUVAE®	For adults with postpartum depression	View Online
AbbVie	ELAHERE®	ELAHERE® treats FRα-positive, platinum-resistant ovarian cancer after 1-3 prior treatments, with an FDA-approved biomarker test.	View Online
Bristol Myers Squibb	Cobenfy	COBENFY™ (xanomeline and trospium chloride), formerly KarXT, is an oral medication for the treatment of schizophrenia in adults.	View Online
Pfizer Inc.	ZAVZPRET™	ZAVZPRET™ (zavegepant) is indicated for the acute treatment of migraine with or without aura in adults. Zavzpret is not indicated for the preventive treatment of migraine.	View Online
Idorsia Pharmaceuticals Ltd.	Aprocitentan	TRYVIO is a dual endothelin receptor antagonist (ERA) indicated for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure (BP) in adult patients who are not adequately controlled on other drugs. Lowering blood pressure reduces the risk of fatal and non-fatal cardiovascular events, primarily strokes and myocardial infarctions.	View Online
Ardelyx, Inc.	XPHOZAH®	XPHOZAH, 30 mg BID, is indicated to reduce serum phosphorus in adults with chronic kidney disease (CKD) on dialysis as add-on therapy in patients who have an inadequate response to phosphate binders or who are intolerant of any dose of phosphate binder therapy.	View Online
Novartis	PLUVICTO®	PLUVICTO is indicated for the treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor pathway inhibitor (ARPI) therapy, and • are considered appropriate to delay taxane-based chemotherapy, or • have received prior taxane-based chemotherapy.	View Online
GIlead Sciences	Yeztugo®	Yeztugo® (lenacapavir) injection, 463.5 mg/1.5 mL, is indicated for pre‑exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 in adults and adolescents (>35kg) who are at risk for HIV-1 acquisition. Individuals must have a negative HIV-1 test prior to initiating Yeztugo.	View Online
Phathom Pharmaceuticals	Vonoprazan	VOQUEZNA is a potassium-competitive acid blocker (PCAB) indicated: - for relief of heartburn associated with non-erosive gastroesophageal reflux disease in adults. - for healing of all grades of erosive esophagitis and relief of heartburn associated with erosive esophagitis in adults. - to maintain healing of all grades of erosive esophagitis and relief of heartburn associated with erosive esophagitis in adults. - in combination with amoxicillin and clarithromycin for the treatment of Helicobacter pylori (H. pylori) infection in adults. - in combination with amoxicillin for the treatment of H. pylori infection in adults.	View Online
Innoviva Specialty Therapeutics	Zevtera	- Adult patients with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), including those with right-sided infective endocarditis - Adult patients with acute bacterial skin and skin structure infections (ABSSSI) and - Adult and pediatric patients (3 months to less than 18 years old) with community-acquired bacterial pneumonia (CABP)	View Online

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Best Product for Orphan / Rare Diseases (13) Export data

Company	Product	Indication		Linda B. BUCK, PhD	Susan DESMOND HELLMANN	Laurie GLIMCHER	Cato T. LAURENCIN	Steven LESTER	Michael ROSENBLATT	Phillip A. SHARP	Marc TESSIER LAVIGNE	Total	Aggregated Vote	Vote 1 Most favorable 5 Least favorable 1=most favorable; 5=least favorable
Marinus Pharmaceuticals	ZTALMY®	Treatment of seizures associated with CDKL5 deficiency disorder (CDD), in patients 2 years of age and older.	View Online
Biogen	QALSODY®	For the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene.	View Online
Biogen	SKYCLARYS®	SKYCLARYS is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older	View Online
SpringWorks Therapeutics	OGSIVEO®	OGSIVEO is a gamma secretase inhibitor indicated for adult patients with progressing desmoid tumors who require systemic treatment.	View Online
Servier Pharmaceuticals	VORANIGO®	VORANIGO is a prescription medicine used to treat adults and children 12 years of age and older with astrocytoma or oligodendroglioma brain tumors with an isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, following surgery.	View Online
Merus N.V.	Zenocutuzumab	- advanced, unresectable, or metastatic non-small cell lung cancer (NSCLC) harboring a neuregulin 1 gene fusion with disease progression on or after prior systemic therapy, or - advanced, unresectable, or metastatic pancreatic adenocarcinoma (PDAC) harboring a neuregulin 1 gene fusion with disease progression on or after prior systemic therapy	View Online
PTC Therapeutics	KEBILIDI	KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.	View Online
Novo Nordisk	Alhemo®	Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: - hemophilia A (congenital factor VIII deficiency) with FVIII inhibitors - hemophilia B (congenital factor IX deficiency) with FIX inhibitors	View Online
Ionis	TRYNGOLZA™ (olezarsen)	TRYNGOLZA™ (olezarsen) is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).	View Online
Syndax	Revumenib	REVUFORJ® (revumenib) is a first-in-class, oral, potent, and selective menin inhibitor approved in the United States for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A (KMT2A) gene translocation in adult and pediatric patients ≥1 year.1 Revumenib has been granted Orphan Drug designation for the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and acute leukemias of ambiguous lineage by the US Food and Drug Administration (FDA).2	View Online
X4 Pharmaceuticals, Inc.	mavorixafor	WHIM syndrome (Warts, Hypogammaglobulinemia, Infections, Myelokathexis) (approved in U.S., pending in EU), Primary Chronic Neutropenia (Phase 3 ongoing)	View Online
Zevra Therapeutics	MIPLYFFA®	MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older	View Online
Jazz Pharmaceuticals	Ziihera® (zanidatamab-hrii)	Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test. People with metastatic HER2-positive (IHC 3+) BTC received Ziihera by IV once every 2 weeks. Confidential information	View Online

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Best Biotechnology Product (15) Export data |Download All

Company	Product	Indication		Linda B. BUCK, PhD	Susan DESMOND HELLMANN	Laurie GLIMCHER	Cato T. LAURENCIN	Steven LESTER	Michael ROSENBLATT	Phillip A. SHARP	Marc TESSIER LAVIGNE	Total	Aggregated Vote	Vote 1 Most favorable 5 Least favorable 1=most favorable; 5=least favorable
Gilead Sciences	Sunlenca®	Sunlenca is a long-acting HIV capsid inhibitor, used with other HIV medications in heavily treatment-experienced adults.	View Online
MGI Tech	DNBSEQ-T20	DNBSEQ-T20×2 automates genetic sequencing for diverse applications, enhancing global genome projects and healthcare with OMICS technologies.	View Online
Merck & Co Inc.	CAPVAXIVE™	CAPVAXIVE™ is a vaccine for adults to prevent invasive Streptococcus pneumoniae disease and pneumonia caused by specific serotypes.	View Online
Johnson & Johnson Services, Inc.	CARVYKTI® (ciltacabtagene autoleucel)	To treat adult patients with relapsed or refractory multiple myeloma	View Online
Arcutis Biotherapeutics	ZORYVE® (roflumilast)	ZORYVE® foam, 0.3%, treats seborrheic dermatitis in adults and children ≥9 years. ZORYVE® cream, 0.3%, treats plaque psoriasis and ZORYVE® cream, 0.15%, treats mild-to-moderate atopic dermatitis in patients ≥6 years.	View Online
AstraZeneca and Sanofi	Beyfortus (nirsevimab-alip)	Beyfortus® prevents RSV lower respiratory tract disease in newborns and infants up to 24 months, administered via single intramuscular injection.	View Online
Johnson & Johnson Services, Inc.	TALVEY™ (talquetamab-tgvs)	TALVEY™ treats relapsed or refractory multiple myeloma in adults after prior therapies, including a PI, IMiD, and anti-CD38 mAb.	View Online
Pfizer Inc.	ABRYSVO®	ABRYSVO® prevents RSV lower respiratory tract disease in pregnant individuals and infants, and in adults aged 60 years and older.	View Online
Regeneron and Sanofi	Dupixent®	To treat patients with Atopic dermatitis, asthma, and others	View Online
Johnson & Johnson Services, Inc.	TECVAYLI™ (teclistamab-cqyv)	To treat relapsed or refractory multiple myeloma among adults who have received at least four specific lines of therapy	View Online
Novartis	SCEMBLIX®	SCEMBLIX is indicated for the treatment of adult patients with: • Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). (This indication is approved under accelerated approval based on major molecular response rate. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial(s).) • Previously treated Ph+ CML in CP. • Ph+ CML in CP with the T315I mutation.	View Online
Amgen Inc.	IMDELLTRA® (tarlatamab-dlle)	IMDELLTRA® (tarlatamab) is approved for adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy. It is also being studied in limited stage small cell lung cancer, earlier treatment lines of ES-SCLC and in other DLL3 expressing tumors.	View Online
Pfizer Inc./Astellas	PADCEV®	PADCEV®, in combination with pembrolizumab, is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer (la/mUC). PADCEV®, as a single agent, is indicated for the treatment of adult patients with la/mUC who: • have previously received a programmed death receptor-1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor and platinum-containing chemotherapy, or • are ineligible for cisplatin-containing chemotherapy and have previously received one or more prior lines of therapy.	View Online
Catalyst Pharmaceuticals	AGAMREE	Duchenne Muscular Dystrophy	View Online
Bavarian Nordic	VINKUNYA (Chikungunya Vaccine, Recombinant)	VIMKUNYA is a vaccine indicated for the prevention of disease caused by chikungunya virus in individuals 12 years of age and older. (Approved under accelerated approval based on neutralizing antibody levels; continued approval may be contingent on confirmatory trials.)	View Online

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