| Voxzogo® |
Vosoritid |
BioMarin Europe |
Achondroplasia |
2021 |
| Strimvelis® |
Autologous CD34+ ADA cells |
Fondazione Telethon |
ADA-SCID |
2016 |
| Kimmtrak® |
Tebentafusp |
Immunocore |
Uveal Melanoma |
2022 |
| Imcivree® |
Setmelanotide |
Rhythm Pharmaceuticals |
Leptin Receptor Deficiency Obesity |
2021 |
| Lamzede® |
Velmanase alfa |
Chiesi Farmaceutici |
Alpha-Mannosidosis |
2018 |
| Qalsody® |
Tofersen |
Biogen |
Amyotrophic Lateral Sclerosis |
2024 |
| Takhzyro® |
Lanadelumab |
Takeda |
Hereditary Angioedema |
2018 |
| Upstaza® |
Eladocagen Exuparvovec |
PTC Therapeutics |
AADC Deficiency |
2022 |
| Imcivree® |
Setmelanotide |
Rhythm Pharmaceuticals |
Bardet–Biedl Syndrome |
2022 |
| Casgevy® |
Exagamglogen Autotemcel |
Vertex |
Beta-Thalassemia |
2024 |
| Reblozyl® |
Luspatercept |
Bristol Myers Squibb |
Beta-Thalassemia Anemia |
2020 |
| Ziihera® |
Zanidatamab |
Jazz Pharmaceuticals |
Biliary Carcinoma |
2025 |
| Cresemba® |
Isavuconazole |
Basilea Pharmaceutica |
Invasive Aspergillosis |
2015 |
| Agamree® |
Vamorolon |
Santhera |
Duchenne Muscular Dystrophy |
2023 |
| Sephience® |
Sepiapterin |
PTC Therapeutics |
Pancreatic Cancer |
2025 |
| Vykat XR |
Diazoxide choline |
Soleno Therapeutics |
Prader-Willi Syndrome |
2023 |
| Duvyzat |
Givinostat |
Italfarmaco SpA |
Duchenne Muscular Dystrophy |
2023 |
| YUTREPIA |
Treprostinil |
United Therapeutics Corp. |
Pulmonary Arterial Hypertension |
2022 |
| Relacorilant |
Relacorilant |
Corcept Therapeutics |
Platinum-resistant Ovarian Cancer |
2023 |
| Revumenib |
Revumenib citrate |
Syndax Pharmaceuticals |
Acute Leukemia |
2023 |
| Papzimeos |
Zopapogene Imadenovec |
Precigen Inc. |
Recurrent Respiratory Papillomatosis |
2024 |
| Bempedoic Acid |
Bempedoic Acid |
Esperion Therapeutics |
Hyperlipidemia |
2020 |
| HERNICORE |
Condoliase |
Seikagaku Corp. |
Lumbar Disc Herniation |
2018 |
| Evkeeza |
Evinacumab |
Regeneron Pharmaceuticals |
Homozygous Familial Hypercholesterolemia |
2021 |
| WIDAPLIK |
Telmisartan + Amlodipine + Indapamide |
George Medicines Pty Ltd |
Hypertension |
2024 |
| Skysona® |
Elivaldogene autotemcel |
bluebird bio |
Cerebral Adrenoleukodystrophy |
2021 |
| Zynteglo® |
Betibeglogene autotemcel |
bluebird bio |
Transfusion-dependent β-thalassemia |
2022 |
| Libmeldy® |
Atidarsagene autotemcel |
Orchard Therapeutics |
Metachromatic Leukodystrophy |
2020 |
| Hemgenix® |
Etranacogene dezaparvovec |
CSL Behring |
Hemophilia B |
2022 |
| Roctavian® |
Valoctocogene roxaparvovec |
BioMarin |
Hemophilia A |
2023 |
| Luxturna® |
Voretigene neparvovec |
Novartis / Spark Therapeutics |
Inherited Retinal Dystrophy (RPE65 mutation) |
2018 |
| Brineura® |
Cerliponase alfa |
BioMarin |
CLN2 Batten Disease |
2017 |
| Orkambi® |
Ivacaftor + Lumacaftor |
Vertex Pharmaceuticals |
Cystic Fibrosis (F508del mutation) |
2015 |
| Symkevi® |
Tezacaftor + Ivacaftor |
Vertex Pharmaceuticals |
Cystic Fibrosis |
2018 |
| Trikafta® |
Elexacaftor + Tezacaftor + Ivacaftor |
Vertex Pharmaceuticals |
Cystic Fibrosis (F508del homozygous) |
2020 |
| Nexviadyme® |
Avapritamide alfa |
Sanofi Genzyme |
Pompe Disease |
2022 |
| Elaprase® |
Idursulfase |
Takeda |
Hunter Syndrome (MPS II) |
2007 |
| Vimizim® |
Elosulfase alfa |
BioMarin |
Morquio A Syndrome (MPS IVA) |
2014 |
| Myalept® |
Metreleptin |
Amryt Pharma |
Generalized Lipodystrophy |
2018 |
| Uplizna® |
Inebilizumab |
Horizon Therapeutics |
Neuromyelitis Optica Spectrum Disorder |
2022 |
| Soliris® |
Eculizumab |
Alexion Pharmaceuticals |
Paroxysmal Nocturnal Hemoglobinuria (PNH) |
2007 |
| Ultomiris® |
Ravulizumab |
Alexion Pharmaceuticals |
PNH / aHUS |
2019 |
| Bylvay® |
Odevixibat |
Albireo Pharma |
Progressive Familial Intrahepatic Cholestasis |
2021 |
| Livmarli® |
Maralixibat |
Mirum Pharmaceuticals |
Alagille Syndrome (Cholestasis) |
2022 |
| Orladeyo® |
Berotralstat |
BioCryst Pharmaceuticals |
Hereditary Angioedema (Prophylaxis) |
2021 |
| Adcetris® |
Brentuximab vedotin |
Takeda / Seagen |
Hodgkin Lymphoma / ALCL |
2012 |
| Blincyto® |
Blinatumomab |
Amgen |
Acute Lymphoblastic Leukemia |
2015 |
| Kymriah® |
Tisagenlecleucel |
Novartis |
B-cell Acute Lymphoblastic Leukemia / DLBCL |
2018 |
| Yescarta® |
Axicabtagene ciloleucel |
Gilead / Kite Pharma |
Large B-cell Lymphoma / Follicular Lymphoma |
2018 |
| Carvykti® |
Ciltacabtagene autoleucel |
Janssen / Legend Biotech |
Multiple Myeloma |
2022 |
| Abecma® |
Idecabtagene vicleucel |
Bristol Myers Squibb / 2seventy bio |
Multiple Myeloma |
2021 |
| Tecartus® |
Brexucabtagene autoleucel |
Gilead / Kite Pharma |
Mantle Cell Lymphoma |
2020 |
| Breyanzi® |
Lisocabtagene maraleucel |
Bristol Myers Squibb |
Diffuse Large B-cell Lymphoma |
2021 |
| Besponsa® |
Inotuzumab ozogamicin |
Pfizer |
Relapsed / Refractory ALL |
2017 |
| Polivy® |
Polatuzumab vedotin |
Roche / Genentech |
Diffuse Large B-cell Lymphoma |
2019 |
| Monjuvi® |
Tafasitamab |
Incyte / MorphoSys |
Relapsed / Refractory DLBCL |
2021 |
| Copiktra® |
Duvelisib |
Verastem Oncology |
Chronic Lymphocytic Leukemia / Follicular Lymphoma |
2019 |
| Calquence® |
Acalabrutinib |
AstraZeneca |
Chronic Lymphocytic Leukemia |
2020 |
| Brukinsa® |
Zanubrutinib |
BeiGene |
Waldenström Macroglobulinemia / CLL |
2021 |
| Imbruvica® |
Ibrutinib |
AbbVie / Janssen |
B-cell Malignancies |
2014 |
| Venclexta® |
Venetoclax |
AbbVie / Roche |
CLL / AML |
2016 |
| Tibsovo® |
Ivosidenib |
Servier |
AML / IDH1-mutant Cholangiocarcinoma |
2019 |
| Idhifa® |
Enasidenib |
Bristol Myers Squibb |
Relapsed / Refractory AML (IDH2-mutant) |
2018 |
| Rydapt® |
Midostaurin |
Novartis |
AML (FLT3-mutant) |
2017 |
| Besremi® |
Ropeginterferon alfa-2b |
AOP Orphan Pharmaceuticals |
Polycythemia Vera |
2019 |
| Scemblix® |
Asciminib |
Novartis |
Chronic Myeloid Leukemia |
2022 |
| Rydapt® |
Midostaurin |
Novartis |
AML (FLT3-mutant) |
2017 |
| Jakafi® |
Ruxolitinib |
Incyte Corporation |
Myelofibrosis / Polycythemia Vera |
2012 |
| Opzelura® |
Ruxolitinib cream |
Incyte Corporation |
Atopic Dermatitis / Vitiligo |
2022 |
| Reblozyl® |
Luspatercept |
Bristol Myers Squibb |
Myelodysplastic Syndromes / β-thalassemia anemia |
2020 |
| Vabysmo® |
Faricimab |
Roche / Genentech |
Wet Age-related Macular Degeneration |
2022 |
| Susvimo® |
Ranibizumab Port Delivery System |
Roche / Genentech |
Wet AMD / Diabetic Retinopathy |
2021 |
| Beovu® |
Brolucizumab |
Novartis |
Wet AMD |
2020 |
| Izervay® |
Avacincaptad pegol |
Iveric Bio / Astellas |
Geographic Atrophy (AMD) |
2023 |
| Syfovre® |
Pegcetacoplan |
Apellis Pharmaceuticals |
Geographic Atrophy (AMD) |
2023 |
| Empaveli® |
Pegcetacoplan |
Apellis Pharmaceuticals |
Paroxysmal Nocturnal Hemoglobinuria |
2021 |
| Ultomiris® |
Ravulizumab |
Alexion Pharmaceuticals |
aHUS / PNH |
2019 |
| Orladeyo® |
Berotralstat |
BioCryst Pharmaceuticals |
Hereditary Angioedema |
2021 |
| Livmarli® |
Maralixibat |
Mirum Pharmaceuticals |
Alagille Syndrome / Cholestasis |
2022 |
| Bylvay® |
Odevixibat |
Albireo Pharma |
Progressive Familial Intrahepatic Cholestasis |
2021 |
| Elaprase® |
Idursulfase |
Takeda |
Hunter Syndrome (MPS II) |
2007 |
| Vimizim® |
Elosulfase alfa |
BioMarin |
Morquio A Syndrome (MPS IVA) |
2014 |
| Nexviadyme® |
Avapritamide alfa |
Sanofi Genzyme |
Pompe Disease |
2022 |
| Myozyme® |
Alglucosidase alfa |
Sanofi Genzyme |
Pomp Disease |
2006 |
| Naglazyme® |
Galsulfase |
BioMarin |
Maroteaux-Lamy Syndrome (MPS VI) |
2005 |
| Aldurazyme® |
Laronidase |
Sanofi Genzyme / BioMarin |
Hurler / Scheie Syndrome (MPS I) |
2003 |
| Fabrazyme® |
Agalsidase beta |
Sanofi Genzyme |
Fabry Disease |
2001 |
| Replagal® |
Agalsidase alfa |
Takeda |
Fabry Disease |
2001 |
| Elaprase® |
Idursulfase |
Takeda |
Hunter Syndrome (MPS II) |
2007 |
| Brineura® |
Cerliponase alfa |
BioMarin |
CLN2 Batten Disease |
2017 |
| Galafold® |
Migalasidase |
Amicus Therapeutics |
Fabry Disease (Amenable Mutations) |
2016 |
| Amondys 45® |
Casimersen |
Sarepta Therapeutics |
Duchenne Muscular Dystrophy (Exon 45 Skipping) |
2021 |
| Vyondys 53® |
Golodirsen |
Sarepta Therapeutics |
Duchenne Muscular Dystrophy (Exon 53 Skipping) |
2020 |
| Exondys 51® |
Eteplirsen |
Sarepta Therapeutics |
Duchenne Muscular Dystrophy (Exon 51 Skipping) |
2016 |
| Elevidys® |
Delandistrogene moxeparvovec |
Sarepta Therapeutics |
Duchenne Muscular Dystrophy (Gene Therapy) |
2023 |
| Spinraza® |
Nusinersen |
Biogen |
Spinal Muscular Atrophy (SMA) |
2017 |
| Zolgensma® |
Onasemnogene abeparvovec |
Novartis |
Spinal Muscular Atrophy (SMA) |
2020 |
| Evrysdi® |
Risdiplam |
Roche / Genentech |
Spinal Muscular Atrophy (SMA) |
2021 |
| Truseltiq® |
Infigratinib |
BridgeBio Pharma |
Cholangiocarcinoma (FGFR2 fusion) |
2021 |
| Pemazyre® |
Pemigatinib |
Incyte Corporation |
Cholangiocarcinoma (FGFR2 rearranged) |
2020 |
| Retevmo® |
Selpercatinib |
Eli Lilly |
RET fusion-positive Thyroid / Lung Cancer |
2021 |
| Gavreto® |
Pralsetinib |
Blueprint Medicines |
RET fusion-positive NSCLC / Thyroid Cancer |
2021 |
| Rozlytrek® |
Entrectinib |
Roche |
NTRK / ROS1-positive Solid Tumors |
2020 |
| Vitrakvi® |
Larotrectinib |
Bayer |
NTRK gene fusion-positive Solid Tumors |
2019 |
| Ayvakit® |
Avapritinib |
Blueprint Medicines |
PDGFRA exon 18 mutant GIST / Advanced SM |
2020 |
| Tabrecta® |
Capmatinib |
Novartis |
MET exon 14 skipping NSCLC |
2021 |
| Tepmetko® |
Tepotinib |
Merck KGaA |
MET exon 14 skipping NSCLC |
2021 |
| Rybrevant® |
Amivantamab |
Janssen Biotech |
EGFR exon 20 insertion NSCLC |
2021 |
| Lumykras® |
Sotorasib |
Amgen |
KRAS G12C-mutated NSCLC |
2021 |
| Krazati® |
Adagrasib |
Mirati Therapeutics |
KRAS G12C-mutated NSCLC |
2023 |
| Rozlytrek® |
Entrectinib |
Roche |
NTRK / ROS1-positive Solid Tumors |
2020 |
| Vitrakvi® |
Larotrectinib |
Bayer |
NTRK gene fusion-positive Solid Tumors |
2019 |
| Elahere® |
Mirvetuximab soravtansine |
ImmunoGen |
Folate receptor-alpha positive Ovarian Cancer |
2023 |
| Tivdak® |
Tisotumab vedotin |
Seagen / Genmab |
Cervical Cancer |
2022 |
| Padcev® |
Enfortumab vedotin |
Astellas / Seagen |
Urothelial Carcinoma |
2020 |
| Trodelvy® |
Sacituzumab govitecan |
Gilead Sciences |
Triple-Negative Breast Cancer |
2021 |
| Enhertu® |
Trastuzumab deruxtecan |
AstraZeneca / Daiichi Sankyo |
HER2-positive Breast / Gastric Cancer |
2021 |
| Margenza® |
Margetuximab |
MacroGenics |
HER2-positive Breast Cancer |
2021 |
| Opdivo® |
Nivolumab |
Bristol Myers Squibb |
Various Cancers (PD-1 inhibitor) |
2015 |
| Keytruda® |
Pembrolizumab |
Merck & Co. |
Various Cancers (PD-1 inhibitor) |
2015 |
| Libtayo® |
Cemiplimab |
Regeneron / Sanofi |
Cutaneous Squamous Cell Carcinoma |
2019 |
| Imfinzi® |
Durvalumab |
AstraZeneca |
NSCLC / SCLC / Bladder Cancer |
2018 |
| Bavencio® |
Avelumab |
Pfizer / Merck KGaA |
Merkel Cell Carcinoma / Bladder Cancer |
2017 |
| Jemperli® |
Dostarlimab |
GSK |
Endometrial Cancer (dMMR/MSI-H) |
2022 |
| Tecentriq® |
Atezolizumab |
Roche / Genentech |
NSCLC / TNBC / Bladder Cancer |
2016 |
| Tabrecta® |
Capmatinib |
Novartis |
NSCLC (MET exon 14 skipping) |
2021 |
| Rybrevant® |
Amivantamab |
Janssen Biotech |
NSCLC (EGFR exon20 insertion) |
2021 |
| Balversa® |
Erdafitinib |
Janssen |
Urothelial Carcinoma (FGFR2/3 alterations) |
2019 |
| Padcev® |
Enfortumab vedotin |
Seagen / Astellas |
Urothelial Carcinoma |
2020 |
| Trodelvy® |
Sacituzumab govitecan |
Gilead Sciences |
Triple-negative Breast Cancer / Urothelial Cancer |
2021 |
| Enhertu® |
Trastuzumab deruxtecan |
AstraZeneca / Daiichi Sankyo |
HER2-positive Breast / Gastric / Lung Cancer |
2021 |
| Margenza® |
Margetuximab |
MacroGenics |
HER2-positive Breast Cancer |
2021 |
| Tukysa® |
Tucatinib |
Seagen |
HER2-positive Breast Cancer |
2020 |
| Orserdu® |
Elacestrant |
Stemline Therapeutics |
ER-positive / ESR1-mutated Breast Cancer |
2023 |
| Verzenio® |
Abemaciclib |
Eli Lilly |
HR+/HER2- Breast Cancer |
2018 |
| Ibrance® |
Palbociclib |
Pfizer |
HR+/HER2- Breast Cancer |
2015 |
| Kisqali® |
Ribociclib |
Novartis |
HR+/HER2- Breast Cancer |
2017 |
| Lynparza® |
Olaparib |
AstraZeneca / Merck |
BRCA-mutated Ovarian / Breast / Pancreatic Cancer |
2014 |
| Zejula® |
Niraparib |
GSK |
Ovarian Cancer (Maintenance Therapy) |
2017 |
| Rubraca® |
Rucaparib |
Clovis Oncology |
Ovarian / Prostate Cancer |
2018 |
| Talzenna® |
Talazoparib |
Pfizer |
BRCA-mutated Breast / Prostate Cancer |
2019 |
| Xtandi® |
Enzalutamide |
Astellas / Pfizer |
Prostate Cancer |
2014 |
| Erleada® |
Apalutamide |
Janssen Biotech |
Prostate Cancer |
2019 |
| Nubeqa® |
Darolutamide |
Bayer |
Non-metastatic Castration-Resistant Prostate Cancer |
2020 |
| Pluvicto® |
Lutetium Lu 177 vipivotide tetraxetan |
Novartis |
PSMA-positive Metastatic Prostate Cancer |
2022 |
| Keytruda® |
Pembrolizumab |
Merck & Co. |
Multiple Cancers (PD-1 inhibitor) |
2015 |
| Opdivo® |
Nivolumab |
Bristol Myers Squibb |
Melanoma / Lung / Renal / Other Cancers |
2015 |
| Libtayo® |
Cemiplimab |
Regeneron / Sanofi |
Cutaneous Squamous Cell Carcinoma / NSCLC |
2019 |
| Imfinzi® |
Durvalumab |
AstraZeneca |
NSCLC / SCLC / Bladder Cancer |
2018 |
| Tepezza® |
Teprotumumab |
Horizon Therapeutics |
Thyroid Eye Disease |
2021 |
| Actemra® |
Tocilizumab |
Roche |
Rheumatoid Arthritis / Cytokine Release Syndrome |
2009 |
| Kevzara® |
Sarilumab |
Sanofi / Regeneron |
Rheumatoid Arthritis |
2017 |
| Dupixent® |
Dupilumab |
Sanofi / Regeneron |
Atopic Dermatitis / Asthma / CRS |
2017 |
| Fasenra® |
Benralizumab |
AstraZeneca |
Severe Eosinophilic Asthma |
2017 |
| Nucala® |
Mepolizumab |
GSK |
Eosinophilic Asthma / EGPA |
2015 |
| Cinqaero® |
Reslizumab |
Teva Pharmaceuticals |
Severe Asthma |
2016 |
| Ocrevus® |
Ocrelizumab |
Roche / Genentech |
Multiple Sclerosis |
2017 |
| Mavenclad® |
Cladribine |
Merck Group |
Relapsing Multiple Sclerosis |
2019 |
| Kesimpta® |
Ofatumumab |
Novartis |
Relapsing Multiple Sclerosis |
2021 |
| Zeposia® |
Ozanimod |
Bristol Myers Squibb |
Multiple Sclerosis / Ulcerative Colitis |
2020 |
| Mayzent® |
Siponimod |
Novartis |
Secondary Progressive Multiple Sclerosis |
2019 |
| Tysabri® |
Natalizumab |
Biogen |
Multiple Sclerosis / Crohn’s Disease |
2006 |
| Lemtrada® |
Alemtuzumab |
Sanofi Genzyme |
Multiple Sclerosis |
2014 |
| Gilenya® |
Fingolimod |
Novartis |
Relapsing Multiple Sclerosis |
2011 |
| Vumerity® |
Diroximel fumarate |
Biogen |
Relapsing Multiple Sclerosis |
2020 |
| Ozempic® |
Semaglutide |
Novo Nordisk |
Type 2 Diabetes Mellitus |
2017 |
| Wegovy® |
Semaglutide |
Novo Nordisk |
Obesity / Weight Management |
2021 |
| Mounjaro® |
Tirzepatide |
Eli Lilly |
Type 2 Diabetes / Obesity |
2022 |
| Insulet Omnipod 5® |
Automated insulin delivery system |
Insulet Corporation |
Type 1 Diabetes |
2022 |
| Leqvio® |
Inclisiran |
Novartis |
Hypercholesterolemia |
2021 |
| Evkeeza® |
Evinacumab |
Regeneron |
Homozygous Familial Hypercholesterolemia |
2021 |
| Repatha® |
Evolocumab |
Amgen |
Hyperlipidemia / ASCVD Risk Reduction |
2015 |
| Praluent® |
Alirocumab |
Sanofi / Regeneron |
Hyperlipidemia / Cardiovascular Risk |
2015 |
| Leqembi® |
Lecanemab |
Eisai / Biogen |
Alzheimer’s Disease |
2023 |
| Aduhelm® |
Aducanumab |
Biogen |
Alzheimer’s Disease |
2021 |
| Exservan® |
Riluzole oral film |
Aquestive Therapeutics |
Amyotrophic Lateral Sclerosis (ALS) |
2019 |
| Relyvrio® |
Sodium phenylbutyrate + taurursodiol |
Amylix Pharmaceuticals |
Amyotrophic Lateral Sclerosis (ALS) |
2022 |
| Radicava ORS® |
Edaravone oral suspension |
Mitsubishi Tanabe Pharma |
Amyotrophic Lateral Sclerosis (ALS) |
2022 |
| Skyrizi® |
Risankizumab |
AbbVie |
Psoriasis / Crohn’s Disease / Ulcerative Colitis |
2019 |
| Rinvoq® |
Upadacitinib |
AbbVie |
Rheumatoid Arthritis / UC / Atopic Dermatitis |
2019 |
| Tremfya® |
Guselkumab |
Janssen |
Psoriasis / Psoriatic Arthritis |
2017 |
| Ilumya® |
Tildrakizumab |
Sun Pharma |
Moderate-to-severe Plaque Psoriasis |
2018 |
| Cosentyx® |
Secukinumab |
Novartis |
Psoriasis / Psoriatic Arthritis / AS |
2015 |
| Taltz® |
Ixekizumab |
Eli Lilly |
Psoriasis / Psoriatic Arthritis / AS |
2016 |
| Sotyktu® |
Deucravacitinib |
Bristol Myers Squibb |
Moderate-to-severe Plaque Psoriasis |
2022 |
