Table 1.
Medicines evaluated in PRIME scheme.
| Medicine | Therapeutic indications | Reason for disease’s unmet medical need |
|---|---|---|
| Avacopan | AAV | Rare disease |
| Axicabtagene ciloleucel (CAR-T) | DLBCL | Rare disease |
| Emapalumab | HLH | Rare disease |
| Onasemnogene abeparvovec | SMA Type 1 in pediatric patients | Rare disease |
| Tisagenlecleucel | R/R ALL | Less than 10% of patients achieve 5-year overall survival. Moreover, the R/R ALL is associated with high relapse rates (Papadantonakis and Advani, 2016) |
| Aducanumab | Alzheimer’s disease | Current drugs improve symptoms, but do not have profound disease-modifying effects (Citron, 2010) |
| Asunercept | Glioblastoma | Rare disease |
| Brexanolone (Allopregnanolone, SAGE-547) | PPD | There is a need for new treatment options for mothers suffering from the disorder (Kanes et al., 2017) |
| Chimeric 2’-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA (RO7234292) | Huntington’s Disease | Rare disease |
| Deoxycytidine / deoxythymidine | TK2 | Rare disease |
| Givosiran | Prevention of acute attacks of hepatic porphyria | Rare disease |
| Lumasiran | PH1 | Rare disease |
| MV-CHIK vaccine | Prevention of Chikungunya fever | Rare disease |
| Mycobacterium tuberculosis (MTBVAC) | TB Vaccine | Immune responses of human newborns and infants are distinct and cannot be predicted from those of human adults or animal models (Sanchez-Schmitz and Levy, 2011) |
| Olipudase alfa | Non-neurological manifestations of acid sphingomyelinase deficiency | Rare disease |
| Polatuzumab vedotin | R/R DLBCL | Rare disease |
| Rapastinel | Adjunctive treatment of MDD | Many patients with MDD fail to achieve a complete response with antidepressant medications and experience periods with residual symptom burdens (Rush et al., 2006) |
| Recombinant Vesicular Stomatitis Virus with Envelope Glycoprotein replaced by Zaire ebolavirus (Kikwit Strain) Glycoprotein | Vaccination against Ebola | Many survivors and their relatives continue to experience stigma and social isolation. Moreover, patients have health, psychological, and social needs (Calnan et al., 2017) |
| Seladelpar (MBX-8025) | Primary Biliary Cholangitis | Rare disease |
| Setmelanotide | Obesity and control of the hunger associated with deficiency disorders of the MC4R receptor pathway | Rare Genetic Disorders |
| Setrusumab | OI types I, III, and IV | Rare disease |
| Tasadenoturev | Recurrent glioblastoma | Rare disease |
| Vocimagene amiretrorepvec | Treatment of high-grade glioma | Rare disease |
| Voxelotor | SCD | Many patients experience poor clinical outcomes in short and longer term (Sarri et al., 2018) |
AAV, active ANCA-associated vasculitis (including granulomatosis with polyangiitis and microscopic polyangiitis); DLBCL, large B-cell lymphoma; HLH, primary hemophagocytic lymphohistiocytosis, SMA: spinal muscular atrophy; R/R ALL, relapsed or refractory B cell acute lymphoblastic leukemia; PPD, Postpartum depression; TK2, Thymidine Kinase 2 Deficiency; PH1, Primary Hyperoxaluria Type 1; TB Vaccine, Active immunization against tuberculosis disease; R/R DLBCL, relapsed and refractory patients with diffuse large B cell lymphoma; MDD, major depressive disorder; OI, Osteogenesis Imperfecta; SCD, Sickle Cell Disease.