Table 1.
New Molecular Entity Applications Accepted Under FDA’s RTOR Pilota
| Molecule | Special Designations | ORBIS | Pivotal Trials | Disease Area | Review Time (Months) | Approved | CDx |
|---|---|---|---|---|---|---|---|
| Piqray (alpelisib) | PR | No | Ph3 randomized, placebo controlled, double blinded; PFS | HR+, HER2−, PIK3CA mutated, adv or mBC | 5 | 2019–05–24 | Therascreen PIK3CA RGQ PCR Kit |
| Tukysa (tucatinib) |
BTD ODD PR |
Yes | Ph3 randomized, placebo/active controlled, double blind; PFS | 2L HER2 + adv unrespectable or mBC (including brain mets) | 4 | 2020–04–17 | No |
| Qinlock (ripretinib) |
BTD ODD PR |
Yes | Ph3 randomized, placebo controlled, double blinded; PFS | 4L Advanced GIST | 5 | 2020–05–15 | No |
| Blenrep (belantamab) |
AA BTD ODD PR |
No | Ph2, open label, randomized; ORR by IRC | 5L Multiple Myeloma | 8 | 2020–08–05 | No |
| Danyelza (naxitamab) |
AA BTD ODD PR |
No | Ph2 single arm, open label; ORR | 2L relapsed or refractory high-risk neuroblastoma | 8 | 2020–11–25 | No |
| Gavreto (pralsetinib) |
AA BTD ODD PR |
No | Ph1/2 open label, single arm; ORR by BICR | Adv or met RET mutant MTC | 5 | 2020–12–01 | No |
| Tepmetko (tepotinib) |
AA BTD ODD PR |
Yes | Ph2 single arm, open label; ORR by BICR | NSCLC with MET Exon 14 skipping | 7 | 2021–02–03 | No |
| Jemperli (dostarlimab) |
AA BTD PR |
No | Ph1 open label, single arm; ORR, DoR by BICR | dMMR recurrent or advanced EC | 16b | 2021–04–22 | VENTANA MMR RxDx Panel |
| Lumakras (sotorasib) |
AA FT BTD ODD PR |
Yes | Ph1/2 open label, single arm; ORR, DoR by BICR | KRAS G12C-mutated LA or met NSCLC | 5.5 | 2021–05–28 |
Therascreen KRAS RGQ PCR Kit Guardant360® CDx (plasma) |
| Truseltiq (infigratinib) |
AA FT ODD PR |
Yes | Ph2 open label, single arm; ORR, DoR by BICR | 2L LA or met cholangiocarcinoma with FGFR2 fusion or other rearrangement | 8 | 2021–05–28 | FoundationOne CDx |
aUp to May 2021
bExtended review timeline due to COVID-19-related travel restrictions preventing manufacturing inspection [4]
AA Accelerated approval, BTD breakthrough therapy designation, ODD orphan drug designation, FT fast track, PR priority review