Grades, scores, and FDA catalyst signals for Rare Disease stocks tracked by BiotechSign
| Ticker | Company | Score | Grade | Sector | Signals |
|---|---|---|---|---|---|
| AGIO | Agios Pharmaceuticals | 79 | B | Rare Disease | — |
| RARE | Ultragenyx Pharmaceutical | 60 | C | Rare Disease | — |
| FOLD | Amicus Therapeutics | 57 | C | Rare Disease | — |
| INSM | Insmed Incorporated | 56 | C | Rare Disease | — |
| BCRX | BioCryst Pharmaceuticals | 55 | C | Rare Disease | — |
| BMRN | BioMarin Pharmaceutical | 53 | C | Rare Disease | — |
| PTCT | PTC Therapeutics | 46 | D | Rare Disease | — |
| PHVS | Pharvaris NV | 39 | D | Rare Disease | — |
| PRQR | ProQR Therapeutics | 35 | F | Rare Disease | — |
| LRMR | Larimar Therapeutics | 29 | F | Rare Disease | — |
| KNSA | Kiniksa Pharmaceuticals | 29 | F | Rare Disease | — |
| STOK | Stoke Therapeutics | 27 | F | Rare Disease | — |
| DSGN | Design Therapeutics | 23 | F | Rare Disease | — |
Orphan drug development targets diseases affecting fewer than 200,000 patients in the United States. The Orphan Drug Act provides significant incentives: 7 years of market exclusivity, a 25% tax credit on clinical trial costs, and waived FDA filing fees. FDA Priority Review, Breakthrough Therapy, Accelerated Approval, and Fast Track designations are common in this space, compressing timelines and reducing capital requirements versus traditional development. Binary PDUFA catalysts in rare disease can be especially enormous in percentage terms given the small patient populations and often monopolistic market dynamics. Investors should track orphan designation status, natural history data strength, and surrogate endpoint acceptability.