Rare Genetic & MetabolicUncontestedData as of 2026-09-04
Antithrombin III Deficiency is uncontested. Grifols, S.A. Sponsored ADR (GRFS) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with Plasma-derived AT-III in phase 2.
There is no public comparable to price it against. GRFS spreads its $2.1B market cap across 2 indications, so $1.0B of it is allocated here.
Nothing here has reached Phase 3: 1 programme in mid stage and 0 in early stage, so the field is still several years from a first approval.
$1.0B of allocated capital sits in Antithrombin III Deficiency, ranking it 152nd of 299 indications by the money committed to it.
| Ticker | Company | Lead asset | Phase | Trials | Market cap | Pure play | Indications | Next catalyst |
|---|---|---|---|---|---|---|---|---|
| GRFS | Grifols, S.A. Sponsored ADR | Plasma-derived AT-III | Phase 2 | 1 | $2.1B | No | 2 | 31 Dec 2026 |
Sorted by phase, then by market capitalisation. Ticker links open the company profile.
Uncontested. One US-listed developer in the size band is running a clinical programme here, so there is no public comparable to price it against.
Rare Genetic & Metabolic holds $103B of the map's capital, 15.3% of the total, spread across 32 indications and 48 companies. 10 of those indications have more than one company competing.
See every Rare Genetic & Metabolic indication on the mapGrifols, S.A. Sponsored ADR (GRFS, Plasma-derived AT-III, Phase 2). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Antithrombin III Deficiency.
Of the 1 clinical-stage programme in Antithrombin III Deficiency, 0 are in Phase 3 or filed, 1 is in mid-stage development and 0 are in early-stage development.
No. GRFS is the only US-listed company in the $100M to $20B market cap band with a clinical-stage programme in Antithrombin III Deficiency, which is why the indication is classified as uncontested.
The next dated catalyst in Antithrombin III Deficiency is GRFS on 31 Dec 2026. 1 dated event across the field fall in the next 12 months.
Allocated capital divides each company’s market capitalisation evenly across the indications it is running clinical programmes in, so every dollar of the universe is counted exactly once. Total market capitalisation competing counts a company in each of its indications and therefore sums to more than the universe.
Third-party market size estimates are not used anywhere on this page. Approved incumbents are excluded from competitor counts, and companies above $20B are outside the universe by construction.
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