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Paroxysmal Nocturnal Hemoglobinuria: one US-listed biotech with a clinical-stage programme

Rare Genetic & MetabolicUncontestedData as of 2026-09-04

Competitors1in the clinic
Median market cap$1.4Buniverse median $1.20B
Allocated capital$461M$1.4B competing
Phase 3 or filed01 mid, 0 early
Pure plays0this indication only
Capital rank211/299crowding rank 216

Paroxysmal Nocturnal Hemoglobinuria is uncontested. Omeros (OMER) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with OMS906 study drug in phase 2.

There is no public comparable to price it against. OMER spreads its $1.4B market cap across 3 indications, so $461M 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.

$461M of allocated capital sits in Paroxysmal Nocturnal Hemoglobinuria, ranking it 211th of 299 indications by the money committed to it.

Companies developing drugs for Paroxysmal Nocturnal Hemoglobinuria 1

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
OMEROmerosOMS906 study drugPhase 21$1.4BNo3No dated event

Sorted by phase, then by market capitalisation. Ticker links open the company profile.

Why Paroxysmal Nocturnal Hemoglobinuria counts as uncontested

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.

The map splits indications on two folds: six or more competitors counts as crowded, and a median company above $1.20B counts as richly priced. Paroxysmal Nocturnal Hemoglobinuria has one competitor and a median of $1.4B.

Rare Genetic & Metabolic

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 map

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Paroxysmal Nocturnal Hemoglobinuria pipeline questions

Which biotech companies are developing drugs for Paroxysmal Nocturnal Hemoglobinuria?

Omeros (OMER, OMS906 study drug, Phase 2). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Paroxysmal Nocturnal Hemoglobinuria.

How advanced is the Paroxysmal Nocturnal Hemoglobinuria pipeline?

Of the 1 clinical-stage programme in Paroxysmal Nocturnal Hemoglobinuria, 0 are in Phase 3 or filed, 1 is in mid-stage development and 0 are in early-stage development.

Is anyone else developing a drug for Paroxysmal Nocturnal Hemoglobinuria?

No. OMER is the only US-listed company in the $100M to $20B market cap band with a clinical-stage programme in Paroxysmal Nocturnal Hemoglobinuria, which is why the indication is classified as uncontested.

How this page is built

  • Universe is US-listed drug developers with a $100M to $20B market capitalisation, identified by SEC SIC code 2833, 2834 or 2836 or by a pharmaceutical or biotechnology industry classification, less a reviewed exclusion list of contract research organisations, device and diagnostics makers and diversified generics manufacturers.
  • An asset attributed to three or more different companies is treated as shared standard of care rather than any one company’s programme and excluded. Pembrolizumab alone appears under 21 tickers in raw trial data.
  • A programme counts only where at least one industry-sponsored trial supports it, so investigator-initiated academic studies of third-party drugs are excluded.

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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