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Hereditary Angioedema: 5 biotech companies with clinical-stage programmes

Rare Genetic & MetabolicConcentrated betData as of 2026-09-04

Competitors5in the clinic
Median market cap$2.6Buniverse median $1.20B
Allocated capital$8.3B$29.2B competing
Phase 3 or filed40 mid, 1 early
Pure plays1this indication only
Capital rank21/299crowding rank 31

Hereditary Angioedema is a concentrated bet: only 5 US-listed developers are in the clinic, and the median one is worth $2.6B against a $1.20B universe median.

Scarcity is doing the work in that number. A thin field means an approval is shared with fewer rivals, and the market pays a premium for that position.

4 of the 5 are in Phase 3 or filed, and 3 dated catalysts fall in the next 12 months, so this ranking is likely to move.

$8.3B of allocated capital sits in Hereditary Angioedema, 21st of 299 indications, and 1 of the 5 companies are pure plays that trade on this indication alone.

Companies developing drugs for Hereditary Angioedema 5

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
IONSIonis PharmaceuticalsDonidalorsenPhase 32$9.7BNo9 22 Sep 2026
BCRXBioCryst PharmaceuticalsBerotralstatPhase 32$2.6BYes1No dated event
PHVSPharvarisDeucrictibantPhase 33$2.3BNo2 30 Sep 2026
NTLAIntellia TherapeuticsNTLA-2002Phase 32$1.8BNo2 31 Dec 2026
BMRNBioMarin PharmaceuticalDose 1 of BMN 331Phase 11$12.9BNo5No dated event

Sorted by phase, then by market capitalisation. Ticker links open the company profile. BMRN holds 44% of the $29.2B competing in this indication.

Why Hereditary Angioedema is a concentrated bet

Few competitors, each one large. The market pays a scarcity premium to compete here.

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. Hereditary Angioedema has 5 competitors and a median of $2.6B.

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.

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Hereditary Angioedema pipeline questions

Which biotech companies are developing drugs for Hereditary Angioedema?

Ionis Pharmaceuticals (IONS, Donidalorsen, Phase 3); BioCryst Pharmaceuticals (BCRX, Berotralstat, Phase 3); Pharvaris (PHVS, Deucrictibant, Phase 3); Intellia Therapeutics (NTLA, NTLA-2002, Phase 3); BioMarin Pharmaceutical (BMRN, Dose 1 of BMN 331, Phase 1). Those are the 5 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Hereditary Angioedema.

How advanced is the Hereditary Angioedema pipeline?

Of the 5 clinical-stage programmes in Hereditary Angioedema, 4 are in Phase 3 or filed, 0 are in mid-stage development and 1 is in early-stage development.

How much is the typical Hereditary Angioedema biotech company worth?

The median company developing a drug for Hereditary Angioedema is worth $2.6B, against a median of $1.20B across the whole universe. $8.3B of allocated capital sits in the indication, ranking it 21st of 299.

When is the next Hereditary Angioedema catalyst?

The next dated catalyst in Hereditary Angioedema is IONS on 22 Sep 2026. 3 dated events across the field fall in the next 12 months.

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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