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Phenylketonuria: 4 biotech companies with clinical-stage programmes

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

Competitors4in the clinic
Median market cap$4.0Buniverse median $1.20B
Allocated capital$5.6B$22.2B competing
Phase 3 or filed21 mid, 1 early
Pure plays0this indication only
Capital rank33/299crowding rank 43

Phenylketonuria is a concentrated bet: only 4 US-listed developers are in the clinic, and the median one is worth $4.0B 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.

BMRN carries 58% of the $22.2B competing here, more than the other 3 companies combined.

$5.6B of allocated capital sits in Phenylketonuria, ranking it 33rd of 299 indications by the money committed to it.

Companies developing drugs for Phenylketonuria 4

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
BMRNBioMarin PharmaceuticalPegvaliasePhase 32$12.9BNo5No dated event
PTCTPTC TherapeuticsPTC923Phase 32$6.0BNo3No dated event
MAZEMaze TherapeuticsMZE782Phase 21$1.4BNo2 31 Dec 2027
AGIOAgios PharmaceuticalsAG-181Phase 11$2.0BNo6 31 Dec 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile. BMRN holds 58% of the $22.2B competing in this indication.

Why Phenylketonuria 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. Phenylketonuria has 4 competitors and a median of $4.0B.

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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Phenylketonuria pipeline questions

Which biotech companies are developing drugs for Phenylketonuria?

BioMarin Pharmaceutical (BMRN, Pegvaliase, Phase 3); PTC Therapeutics (PTCT, PTC923, Phase 3); Maze Therapeutics (MAZE, MZE782, Phase 2); Agios Pharmaceuticals (AGIO, AG-181, Phase 1). Those are the 4 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Phenylketonuria.

How advanced is the Phenylketonuria pipeline?

Of the 4 clinical-stage programmes in Phenylketonuria, 2 are in Phase 3 or filed, 1 is in mid-stage development and 1 is in early-stage development.

Which company leads the Phenylketonuria field?

BMRN is the largest developer in Phenylketonuria, holding 58% of the $22.2B of market capitalisation competing in the indication. The median competitor is worth $4.0B.

When is the next Phenylketonuria catalyst?

The next dated catalyst in Phenylketonuria is AGIO on 31 Dec 2026. 1 dated event 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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