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Facioscapulohumeral Muscular Dystrophy: two biotech companies racing to a treatment

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

Competitors2in the clinic
Median market cap$3.5Buniverse median $1.20B
Allocated capital$3.5B$7.1B competing
Phase 3 or filed11 mid, 0 early
Pure plays0this indication only
Capital rank56/299crowding rank 76

Facioscapulohumeral Muscular Dystrophy is a two-horse race: Atrium Therapeutics (RNA, $250M) against Scholar Rock Holding (SRRK, $6.8B).

A duopoly prices differently from an empty field. Each company is the other's comparable, so a readout from either side repositions both, and $3.5B of allocated capital sits on the pair.

SRRK alone accounts for 97% of the $7.1B of market capitalisation competing here, so this is a leader plus a tail rather than a level race.

$3.5B of allocated capital sits in Facioscapulohumeral Muscular Dystrophy, ranking it 56th of 299 indications by the money committed to it.

Companies developing drugs for Facioscapulohumeral Muscular Dystrophy 2

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
RNAAtrium TherapeuticsAOC 1020Phase 32$250MNo3 31 Dec 2026
SRRKScholar Rock HoldingApitegromabPhase 21$6.8BNo2 30 Sep 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile. SRRK holds 97% of the $7.1B competing in this indication.

Why Facioscapulohumeral Muscular Dystrophy 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. Facioscapulohumeral Muscular Dystrophy has 2 competitors and a median of $3.5B.

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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Facioscapulohumeral Muscular Dystrophy pipeline questions

Which biotech companies are developing drugs for Facioscapulohumeral Muscular Dystrophy?

Atrium Therapeutics (RNA, AOC 1020, Phase 3); Scholar Rock Holding (SRRK, Apitegromab, Phase 2). Those are the 2 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Facioscapulohumeral Muscular Dystrophy.

How advanced is the Facioscapulohumeral Muscular Dystrophy pipeline?

Of the 2 clinical-stage programmes in Facioscapulohumeral Muscular Dystrophy, 1 is in Phase 3 or filed, 1 is in mid-stage development and 0 are in early-stage development.

Which company leads the Facioscapulohumeral Muscular Dystrophy field?

SRRK is the largest developer in Facioscapulohumeral Muscular Dystrophy, holding 97% of the $7.1B of market capitalisation competing in the indication. The median competitor is worth $3.5B.

When is the next Facioscapulohumeral Muscular Dystrophy catalyst?

The next dated catalyst in Facioscapulohumeral Muscular Dystrophy is SRRK on 30 Sep 2026. 2 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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