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Prader-Willi Syndrome: 4 biotech companies with clinical-stage programmes

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

Competitors4in the clinic
Median market cap$3.6Buniverse median $1.20B
Allocated capital$10.7B$15.1B competing
Phase 3 or filed22 mid, 0 early
Pure plays2this indication only
Capital rank10/299crowding rank 41

Prader-Willi Syndrome is a concentrated bet: only 4 US-listed developers are in the clinic, and the median one is worth $3.6B 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.

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

$10.7B of allocated capital sits in Prader-Willi Syndrome, 10th of 299 indications, and 2 of the 4 companies are pure plays that trade on this indication alone.

Companies developing drugs for Prader-Willi Syndrome 4

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
ACADACADIA PharmaceuticalsCarbetocinPhase 31$4.8BNo3 31 Oct 2026
HRMYHarmony Biosciences HoldingsPitolisantPhase 32$2.4BNo2 31 Dec 2026
RYTMRhythm PharmaceuticalsSetmelanotidePhase 21$7.2BYes1 8 Sep 2026
DRUGBright Minds BiosciencesBMB-101Phase 21$662MYes1No dated event

Sorted by phase, then by market capitalisation. Ticker links open the company profile. RYTM holds 48% of the $15.1B competing in this indication.

Why Prader-Willi Syndrome 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. Prader-Willi Syndrome has 4 competitors and a median of $3.6B.

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.

See every Rare Genetic & Metabolic indication on the map

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Prader-Willi Syndrome pipeline questions

Which biotech companies are developing drugs for Prader-Willi Syndrome?

ACADIA Pharmaceuticals (ACAD, Carbetocin, Phase 3); Harmony Biosciences Holdings (HRMY, Pitolisant, Phase 3); Rhythm Pharmaceuticals (RYTM, Setmelanotide, Phase 2); Bright Minds Biosciences (DRUG, BMB-101, Phase 2). Those are the 4 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Prader-Willi Syndrome.

How advanced is the Prader-Willi Syndrome pipeline?

Of the 4 clinical-stage programmes in Prader-Willi Syndrome, 2 are in Phase 3 or filed, 2 are in mid-stage development and 0 are in early-stage development.

How much is the typical Prader-Willi Syndrome biotech company worth?

The median company developing a drug for Prader-Willi Syndrome is worth $3.6B, against a median of $1.20B across the whole universe. $10.7B of allocated capital sits in the indication, ranking it 10th of 299.

When is the next Prader-Willi Syndrome catalyst?

The next dated catalyst in Prader-Willi Syndrome is RYTM on 8 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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