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Charcot-Marie-Tooth Disease: one US-listed biotech with a clinical-stage programme

Neurology & PsychiatryUncontestedData as of 2026-09-04

Competitors1in the clinic
Median market cap$323Muniverse median $1.20B
Allocated capital$46M$323M competing
Phase 3 or filed00 mid, 1 early
Pure plays0this indication only
Capital rank290/299crowding rank 290

Charcot-Marie-Tooth Disease is uncontested. Vanda Pharmaceuticals (VNDA) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with VCA-894A in phase 1.

There is no public comparable to price it against. VNDA spreads its $323M market cap across 7 indications, so $46M of it is allocated here.

Nothing here has reached Phase 3: 0 programmes in mid stage and 1 in early stage, so the field is still several years from a first approval.

$46M of allocated capital sits in Charcot-Marie-Tooth Disease, ranking it 290th of 299 indications by the money committed to it.

Companies developing drugs for Charcot-Marie-Tooth Disease 1

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
VNDAVanda PharmaceuticalsVCA-894APhase 11$323MNo7 12 Dec 2026

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

Why Charcot-Marie-Tooth Disease 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. Charcot-Marie-Tooth Disease has one competitor and a median of $323M.

Neurology & Psychiatry

Neurology & Psychiatry holds $120B of the map's capital, 17.9% of the total, spread across 46 indications and 58 companies. 19 of those indications have more than one company competing.

See every Neurology & Psychiatry indication on the map

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Charcot-Marie-Tooth Disease pipeline questions

Which biotech companies are developing drugs for Charcot-Marie-Tooth Disease?

Vanda Pharmaceuticals (VNDA, VCA-894A, Phase 1). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Charcot-Marie-Tooth Disease.

How advanced is the Charcot-Marie-Tooth Disease pipeline?

Of the 1 clinical-stage programme in Charcot-Marie-Tooth Disease, 0 are in Phase 3 or filed, 0 are in mid-stage development and 1 is in early-stage development.

Is anyone else developing a drug for Charcot-Marie-Tooth Disease?

No. VNDA is the only US-listed company in the $100M to $20B market cap band with a clinical-stage programme in Charcot-Marie-Tooth Disease, which is why the indication is classified as uncontested.

When is the next Charcot-Marie-Tooth Disease catalyst?

The next dated catalyst in Charcot-Marie-Tooth Disease is VNDA on 12 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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