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NASDAQ · mid · thesis board

BMRN BioMarin

AAV gene therapy for hemophilia A (Roctavian)

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Thesis

Editorial summary — not a primary filing

Roctavian commercial rebuild plus rare disease pipeline cash generation.

Next gate

2026-10-01Company-guidedlabel expand

Commercial update

49 days · Company: Company guidance

Survive-to-Event

ok

Cash covers the next gate with ~17 months spare (curated estimate).

Unverified estimate — not from a live SEC parse

Cash runway (~557d)100%
Days to gate (49d)9%
Show math

$1.1B cash ÷ $60M/mo burn ≈ 557 days. Source: curated SEC quarter figures.

Cash $1.1B as of 2026-03-31 (SEC)

Pipeline spine

  1. Preclinical
  2. Ph1
  3. Ph2
  4. Ph3
  5. Filed
  6. Approved

Rivals in this race

Open race →
PFEApprovedokNVOFiledokSGMOPh3tight

Risks

  • Gene therapy uptake
  • Competing mimetics
  • Pricing
  • Pipeline gaps

Source trail

  1. SECCash from latest reported quarter (curated)as of 2026-03-31
  2. ClinicalTrials.govNCT03392974as of 2026-07-01
  3. CompanyIR / press disclosuresas of 2026-07-01