Sarepta Therapeutics, Inc.

Sarepta Therapeutics is a U.S.-based commercial-stage biopharmaceutical company focused on RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy, and other genetic medicine modalities for rare diseases. Its portfolio includes approved Duchenne muscular dystrophy products and a pipeline spanning Duchenne, LGMD, and other neuromuscular and genetic disorders.

−29,8 %

98,8 %

−32,5 %

+15,6 %

2.32

1.48

— Sarepta Therapeutics, Inc.
%
Duchenne PMO therapies55% Exon-skipping therapies for Duchenne muscular dystrophy, including EXONDYS 51, VYONDYS 53, and AMONDYS 45.
Duchenne gene therapy35% ELEVIDYS, a gene therapy product for Duchenne muscular dystrophy.
Collaboration and royalty revenue8% Revenue from Roche collaboration, supply, and royalties tied to ELEVIDYS outside the U.S.
Pipeline and partnered programs2% Precommercial RNA, siRNA, and gene therapy programs for rare neuromuscular and genetic diseases.

Sarepta sells primarily to patients with rare neuromuscular diseases through specialty pharmacy, home infusion, and...

  • Duchenne muscular dystrophy patientsprimary

    Buyers are patients through their care providers; they use approved PMO therapies and ELEVIDYS to treat Duchenne.

  • Hospitals and infusion centersprimary

    They administer ELEVIDYS and handle infusion-based treatment delivery.

  • Specialty pharmacies and distributorsprimary

    They distribute PMO products to patients and hospitals in the U.S.

  • Payors and reimbursement systemsprimary

    They do not buy the drug directly but determine access and timing of payment.

  • International partner channelssecondary

    Roche and other distributors support ex-U.S. supply and commercialization of ELEVIDYS.

Sarepta is headquartered in the United States and generates most commercial activity through U.S...

  • •Headquartered in the United States
  • •U.S. sales run through specialty pharmacy and hospital channels
  • •Ex-U.S. access relies on third-party distributors and partners
  • •Roche supplies and commercializes ELEVIDYS in approved territories
  • •International expansion depends on local regulatory approvals

Sarepta’s strategy centers on extending its Duchenne franchise while broadening its RNA and gene-therapy platform into...

01
Grow the Duchenne franchiseshort-term

Duchenne is the core commercial base and supports the company’s brand, infrastructure, and cash generation.

02
Advance gene therapy and RNA pipelinemedium-term

New modalities and indications can broaden the addressable market beyond current approved products.

03
Scale manufacturing and supply partnershipsshort-term

Complex biologics and gene therapies require reliable third-party capacity and quality control.

04
Expand ex-U.S. commercializationmedium-term

International approvals and partner-led distribution can extend product reach without building full direct infrastructure everywhere.

Sarepta depends heavily on a small number of commercial products, so product performance, safety data, reimbursement,...

high

Commercial concentration in Duchenne products

A large share of value depends on a narrow set of approved therapies and one disease area.

Scope
EXONDYS 51, VYONDYS 53, AMONDYS 45, ELEVIDYS
Materiality
high
high

Regulatory and safety risk

Accelerated approval status and label perception depend on ongoing clinical and safety evidence.

Scope
Duchenne therapies and gene therapy programs
Materiality
high
high

Reimbursement and access risk

Specialty drugs require timely payor approval and favorable coverage to convert demand into sales.

Scope
U.S. managed markets and prior authorization
Materiality
high
high

Manufacturing and supply-chain disruption

Gene therapies and PMO products rely on third-party manufacturing, testing, and single-source inputs.

Scope
Contract manufacturers, raw materials, logistics
Materiality
high
medium

Competitive and IP risk

Other RNA, gene therapy, and gene editing programs may compete in the same rare-disease markets.

Scope
Duchenne, LGMD, FSHD, DM1, SCA, IPF
Materiality
medium
Inventory valuation
Cost of sales and gross margin
Collaboration and royalty revenue
Revenue timing and mix
Clinical trial commitments
Operating expense and cash planning
Income tax estimates
Tax provision

Entrada Therapeutics, Inc.

TRDA · Pharmaceutical Preparations

CAPRICOR THERAPEUTICS, INC.

CAPR · Pharmaceutical Preparations

Stoke Therapeutics, Inc.

STOK · Pharmaceutical Preparations

Solid Biosciences Inc.

SLDB · Biological Products, (No Diagnostic Substances)

Ultragenyx Pharmaceutical Inc.

RARE · Pharmaceutical Preparations

ARROWHEAD PHARMACEUTICALS, INC.

ARWR · Pharmaceutical Preparations

: 29/04/2026