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.
Tilikausi 2025 · muutos edellisvuodesta
Kehitys 2020–2025
Liiketoiminta
Osuus liikevaihdostaExon-skipping therapies for Duchenne muscular dystrophy, including EXONDYS 51, VYONDYS 53, and AMONDYS 45.
ELEVIDYS, a gene therapy product for Duchenne muscular dystrophy.
Revenue from Roche collaboration, supply, and royalties tied to ELEVIDYS outside the U.S.
Precommercial RNA, siRNA, and gene therapy programs for rare neuromuscular and genetic diseases.
Tuotteet
Raportoitu · osuus liikevaihdosta- Pmo Products51,8 %
- Elevidys48,2 %
Lähde: 10-K FY2025, yhtiön tuotelinjoittain raportoima liikevaihto (SEC XBRL). Osuudet on laskettu näytetyistä riveistä ja ne summautuvat 100 %:iin.
Asiakkaat
Sarepta sells primarily to patients with rare neuromuscular diseases through specialty pharmacy, home infusion, and hospital channels, with reimbursement decisions made by government and private payors. Its commercial products are used by physicians and care teams treating Duchenne muscular dystrophy, while its partnered and precommercial programs are aimed at future patients in additional rare disease indications.
Buyers are patients through their care providers; they use approved PMO therapies and ELEVIDYS to treat Duchenne.
They administer ELEVIDYS and handle infusion-based treatment delivery.
They distribute PMO products to patients and hospitals in the U.S.
They do not buy the drug directly but determine access and timing of payment.
Roche and other distributors support ex-U.S. supply and commercialization of ELEVIDYS.
Strategia
Sarepta’s strategy centers on extending its Duchenne franchise while broadening its RNA and gene-therapy platform into additional rare diseases. It also relies on manufacturing partnerships and collaborations, especially with Roche and Arrowhead, to support clinical development, supply, and future commercialization.
Duchenne is the core commercial base and supports the company’s brand, infrastructure, and cash generation.
New modalities and indications can broaden the addressable market beyond current approved products.
Complex biologics and gene therapies require reliable third-party capacity and quality control.
International approvals and partner-led distribution can extend product reach without building full direct infrastructure everywhere.
Maantiede
Sarepta is headquartered in the United States and generates most commercial activity through U.S. specialty distribution and hospital channels. Outside the U.S., it uses partners and third-party distributors for precommercial access and for ELEVIDYS supply under the Roche collaboration, making international commercialization dependent on local approvals and partner execution.
Riskit
Sarepta depends heavily on a small number of commercial products, so product performance, safety data, reimbursement, and regulatory outcomes can materially affect results. Its gene-therapy and RNA platforms also face manufacturing complexity, supply-chain concentration, intellectual-property disputes, and clinical-development uncertainty typical of rare-disease biopharma.
- Commercial concentration in Duchenne productsVaikutus: KorkeaTodennäköisyys: Korkea
A large share of value depends on a narrow set of approved therapies and one disease area.
Kohdistuu - Regulatory and safety riskVaikutus: KorkeaTodennäköisyys: Korkea
Accelerated approval status and label perception depend on ongoing clinical and safety evidence.
Kohdistuu: Duchenne therapies and gene therapy programs
- Reimbursement and access riskVaikutus: KorkeaTodennäköisyys: Korkea
Specialty drugs require timely payor approval and favorable coverage to convert demand into sales.
Kohdistuu: U.S. managed markets and prior authorization
- Manufacturing and supply-chain disruptionVaikutus: KorkeaTodennäköisyys: Kohtalainen
Gene therapies and PMO products rely on third-party manufacturing, testing, and single-source inputs.
Kohdistuu - Competitive and IP riskVaikutus: KeskitasoTodennäköisyys: Kohtalainen
Other RNA, gene therapy, and gene editing programs may compete in the same rare-disease markets.
Kohdistuu
Kirjanpito
Harkinnanvaraiset erätApproved-product inventory is carried at lower of cost and net realizable value, so excess or quality issues can trigger write-downs.
Vaikutus: Cost of sales and gross margin
Roche-related collaboration, supply, and royalty arrangements create multiple revenue streams with different recognition patterns.
Vaikutus: Revenue timing and mix
CRO contracts create cancellable future commitments that affect liquidity planning and expense visibility.
Vaikutus: Operating expense and cash planning
Tax positions and valuation judgments can materially affect reported tax expense and deferred tax balances.
Vaikutus: Tax provision
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Päivitetty: 24.9.2026