PTCTNasdaqPharmaceutical PreparationsUS GAAP · USD

PTC Therapeutics, Inc

PTC Therapeutics is a U.S.-based global biopharmaceutical company focused on discovering, developing, and commercializing medicines for rare disorders. Its portfolio includes approved therapies and clinical-stage programs for neurology and metabolism, with commercialization across the United States, Europe, Latin America, and other international markets through direct operations and partners.

Kysy yhtiöstä
Liikevaihto1 731milj. USD
+114,5 %v/v
Liikevoitto (EBIT)867milj. USD
+386,5 %v/v
EBIT-marginaali50,1%
+87,6 %-yks.v/v
Nettotulos683milj. USD
+287,9 %v/v
Vapaa kassavirta702milj. USD
+715,1 %v/v

Tilikausi 2025 · muutos edellisvuodesta

Kehitys 2020–2025

−50005001 0001 5002 000milj. USD2020: 381 milj. USD2020: EBIT −432 milj. USD20202021: 539 milj. USD2021: EBIT −374 milj. USD20212022: 699 milj. USD2022: EBIT −447 milj. USD20222023: 938 milj. USD2023: EBIT −440 milj. USD20232024: 807 milj. USD2024: EBIT −303 milj. USD20242025: 1 731 milj. USD2025: EBIT 867 milj. USD20251 731867
LiikevaihtoLiikevoitto (EBIT)
Kysy Clarifolta: PTC Therapeutics, Inc

Liiketoiminta

Osuus liikevaihdosta
Commercial rare-disease medicines85 %

Approved therapies sold in the U.S., Europe, Latin America, and other territories.

Early access and named-patient programs5 %

Access programs used to supply medicines in markets without full commercial launch.

Milestone and royalty revenue5 %

Collaborative revenue tied to partnered programs such as SMA-related agreements.

Pipeline development programs5 %

Research, discovery, and clinical-stage programs in neurology and metabolism.

Tuotteet

Raportoitu · osuus liikevaihdosta
54,6%13,4%12,9%FY2025100 %
  • Collaboration and License Revenue54,6 %
  • Royalty13,4 %
  • Translarna112,9 %
  • Emflaza8,0 %
  • Sephience6,1 %
  • Muut5,1 %

Lähde: 10-K FY2025, yhtiön tuotelinjoittain raportoima liikevaihto (SEC XBRL). Osuudet on laskettu näytetyistä riveistä ja ne summautuvat 100 %:iin.

Asiakkaat

PTC Therapeutics sells primarily to patients with rare diseases through specialty pharmacies, hospitals, distributors, and reimbursement systems rather than to mass-market consumers. The end payer is often a government health program or third-party insurer, so access depends on pricing, reimbursement, and regulatory authorization in each market.

Rare disease patients and caregivers

Patients with DMD, PKU, AADC deficiency, hATTR amyloidosis, and FCS who receive treatment through prescribed specialty channels.

Specialty pharmacies

U.S. dispensing partners that receive prescriptions, manage fulfillment, and ship products directly to patients.

Government and public health payers

State programs and national health systems that reimburse high-cost orphan medicines and determine access.

Third-party insurers

Commercial and managed-care payers that cover specialty drugs and influence net realized pricing.

Hospitals, distributors, and named-patient channels

European and international channels that buy for reimbursed access, compassionate use, or early access programs.

Strategia

PTC Therapeutics is focused on expanding its rare-disease franchise by commercializing approved products and advancing a diversified pipeline in neurology and metabolism. It also uses partnerships, early-access channels, and global infrastructure to broaden patient access while pursuing new approvals and label opportunities.

Commercialize approved orphan medicines globallyLyhyt aikaväli

Approved products generate the core revenue base and fund development.

Expand access through reimbursement and partner channelsLyhyt aikaväli

Rare-disease drugs depend on payer approval and local distribution infrastructure.

Advance pipeline programs in neurology and metabolismKeskipitkä aikaväli

Pipeline breadth supports future growth beyond current products.

Pursue new indications and lifecycle opportunitiesKeskipitkä aikaväli

Additional labels can extend product longevity and broaden addressable patients.

Maantiede

Q1/2026

PTC Therapeutics operates globally, with commercial activity in the United States, the EEA, Latin America, and selected other territories. The company relies heavily on international markets for several products, while U.S. commercialization is important for Emflaza and Sephience and Europe is important for Translarna and Upstaza/Kebilidi.

USA75 %
Brazil23 %
Venäjä1,8 %

Osuus liikevaihdosta, jonka yhtiö raportoi maantieteellisesti.

Riskit

PTC Therapeutics depends on maintaining marketing authorizations, reimbursement, and supply for a concentrated set of rare-disease products, so regulatory or payer setbacks can quickly affect revenue. Its international footprint also adds exposure to country-specific pricing, reimbursement, and order timing volatility, while pipeline development and financing needs create typical biotech execution risk.

Järjestetty vakavuuden mukaan
MatalaKohtalainenKorkeaMatalaKohtalainenKorkeaVaikutus →Todennäköisyys →Marketing authorization renewal and compliance risk1Reimbursement and pricing pressure2International concentration and country-specific volatility3Third-party manufacturing and supply chain dependence4Clinical development and financing risk5
Vaikutus on riskin vakavuus ja todennäköisyys sen toteutumisen todennäköisyys lähivuosina, molemmat arvioitu yhtiön raporttien perusteella. Numerot vastaavat listaa.
  1. Marketing authorization renewal and compliance riskVaikutus: KorkeaTodennäköisyys: Korkea

    Several products depend on maintaining approvals and meeting local conditions.

    KohdistuuTranslarnaTegsediWaylivraUpstaza/Kebilidi
  2. Reimbursement and pricing pressureVaikutus: KorkeaTodennäköisyys: Korkea

    Rare-disease medicines require payer coverage and acceptable pricing to drive uptake.

    Kohdistuu: U.S. specialty pharmacy sales and international market access

  3. International concentration and country-specific volatilityVaikutus: KeskitasoTodennäköisyys: Korkea

    A substantial portion of sales occurs outside the United States and can be uneven by quarter.

    KohdistuuEuropeBrazilLatin AmericaNamed-patient markets
  4. Third-party manufacturing and supply chain dependenceVaikutus: KeskitasoTodennäköisyys: Kohtalainen

    Commercial supply relies on external manufacturers and logistics partners.

    KohdistuuSephienceTranslarnaVatiquinoneOther programs
  5. Clinical development and financing riskVaikutus: KeskitasoTodennäköisyys: Kohtalainen

    Pipeline programs require successful trials and ongoing capital to advance.

    KohdistuuResearchDiscoveryClinical-stage rare-disease assets
Kysy Clarifolta: PTC Therapeutics, Inc

Kirjanpito

Harkinnanvaraiset erät
Net product revenue and variable consideration

Revenue is recognized net of estimated rebates, discounts, and allowances, which requires judgment.

Vaikutus: Can materially change reported product sales and quarterly comparability

Quarterly order timing in international markets

Named-patient and centralized purchases may cover multiple months of therapy.

Vaikutus: Creates uneven revenue recognition across quarters

Milestone and royalty accounting

Collaborative revenue from partners such as Roche and others depends on contractual triggers.

Vaikutus: Can create non-recurring or lumpy revenue streams

Convertible debt and royalty financing

Interest expense includes convertible notes and royalty-related liabilities.

Vaikutus: Affects financing costs and balance sheet presentation

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Päivitetty: 24.9.2026

PTC Therapeutics, Inc (PTCT) liikevaihto, tulos ja tunnusluvut 2025 – Clarifo