PTC Therapeutics, Inc.

PTCT

$69.82

-2.22%

2026-09-04 12:30:32 EDT-$1.58

Healthcare

Biotechnology

NASDAQ

Negociación activa

PTC Therapeutics, Inc. (PTCT) is a biopharmaceutical company committed to the research, development, and commercialization of innovative therapies for patients afflicted with ...

Cadena de suministro de PTCT

Aguas arriba

Fundiciones, empaquetado, memoria, obleas, componentes y proveedores de insumos.

Aguas arriba

Alcami Corporation

Aldevron (Danaher Corporation)

MassBiologics (University of Massachusetts Medical School)

Roche/Genentech (F. Hoffmann-La Roche AG)

Ionis Pharmaceuticals (via Akcea Therapeutics)

Merck KGaA (MilliporeSigma)

Thermo Fisher Scientific (Patheon)

Complete Pharma Holdings, LLC

Alcami Corporation

Exclusive commercial manufacturer of Emflaza (deflazacort) tablets and other oral solid dosage forms

Aldevron (Danaher Corporation)

NYSE:DHR

GMP plasmid DNA manufacturing for PTC's gene therapy portfolio (AADC deficiency, Angelman syndrome, Friedreich ataxia programs)

MassBiologics (University of Massachusetts Medical School)

Contract manufacturer of AAV-based gene therapy for Upstaza/Kebilidi (AADC deficiency)

Roche/Genentech (F. Hoffmann-La Roche AG)

SIX:ROG

Development & commercialization partner for Evrysdi (risdiplam) — PTC receives royalties on global net sales

Ionis Pharmaceuticals (via Akcea Therapeutics)

NASDAQ:IONS

License partner for Tegsedi (inotersen) and Waylivra (volanesorsen) commercialization rights in Latin America

Aguas abajo

Clientes, distribuidores, OEM y canales que absorben la producción.

Aguas abajo

Orsini Specialty Pharmacy

Optum Frontier Therapies (UnitedHealth Group)

RareMed Solutions

Genentech (Roche Group)

Akcea Therapeutics (Ionis affiliate)

Novartis AG

NYSE:NVS

Global license and collaboration partner for PTC518 (Votoplam) Huntington's disease program; $1B upfront + up to $1.9B milestones; Novartis assumed global development and commercialization

Orsini Specialty Pharmacy

Lead specialty pharmacy partner for Sephience (sepiapterin) distribution in the US; also limited distribution partner for Emflaza

Optum Frontier Therapies (UnitedHealth Group)

NYSE:UNH

Specialty pharmacy distribution partner for Sephience (sepiapterin) in the US

RareMed Solutions

Non-commercial pharmacy dispensing services for Sephience; expanded concierge patient services partnership

Royalty Pharma plc

NASDAQ:RPRX

Purchased PTC's retained royalty interest in Evrysdi (risdiplam) for $240M upfront in Dec 2025; previously held option to acquire portions of the royalty stream

Comparables

Empresas cotizadas comparables y valores de seguimiento usados como representantes del análisis.

Comparables

BioMarin Pharmaceutical Inc.

Royalty Pharma plc

NASDAQ:RPRX

Direct financial counterparty to PTC's largest royalty asset (Evrysdi); Royalty Pharma's acquisition of PTC's royalty interest provides a valuation benchmark for PTC's royalty monetization strategy.

Ionis Pharmaceuticals, Inc.

NASDAQ:IONS

Partner through Akcea for Tegsedi/Waylivra in Latin America; similar rare disease focused biotech with licensing-based commercial model.

Sarepta Therapeutics, Inc.

NASDAQ:SRPT

Direct competitor in Duchenne muscular dystrophy (DMD) space; Sarepta's gene therapy Elevidys competes with PTC's DMD franchise (Translarna/Emflaza). Comparable rare disease commercial and supply chain model.

BioMarin Pharmaceutical Inc.

NASDAQ:BMRN

Peer rare disease biopharmaceutical company with both small molecule and gene therapy products; similar reliance on CDMO network for manufacturing and specialty pharmacy distribution.

Geografía

Exposiciones regionales que pueden distorsionar la red de suministro.

Geografía

United States

European Union / United Kingdom

Latin America

Orsini Specialty Pharmacy

Optum Frontier Therapies (UnitedHealth Group)

Alcami Corporation

MassBiologics (UMass)

Various EU specialty distributors and hospitals

Akcea Therapeutics (Ionis Pharmaceuticals)

United States

Primary commercial market for Emflaza, Sephience, and Upstaza/Kebilidi; all major CDMO relationships are US-based

Orsini Specialty PharmacySephience and Emflaza specialty distribution

Optum Frontier Therapies (UnitedHealth Group)NYSE:UNHSephience specialty pharmacy distribution

Alcami CorporationExclusive Emflaza manufacturer

European Union / United Kingdom

Key market for Translarna (ataluren) and Sephience; Translarna faces regulatory risk (CHMP reviews, conversion to full authorization)

Various EU specialty distributors and hospitalsTranslarna and Sephience end-market patients via national health systems

Latin America

Commercialization region for Tegsedi and Waylivra under license from Akcea/Ionis; smaller revenue contribution

Akcea Therapeutics (Ionis Pharmaceuticals)NASDAQ:IONSLicense partner for Tegsedi/Waylivra in Latin America

Switzerland (Roche HQ)

Royalty revenue from Evrysdi relies on Roche's global commercial execution; PTC sold majority royalty interest to Royalty Pharma

F. Hoffmann-La Roche AGSIX:ROGSMA collaboration partner; Evrysdi royalty payer

Royalty Pharma plcNASDAQ:RPRXPurchased Evrysdi royalty stream

Concentración

Exposiciones de concentración de clientes, proveedores o socios que pueden distorsionar la red de suministro.

Customer

Critical

FY2025 total revenue ($1.73B) heavily influenced by $1B non-recurring upfront from Novartis PTC518 deal. Excluding collaboration revenue, product and royalty revenue was ~$823M with significant reliance on Translarna ($235M) and Emflaza (~$200M), both for Duchenne muscular dystrophy.

Foundry

Critical

No internal manufacturing capabilities for small molecules — PTC is fully reliant on a small number of CDMOs (single-source for Emflaza via Alcami; single-source for Translarna). Loss of any CDMO would severely disrupt supply.

Equipment

High

Gene therapy manufacturing relies on specialized AAV vector production capacity via MassBiologics and plasmid supply via Aldevron; limited redundancy in this highly specialized supply chain.

Other

High

Translarna revenue declining ($235M in FY2025 vs $321M in FY2024) due to regulatory pressures; NDA resubmission withdrawn in Feb 2026. Future revenue stream uncertain in key EU markets.

Composición de ingresos

Cómo la composición de ingresos se traduce en apalancamiento y dependencia en la cadena de suministro.

Collaboration and License Revenue

57.7%

$1.0B

Driven largely by $1B upfront from Novartis PTC518 deal in Dec 2024; non-recurring. Low ongoing supply chain intensity.

Product Revenue — Translarna (ataluren)

13.6%

$0.2B

Single-source small molecule API & finished drug product manufacturing via CDMOs; declining revenue due to regulatory headwinds and EU market contraction.

Product Revenue — Emflaza (deflazacort)

11.6%

$0.2B

Manufactured under exclusive agreement with Alcami Corporation (single-source CDMO); tablets made in Spain. Stable DMD anchor product.

Product Revenue — Sephience (sepiapterin)

6.5%

$0.1B

Newly launched (July 2025). Rapidly scaling; requires dedicated CDMO capacity for API and oral powder manufacturing. Growth driver for 2026.

Product Revenue — Upstaza/Kebilidi (gene therapy)

2.3%

$0.0B

AAV-based gene therapy; manufactured via MassBiologics and Aldevron (plasmids). Complex cold-chain logistics, low volume high value.

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PTC Therapeutics, Inc.

Cadena de suministro de PTCT

Aguas arriba

Alcami Corporation

Aldevron (Danaher Corporation)

MassBiologics (University of Massachusetts Medical School)

Roche/Genentech (F. Hoffmann-La Roche AG)

Ionis Pharmaceuticals (via Akcea Therapeutics)

Merck KGaA (MilliporeSigma)

Thermo Fisher Scientific (Patheon)

Complete Pharma Holdings, LLC

Aguas abajo

Novartis AG

Orsini Specialty Pharmacy

Optum Frontier Therapies (UnitedHealth Group)

RareMed Solutions

Royalty Pharma plc

Genentech (Roche Group)

Akcea Therapeutics (Ionis affiliate)

Comparables

Royalty Pharma plc

Ionis Pharmaceuticals, Inc.

Sarepta Therapeutics, Inc.

BioMarin Pharmaceutical Inc.

Geografía

United States

European Union / United Kingdom

Latin America

View More

Cadena de suministro de PTCT

Aguas arriba

Alcami Corporation

Aldevron (Danaher Corporation)

MassBiologics (University of Massachusetts Medical School)

Roche/Genentech (F. Hoffmann-La Roche AG)

Ionis Pharmaceuticals (via Akcea Therapeutics)

Merck KGaA (MilliporeSigma)

Thermo Fisher Scientific (Patheon)

Complete Pharma Holdings, LLC

Aguas abajo

Novartis AG

Orsini Specialty Pharmacy

Optum Frontier Therapies (UnitedHealth Group)

RareMed Solutions

Royalty Pharma plc

Genentech (Roche Group)

Akcea Therapeutics (Ionis affiliate)

Comparables

Royalty Pharma plc

Ionis Pharmaceuticals, Inc.

Sarepta Therapeutics, Inc.

BioMarin Pharmaceutical Inc.

Geografía

United States

European Union / United Kingdom

Latin America

View More