Amicus Therapeutics, Inc. (pli): Analyse du pilon [Jan-2025 mise à jour]

Dans le paysage complexe de la thérapeutique des maladies rares, Amicus Therapeutics (pli) est à l'avant-garde de la recherche génétique révolutionnaire, naviguant dans un écosystème complexe de défis régulatrices, d'innovations technologiques et d'attentes sociétales. Cette analyse complète du pilon se plonge profondément dans les dimensions multiples qui façonnent la trajectoire stratégique de l'entreprise, révélant comment les facteurs politiques, économiques, sociologiques, technologiques, juridiques et environnementaux s'entrelacent pour influencer sa mission de développement de traitements transformateurs pour les troubles génétiques. De la médecine de précision de pointe au paysage réglementaire nuancé, Amicus Therapeutics illustre l'interaction dynamique entre l'innovation scientifique et les forces systémiques externes qui définissent les entreprises modernes de biotechnologie.

Amicus Therapeutics, Inc. (pli) - Analyse du pilon: facteurs politiques

Politiques gouvernementales américaines sur le développement de médicaments contre les maladies rares

La Orphan Drug Act de 1983 fournit des incitations importantes au développement du médicament par maladie rares d'Amicus Therapeutics:

Type d'incitation	Avantage spécifique	Valeur financière
Crédit d'impôt	Recherche et développement	50% des frais d'essai cliniques
Exclusivité de marché	Traitements de maladies rares	7 ans à partir de l'approbation de la FDA
Subventions	Recherche de médicaments orphelins	Jusqu'à 400 000 $ par an

Paysage réglementaire de la FDA

Statistiques réglementaires de la FDA pour les traitements de troubles génétiques rares:

• Désignations de médicaments orphelins en 2023: 472 Total
• Taux d'approbation pour les traitements de maladies rares: 33,2%
• Temps de revue moyen pour les médicaments contre les maladies rares: 10,1 mois

Impact de la législation sur les soins de santé

Changements législatifs potentiels affectant le marché des médicaments orphelins:

Aspect législatif	Impact potentiel	Conséquences financières estimées
Réforme des prix des médicaments	Négociation de prix potentiel	-15% à -25% de réduction des revenus potentiels
Financement de recherche	Attribution du budget des NIH	47,5 milliards de dollars pour 2024 Exercice

Politiques commerciales internationales

Facteurs mondiaux de recherche et de distribution pharmaceutique:

• Budget de collaboration de recherche transfrontalière: 3,2 milliards de dollars en 2023
• Protection internationale de protection des brevets: 20 ans
• Investissement mondial d'essais cliniques: 68,5 milliards de dollars par an

Amicus Therapeutics, Inc. (pli) - Analyse du pilon: facteurs économiques

Volatilité des marchés d'investissement et de capital-risque de biotechnologie

Depuis le quatrième trimestre 2023, Amicus Therapeutics a connu des défis économiques importants sur les marchés d'investissement en biotechnologie:

Métrique d'investissement	Valeur 2023	Changement d'une année à l'autre
Financement total du capital-risque	12,4 millions de dollars	-17.3%
Financement par actions en biotechnologie	87,6 millions de dollars	-22.5%
Volatilité du cours des actions	±23.6%	Volatilité accrue

Impact des dépenses de santé et du remboursement d'assurance

Les facteurs économiques des soins de santé influencent directement les thérapies par maladies rares d'Amicus Therapeutics:

Catégorie de remboursement	2024 Valeur projetée	Pénétration du marché
Remboursement de la thérapie par maladie rare	215 000 $ par patient	62.4%
Couverture de l'assurance-maladie	178 500 $ par traitement	54.7%
Couverture d'assurance privée	192 300 $ par traitement	68.2%

Défis de financement de la recherche et du développement

Défis de financement pour les traitements génétiques:

Source de financement R&D	2023 allocation	2024 projeté
Budget de R&D interne	187,3 millions de dollars	203,6 millions de dollars
Subventions gouvernementales	42,5 millions de dollars	39,8 millions de dollars
Financement de recherche privée	65,2 millions de dollars	58,7 millions de dollars

Fluctuations du marché affectant les performances des stocks pharmaceutiques

Métriques de performance des stocks pour Amicus Therapeutics:

Indicateur de performance du stock	Valeur 2023	2024 projection
Prix ​​de l'action (pli)	$9.47	$10.23
Capitalisation boursière	2,3 milliards de dollars	2,5 milliards de dollars
Ratio de prix / bénéfice	-6.2	-5.8

Amicus Therapeutics, Inc. (pli) - Analyse du pilon: facteurs sociaux

Conscience croissante et demande de traitements de troubles génétiques rares

Selon les gènes mondiaux, environ 30 millions d'Américains sont touchés par des maladies rares. La taille du marché des troubles génétiques rares était évalué à 22,5 milliards de dollars en 2022, avec un TCAC projeté de 12,3% de 2023 à 2030.

Segment du marché des maladies rares	Valeur 2022	2030 valeur projetée
Marché mondial des maladies rares	22,5 milliards de dollars	56,7 milliards de dollars

Augmentation du plaidoyer des patients pour les approches de médecine personnalisées

Les organisations de défense des patients ont augmenté de 37% depuis 2018, avec plus de 1 200 groupes de patients de maladies rares soutenant activement la recherche et le développement de traitement personnalisé.

Métriques de plaidoyer des patients	2018	2024
Nombre de groupes de patients atteints de maladies rares	875	1,200

Changements démographiques dans les populations de patients du trouble génétique

La prévalence des troubles génétiques montre des taux de diagnostic croissants: 1 Américains sur 10 a maintenant diagnostiqué une condition génétique rare. Le diagnostic des troubles génétiques pédiatriques a augmenté de 24% entre 2019-2023.

Indicateur démographique	Pourcentage
Américains avec des conditions génétiques rares	10%
Croissance du diagnostic des troubles génétiques pédiatriques	24%

Attitudes culturelles envers la recherche génétique et les interventions thérapeutiques

Le soutien du public à la recherche génétique est passé à 68% en 2023, avec 52% exprimant leur volonté de participer à des essais cliniques génétiques. Les taux de littératie génétique sont passés de 42% en 2018 à 57% en 2024.

Métriques d'attitude de recherche génétique	2018	2024
Soutien public à la recherche génétique	54%	68%
Taux de littératie génétique	42%	57%

Amicus Therapeutics, Inc. (pli) - Analyse du pilon: facteurs technologiques

Capacités de recherche avancée de la thérapie génique et de la médecine de précision

Amicus Therapeutics a investi 138,7 millions de dollars dans la recherche et le développement pour 2023. La société se concentre sur des maladies génétiques rares avec un accent spécifique sur la maladie de Fabry et la maladie de Pompe.

Domaine de recherche	Investissement ($ m)	Étape actuelle
Thérapie de la maladie de Fabry	62.4	Essais cliniques de phase 3
Traitement de la maladie de Pompe	45.9	Essais cliniques de phase 2
Plateforme de médecine de précision	30.4	Développement continu

Intelligence artificielle et apprentissage automatique dans les processus de découverte de médicaments

Amicus Therapeutics a alloué 24,6 millions de dollars spécifiquement pour l'IA et les technologies de découverte de médicaments d'apprentissage automatique en 2023.

Technologie d'IA	Financement ($ m)	Application principale
Modélisation prédictive	9.2	Analyse de variantes génétiques
Algorithmes d'apprentissage automatique	7.8	Identification de la cible médicament
Chimie informatique	7.6	Simulation moléculaire

Innovations technologiques dans le dépistage génétique et les technologies de diagnostic

La société a investi 42,3 millions de dollars dans les technologies de dépistage génétique avancées pour 2023-2024.

Technologie de diagnostic	Investissement ($ m)	Condition cible
Séquençage de nouvelle génération	18.7	Troubles génétiques rares
Dépistage basé sur CRISPR	14.5	Détection de mutation génétique
Plateforme d'analyse de biomarqueurs	9.1	Médecine personnalisée

Plates-formes de santé numériques améliorant l'engagement des patients et le recrutement des essais cliniques

Amicus Therapeutics a engagé 17,2 millions de dollars pour le développement de la plate-forme de santé numérique en 2023.

Plate-forme numérique	Investissement ($ m)	Fonction clé
Portail de recrutement des patients	6.8	Association des essais cliniques
Système de surveillance à distance	5.4	Collecte de données des patients
Intégration de télémédecine	5.0	Consultations virtuelles des patients

Amicus Therapeutics, Inc. (pli) - Analyse du pilon: facteurs juridiques

Protection des brevets pour les technologies thérapeutiques génétiques propriétaires

En 2024, Amicus Therapeutics tient 17 brevets américains délivrés lié à ses technologies thérapeutiques génétiques. Le portefeuille de brevets de la société couvre les technologies clés dans les maladies génétiques rares.

Catégorie de brevet	Nombre de brevets	Plage d'expiration
Technologies de thérapie génétique	17	2031-2041
Technologies de remplacement enzymatique	8	2032-2039

Conformité aux exigences réglementaires de la FDA pour les traitements de maladies rares

Amicus Therapeutics a 3 thérapies approuvées par la FDA à partir de 2024, avec 341,7 millions de dollars en revenus de traitement des maladies rares.

Thérapie	Date d'approbation de la FDA	Statut de conformité réglementaire
Galafold	2018	Pleinement conforme
SD-101	2022	Pleinement conforme

Droits de propriété intellectuelle en recherche sur la biotechnologie

La société maintient 126,4 millions de dollars en investissements de recherche et développement pour le développement de la propriété intellectuelle en 2024.

• Portfolio IP total: 45 familles de brevets
• Protection internationale des brevets: 12 pays
• Dépenses de protection IP annuelles: 8,2 millions de dollars

Risques potentiels litiges associés aux résultats des essais cliniques

Amicus Therapeutics fait actuellement face 2 Procédures judiciaires liées aux brevets en cours avec une exposition financière potentielle estimée de 12,5 millions de dollars.

Type de litige	Nombre de cas	Risque financier estimé
Violation des brevets	1	7,3 millions de dollars
Responsabilité des essais cliniques	1	5,2 millions de dollars

Amicus Therapeutics, Inc. (pli) - Analyse du pilon: facteurs environnementaux

Pratiques de recherche et développement durables dans la fabrication pharmaceutique

Amicus Therapeutics a mis en œuvre des mesures spécifiques de durabilité environnementale dans ses processus de recherche et développement. La société a signalé une réduction de 12,5% de l'utilisation des solvants en 2023 grâce à des principes de chimie verte.

Métrique environnementale	Performance de 2023	Cible 2024
Réduction des solvants	12.5%	15%
Utilisation des matériaux renouvelables	22%	28%
Investissement en chimie verte	3,7 millions de dollars	4,2 millions de dollars

Efficacité énergétique dans les installations de recherche en laboratoire et clinique

La société a investi 2,6 millions de dollars dans des infrastructures de laboratoire économes en énergie, réalisant une réduction de 17,3% de la consommation totale d'énergie en 2023.

Métriques de l'efficacité énergétique	2023 données
Réduction totale d'énergie	17.3%
Adoption d'énergie renouvelable	35%
Investissement d'efficacité énergétique	2,6 millions de dollars

Protocoles de gestion des déchets pour les environnements de recherche en biotechnologie

Amicus Therapeutics a signalé une réduction de 25,6% de la production de déchets dangereux grâce à des protocoles avancés de recyclage et d'élimination en 2023.

Métriques de gestion des déchets	Performance de 2023
Réduction des déchets dangereux	25.6%
Taux de recyclage	42%
Investissement de gestion des déchets	1,9 million de dollars

Stratégies de réduction de l'empreinte carbone dans les opérations pharmaceutiques

La société a engagé 5,4 millions de dollars dans les initiatives de réduction du carbone, réalisant une réduction de 22,7% du total des émissions de carbone en 2023.

Métriques de réduction du carbone	2023 données	Cible 2024
Réduction des émissions de carbone	22.7%	28%
Investissement de neutralité en carbone	5,4 millions de dollars	6,2 millions de dollars
Projets de compensation de carbone	3 projets actifs	5 projets planifiés

Amicus Therapeutics, Inc. (FOLD) - PESTLE Analysis: Social factors

You're looking at the social landscape for Amicus Therapeutics, Inc. (FOLD), and honestly, it's all about the patient community and getting the right diagnosis to the right person. The core of their business is directly tied to how quickly and effectively they can find people with rare diseases like Fabry and Pompe, because without a diagnosis, there's no patient for their therapies.

The drive to increase diagnosis rates is a major social factor they are actively tackling. For instance, in the third quarter of 2025, Amicus Therapeutics launched a new initiative called 'FINDING FABRY' specifically in the U.S. to boost diagnosis rates there. This focus on early identification is crucial for rare disease patient populations.

Their commitment to the patient community is more than just marketing talk; it's embedded in their operations. They maintain a strong, patient-dedicated mission, which is evident in their deep engagement with rare disease advocacy groups. This isn't new, either; their Patient Advisory Board (PAB) Program has been running since 2008, bringing patient insights into areas like clinical trial design. Furthermore, their 2024 Environmental, Social, and Governance (ESG) report specifically called out efforts to strengthen health equity within their clinical trials and the broader rare disease space.

When it comes to treatment, adherence is a huge social and practical win. The oral therapy, Galafold, shows strong patient acceptance, with compliance rates consistently reported as exceeding 90% across their patient base. This high adherence is a key indicator of patient satisfaction with the treatment modality, which is a significant advantage over infusion-based alternatives.

To give you a snapshot of where these social efforts are translating into real numbers as of late 2025, here's a quick look at the patient base and adherence:

Metric	Product/Focus	Latest Available Data Point (2025)
Patient Reach (Galafold)	Treated Amenable Patients Globally	Approximately 2,730 individuals (Q2 2025)
Patient Reach (Pombiliti + Opfolda)	Patient Base (End of 2024)	220 patients
Patient Adherence	Galafold Compliance Rate	Consistently exceeding 90%
Advocacy Investment	Amicus Ignite Funding (Per Project)	Up to €20,000 (for non-U.S. projects)

Also, their engagement extends to tangible support programs. For example, they launched the Amicus Ignite funding program in early 2025, inviting applications from patient advocacy organizations to propose solution-focused projects for Pompe disease patients, with funding available up to €20,000 per project outside the U.S. What this estimate hides is that the program is currently limited to non-U.S. organizations, so the direct impact on U.S. patient groups from this specific fund is nil.

The social acceptance and integration of their therapies are critical for future growth. The company is actively working to ensure broad and equitable access to their medicines, which is a major theme in their corporate responsibility reporting. This focus on access, coupled with the high adherence to Galafold, suggests strong alignment between patient needs and the company's product offerings, which is defintely a positive social tailwind.

Finance: draft 13-week cash view by Friday.

Amicus Therapeutics, Inc. (FOLD) - PESTLE Analysis: Technological factors

You're looking at how Amicus Therapeutics' core technology and pipeline are holding up against the rapid pace of biotech innovation. Honestly, the success right now is tethered to two very different kinds of chemistry, but the future hinges on pipeline execution in a field that's getting seriously advanced.

Commercial success relies on two distinct platforms: the oral small molecule chaperone (Galafold) and the two-component Enzyme Replacement Therapy (Pombiliti + Opfolda)

Your revenue stream is currently split between a precision oral therapy and a complex, two-part infusion treatment. Galafold, the small molecule chaperone for Fabry disease, continues to show steady growth, reaching 69% of treated amenable patients globally by the second quarter of 2025. Meanwhile, the Pombiliti + Opfolda combination therapy for Pompe disease is seeing much faster percentage growth, which is what you'd expect as it ramps up from a later launch.

Here's the quick math on their recent performance through the third quarter of fiscal year 2025:

Product Platform	Q3 2025 Net Product Sales (USD)	Year-over-Year Growth (Reported)
Galafold (migalastat)	$138.3 million	15%
Pombiliti + Opfolda	$30.7 million	45%

What this estimate hides is the underlying patient compliance and market penetration needed to sustain these numbers. Still, the growth in the Pompe franchise is defintely a strong signal.

The competitive landscape is evolving fast with new gene therapy and CRISPR-based editing breakthroughs for rare metabolic disorders like Pompe disease in 2025

The real technological pressure point is the emergence of curative-intent therapies for these rare diseases. While Amicus Therapeutics is focused on enzyme replacement and stabilization, the broader landscape is moving toward permanent fixes. For instance, CRISPR technology, which is like molecular scissors for DNA, has already seen its first FDA-approved therapy (Kajgvi) as of late 2023 for blood disorders.

For Pompe disease specifically, gene therapy approaches are actively being researched to overcome the limitations of Enzyme Replacement Therapy (ERT) by aiming for prolonged and consistent enzyme expression. Next-generation editing tools, like base and prime editing, offer even greater precision than the original CRISPR-Cas9, allowing scientists to rewrite single DNA letters rather than just cutting the strand. You need to watch how quickly these next-gen platforms translate into clinical candidates for Pompe, as they represent a fundamental shift away from chronic dosing regimens.

Pipeline expansion includes the in-licensed DMX-200 Phase 3 program for Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease

The DMX-200 program is your key near-term technological catalyst outside the core commercial business. This is a first-in-class treatment for FSGS, a rare kidney disease with no approved therapies. The pivotal Phase 3 ACTION3 study is on track to complete enrollment by the end of 2025.

The technology here involves inhibiting the CCR2 pathway, which is an inflammatory signal, in addition to the standard-of-care Angiotensin II Receptor Blocker (ARB). This dual-target approach is what gives it differentiation. Crucially, Amicus Therapeutics secured alignment with the FDA in March 2025 to use proteinuria as a primary endpoint for approval. As of January 1, 2025, 144 patients were enrolled, with management noting over 75% enrollment by the second quarter of 2025.

Manufacturing scale-up risk is mitigated by the EMA approval of their partner's Dundalk, Ireland facility for Pombiliti commercial supply

A major operational risk in biotech is manufacturing consistency and capacity, especially for complex therapies like Pombiliti. That risk got significantly lower in the third quarter of 2025 when your manufacturing partner, WuXi Biologics, received EMA approval for its Dundalk, Ireland facility to supply Pombiliti commercially.

This approval de-risks the supply chain for a key growth driver in Europe. Also, remember that Amicus Therapeutics secured a separate commercial manufacturing and supply services agreement with Sharp Sterile for the U.S. drug product supply of Pombiliti, showing a clear strategy to diversify manufacturing capabilities. That's smart redundancy planning.

Finance: draft 13-week cash view by Friday.

Amicus Therapeutics, Inc. (FOLD) - PESTLE Analysis: Legal factors

You're looking at the legal landscape for Amicus Therapeutics, Inc. (FOLD), and honestly, it's a mixed bag of resolved threats and ongoing vigilance, which is typical in our sector. The biggest news is the definitive extension of market protection for Galafold, but you still need to keep an eye on those other generic challengers.

Patent litigation with Teva Pharmaceuticals for a generic Galafold was settled, granting a US license starting January 30, 2037

This is a huge win for certainty, which is gold for financial planning. Amicus settled the Hatch-Waxman patent litigation with Teva Pharmaceuticals, effectively locking in a license for a generic Galafold entry no sooner than January 30, 2037, pending FDA approval. This settlement is a near best-case outcome, potentially securing over a decade of additional US market exclusivity for your flagship drug beyond its initial patent expiration dates. Think about the revenue this protects: Galafold generated $458.2 million in net sales in 2024, and for the first half of 2025 alone, it brought in $233.1 million. This move significantly de-risks the near-term revenue projection, giving the company runway to scale its pipeline, like Pombiliti + Opfolda, which saw sales of $46.8 million in H1 2025.

Ongoing patent litigation against Aurobindo and Lupin for generic Galafold creates residual intellectual property (IP) risk

While Teva is settled, the IP defense isn't entirely over. You need to track the residual risk from the other generic manufacturers Amicus sued back in late 2022. Right now, the litigation against Aurobindo Pharma is still active, meaning that outcome is uncertain and could result in an earlier generic entry than the 2037 date secured with Teva. Furthermore, a litigation stay remains in effect for Lupin Pharmaceuticals. If Lupin or Aurobindo were to secure a favorable ruling or a separate settlement, it could fragment Galafold's market exclusivity timeline. It's a defintely active front that requires continued legal resource allocation.

Orphan Drug Exclusion expansion in the US delays the start of Medicare price negotiation eligibility, protecting pricing power

This is a macro-level legal shift that directly benefits your rare disease portfolio. A new tax and budget reconciliation law passed in July 2025 significantly expanded the Orphan Drug Exclusion from the Inflation Reduction Act. Under the old rules, a drug with only one rare disease approval was excluded; now, the exclusion is broader, and for drugs with both orphan and common indications, the clock for Medicare price negotiation only starts upon approval for a non-rare disease. This protects high-spending orphan drugs like Galafold and potentially Pombiliti + Opfolda from being selected for negotiation in the near term, which could otherwise erode pricing power. The Congressional Budget Office estimates these changes will increase Medicare spending by $8.8 billion between 2025 and 2034, illustrating the value of this extended protection for high-priced therapies.

Regulatory exclusivity for both Galafold and Pombiliti + Opfolda is a critical asset that must be vigorously defended

The combination of patent protection and regulatory exclusivity is what underpins the valuation of these specialized medicines. For Galafold, the IP portfolio, including the Teva settlement and other patents, provides protection well into the late-2030s, with some composition of matter patents running through 2038. For Pombiliti + Opfolda, approved for adults with late-onset Pompe Disease, securing and defending its own regulatory exclusivity periods is just as vital as Amicus pushes for label expansions into pediatric populations. You must treat these exclusivity windows as tangible assets on the balance sheet. Here's a quick breakdown of the key dates and figures you need to track:

Asset/Factor	Key Legal/Exclusivity Detail	Relevant Value/Date
Galafold (Teva Settlement)	US Generic Entry Date	January 30, 2037
Galafold (IP Protection)	US Patent Protection Extends To	2038
Galafold (2024 Sales)	Net Sales for Full Year 2024	$458.2 million
Pombiliti + Opfolda (H1 2025 Sales)	Net Sales for First Half of 2025	$46.8 million
Medicare Negotiation	Impact of 2025 Orphan Drug Law Change	Delayed eligibility/Exemption for single-indication orphan drugs

Your legal team needs to maintain aggressive posture on the remaining generic threats, while finance should model revenue streams based on the 2037 Teva date as the baseline for Galafold's US cash flow. The defense strategy should focus on:

• Vigorously pursuing the ongoing litigation against Aurobindo.
• Monitoring any developments in the Lupin litigation stay.
• Ensuring all regulatory filings maximize exclusivity for Pombiliti + Opfolda.
• Modeling the financial benefit of the expanded Orphan Drug Exclusion.

Finance: draft 13-week cash view by Friday.

Amicus Therapeutics, Inc. (FOLD) - PESTLE Analysis: Environmental factors

You're looking at how Amicus Therapeutics, Inc. handles its impact on the planet, which, honestly, is a key part of modern investment due diligence, even for a focused biotech firm.

The good news is that Amicus Therapeutics definitely sees this coming. As a biotech company, its environmental footprint is generally smaller than, say, a massive chemical manufacturer, but they are still committed to minimizing it. They are actively working to embed environmental responsibility into their day-to-day and long-term plans.

Minimizing Footprint and Supply Chain Engagement

Amicus Therapeutics is focused on practicing environmental responsibility by identifying and implementing sustainability objectives across its operations and, crucially, with its manufacturing and supply partners. This focus on the supply chain is smart; for a company of this size, the biggest impact often lies upstream. To get a handle on this, they joined the CDP Supply Chain Program. This lets them engage directly with partners to consolidate environmental data across the entire chain, which is a concrete step toward oversight. What this estimate hides is the actual baseline GHG number, which they are working to establish.

Here's a quick look at their scale and recent performance, which frames their environmental scope:

Metric	Value (2024/Q3 2025)	Source/Period
2024 Total Revenue	$528.5 million	Preliminary 2024
Galafold Patients	~2,730	As of end of 2024
Q3 2025 Total Revenue	$169.1 million	Q3 2025
Q3 2025 GAAP Net Income	$17.3 million	Q3 2025

Greenhouse Gas Reduction and ESG Reporting

A major stated goal is achieving a reduction in greenhouse gas (GHG) emissions across their operations and the supply chain. This commitment is formally documented in their annual Environmental, Social, and Governance (ESG) report, which reinforces that environmental stewardship is integrated into their corporate responsibility structure. The 2024 report, for instance, highlighted increased efforts toward transparency in this area. They are aligning these initiatives with the Sustainability Accounting Standards Board (SASB) for the biotechnology and pharmaceuticals sector, which gives investors a standardized way to track progress.

Their environmental focus areas, as detailed in their reporting structure, include:

• Establishing baseline GHG emission metrics.
• Setting specific reduction targets.
• Tracking performance against those targets.
• Engaging suppliers on GHG management.

If onboarding new manufacturing partners takes longer than 14 weeks due to sustainability audits, churn risk for those partnerships rises.

Next Step: Portfolio Managers should model the NPV impact of the extended Galafold patent exclusivity to 2037 against the potential threat of competing gene therapies by year-end.