Amicus Therapeutics - Expanded Access Programs

Expanded Access Intelligence

Official Statements

Score contribution: 100 — 2 supporting sources.

Score 1002 references

Featured Reference

Amicus Therapeutics Expanded Access PolicyOpens in new window

“Since the company’s earliest days, extraordinary dedication to people affected by rare and orphan diseases has been a hallmark of Amicus Therapeutics. Our mission is to develop new medicines that improve the health and well-being of people living with rare diseases. With the development of medicines designed to satisfy unmet medical needs, comes the promise that these medicines will be broadly accessible.”

Amicus Therapeutics Patient & Professional AdvocacySource

“Amicus is committed to carefully designed and considered paths to Expanded Access to our investigational drugs.”

EAP Related Articles from Sponsor

Score contribution: 80 — 8 supporting sources.

Score 808 references

Featured Reference

Amicus Therapeutics Announces United Kingdom’s MHRA Grants Early Access to AT-GAA | Amicus TherapeuticsOpens in new window

Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) to AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. This positive opinion means that eligible adults living with late-onset Pompe disease (LOPD) who have received alglucosidase alfa for at least 2 years can now switch and have access to AT-GAA prior to marketing authorization in the UK.

Amicus Therapeutics Announces Participation at the World Muscle Society Annual Congress 2024 | Amicus TherapeuticsSource

Post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease

Amicus Therapeutics Announces U.S. Regulatory Pathway for Migalastat for Fabry Disease | Amicus TherapeuticsSource

Intermediate Expanded Access Program (EAP) planned to ensure short-term access to migalastat for U.S. patients who are currently on ERT and meet EAP requirements.

Amicus Therapeutics | Advancing Therapies to Treat Rare DiseasesSource

To Ensuring Patient Access to Medicines. Read more about our Expanded Access Program

Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom | Amicus TherapeuticsSource

Prior to Great Britain (GB) approval, Pombiliti + Opfolda was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP), a Priority Innovative Medicines designation, as well as a positive scientific opinion under the Early Access to Medicines Scheme (EAMS). This accelerated the time to market and enabled healthcare professionals to prescribe the treatment prior to marketing authorization based on clinical factors for patients with a clear unmet need.

Amicus Therapeutics Provides Updates on Clinical, Regulatory and Manufacturing Advancements for AT-GAA for Pompe Disease | Amicus TherapeuticsSource

Promising Innovative Medicine (PIM) Designation Issued by British MHRA with Potential for Early Access for Pompe Patients in United Kingdom Based on Phase 2 Results

Active EAPs on ClinicalTrials.gov

Score contribution: 60 — 3 supporting sources.

Score 603 references

Featured Reference

Expanded Access for ATB200/AT2221 for the Treatment of IOPDOpens in new window

Conditions: Pompe Disease Infantile-Onset

Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseSource

Conditions: Pompe Disease

Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseaseSource

Conditions: Fabry Disease

Reagan-Udall Foundation Insights

Company

Amicus Therapeutics

Expanded Access Website

https://www.amicusrx.com/advocacy/expanded-access/

Additional Information

Single-Patient EA Policies/Criteria Requests for Early Access cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver. Patients and caregivers seeking general information may reach out to Amicus’ Global Patient & Professional Advocacy Department at patientadvocacy@amicusrx.com or call toll-free in the United States at 1-866-9AMICUS (1-866-926-4287), or +44-1753-888-567 for International inquiries.

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