Protalix Fabry Disease Expanded Access Policy
Expanded Access Policy
Protalix is focused on the development of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system.
Title of Program: Protalix Expanded Access for the treatment of individual Fabry patients with pegunigalsidase alfa (PRX-102)
Unlicensed Product: pegunigalsidase alfa (PRX-102), a recombinant human alpha galactosidase-A
Indication: Fabry disease
The best way for patients to gain access to pegunigalsidase alfa is through Protalix’s existing, on-going clinical trials.
However, where participation in existing clinical trials is not feasible, patients may be eligible for treatment with pegunigalsidase alfa through Protalix’s Expanded Access Program (EAP).
The objective of this EAP Policy is to provide guidance as to how to seek access to pegunigalsidase alfa through the EAP.
• “BALANCE”: https://clinicaltrials.gov/ct2/show/NCT02795676
• “BRIDGE”(not available in the US): https://clinicaltrials.gov/ct2/show/NCT03018730
• “BRIGHT”: https://clinicaltrials.gov/ct2/show/NCT03180840
Criteria for Consideration of Expanded Access Program
Key Inclusion Criteria:
- Subjects with confirmed diagnosis of Fabry disease whose clinical condition, in the opinion of the treating physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa. This criterion includes non-naive subjects who were treated in the past with agalsidase alfa, agalsidase beta or pegunigalsidase alfa in any other program.
- Requests for EAP participation must be made by a qualified physician who agrees to directly supervise the treatment of the patient.
- The requesting physician must have the resources and facilities to appropriately treat the patient with pegunigalsidase alfa
Please note: Protalix may not be able to provide pegunigalsidase alfa to all requesting physicians and patients. Factors that may lead to a denial of pegunigalsidase alfa may include; availability of pegunigalsidase alfa; applicable country or other rules and regulations prohibiting the supply of pegunigalsidase alfa; customs and import restrictions into the requesting country or jurisdiction; lack of medical expertise; and patients not meeting the inclusion criteria or taking other drugs that prohibit the use of pegunigalsidase alfa. Other limiting factors may also apply.
Process for Requesting Early Access to pegunigalsidase alfa
Only a qualified physician who agrees to directly supervise treatment of the patient may submit a request. Patients interested in participating in the EAP should talk to their physician about their treatment options.
- Upon a physician request to firstname.lastname@example.org, Protalix will evaluate the request and respond within 30 days of receipt
- If Expanded Access is granted, Protalix will facilitate the supply of pegunigalsidase alfa via this EAP
- Treatment guidelines will be provided
- Pertinent patient data and safety data should be recorded in a dedicated web-based patient adverse events recording system
Dr. Raul Chertkoff
Mobile: +972 (54) 222-8472
Fax: +972(4) 988-8092