Benzinga | Oct 2, 2020 08:08AM EDT

Chiesi Global Rare Disease, Protalix Highlight Launch Of Expanded Access Program In US For Pegunigalsidase Alfa For Proposed Treatment Of Fabry Disease

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare group (Chiesi Group), and Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx(r) plant cell-based protein expression system, today announced the launch of an Expanded Access Program (EAP) in the United States for pegunigalsidase alfa for the proposed treatment of Fabry disease. A biologics license application (BLA) for pegunigalsidase alfa is currently under review by the U.S. Food and Drug Administration (FDA). This EAP will run concurrently with Protalix's ongoing Phase III clinical program.

"The launch of this Expanded Access Program for pegunigalsidase alfa is another example of Chiesi's and Protalix's shared commitment to support patients whose condition cannot be adequately treated by currently available FDA-approved therapies for Fabry disease," said Marcel van Kuijck, Ph.D., Global Head of Medical Affairs at Chiesi Global Rare Diseases.

"We are excited that a broader group of physicians and patients beyond those in our Phase III program will have access to pegunigalsidase alfa, and that such support to Fabry patients in the U.S. is available prior to FDA's final review," added Raul Chertkoff, M.D., Vice President and Chief Medical Officer at Protalix.

The EAP is open to patients with a clinical diagnosis of Fabry disease who, in the opinion of the treating physician, have no comparable or satisfactory alternative treatment options with currently available FDA-approved therapies for Fabry disease. Other eligibility criteria apply. Patients participating in the EAP will receive infusions of pegunigalsidase alfa every two weeks at 1mg/kg body weight. Information related to adverse events and other limited data will be collected from participants.

"The National Fabry Disease Foundation and the Fabry community are very excited about the launch of Chiesi's Expanded Access Program for pegunigalsidase alfa for the treatment of Fabry disease," said Jerry Walter, Founder and President, National Fabry Disease Foundation. "As the number of people diagnosed with Fabry disease continues to exceed predictions, access to treatment through Expanded Access Programs can play an important role in helping as many eligible patients as possible access the treatment they need."

Pegunigalsidase alfa is an investigational product and currently not approved by the FDA. The effectiveness and safety of pegunigalsidase alfa have not been reviewed or approved by the FDA. Before FDA review and approval, no conclusions can be drawn on pegunigalsidase alfa's efficacy and safety profile. When seeking expanded access, treating physicians should consider all possible risks of treatment with pegunigalsidase alfa. Access must be compliant with all applicable federal and state laws and regulations. Investigators should not seek reimbursement for product provided to patients who participate in a government-funded insurance program.

In August 2020, the FDA accepted the pegunigalsidase alfa BLA and granted Priority Review designation for pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease. Concurrently with the EAP, pegunigalsidase alfa is being evaluated by Protalix BioTherapeutics in patients with Fabry disease under an open Investigational New Drug application in the Phase III BALANCE, BRIGHT and BRIDGE clinical trials and other related open label studies.

The Expanded Access Program is listed on ClinicalTrials.gov Identifier: NCT04552691 ( https://clinicaltrials.gov/ct2/show/NCT04552691). Additional information on Chiesi's Expanded Access policy is available at https://www.chiesiusa.com/sustainability/expanded-access-programs/. Treating physicians must submit requests on behalf of their patients for consideration via the EAP request portal at https://chiesi.versaic.com.