Chiesi Global Rare Diseases, a business unit of Chiesi Farmacuetici S. p.A., and Protalix Bio Therapeutics, Inc., announced the launch of an Expanded Access Program (EAP) in the United States for pegunigalsidase alfa for the proposed treatment of Fabry disease. The US FDA is currently reviewing a biologics license application for pegunigalsidase alfa. The EAP will run concurrently with Protalix’s ongoing phase 3 clinical program.
Marcel van Kuijck, PhD, global head of medical affairs at Chiesi Global Rare Diseases, said, “The launch of this Expanded Access Program for pegunigalsidase alfa is another example fo 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.”
Raul Chertoff, MD, vice president and chief medical officer at Protalix, said, “We are excited that a broader group of physicians and patients beyond those in our phase 3 program will have access to pegunigalsidase alfa, and that such support to Fabry patients in the US is available prior to FDA’s final review.”
According to a press release, 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. There are other eligibility criteria as well. Patients participating in the EAP will receive infusions of pegunigalsidase alfa every 2 weeks at 1 mg/kg body weight.
Jerry Walter, founder and president of the National Fabry Disease Foundation, said, “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. 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 the treatment they need.”
The EAP is listed on Clinical Trials.gov identifier: NCT04552691. Treating physicians should submit requests on behalf of their patients for consideration via the EAP portal at https://chiesi.versalc.com