Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the regulatory proceedings of their treatment, PRX-102. This update is in regard to a Type A meeting with the FDA that was held on September 9, 2021.
Updates on PRX-102
Protalix and Chiesi recently received the minutes of their Type A meeting with the FDA, and these minutes included information about the Complete Response Letter (CRL) received in regard to the Biologics License Application (BLA) submitted for PRX-102. This information was positive, especially as it outlines a potential road to resubmission of a BLA.
The information held in the meeting’s minutes includes:
- Meeting discussion
- Preliminary comments
- Planned data package that will go along with the resubmitted BLA
Both companies are excited by the content of their Type A meeting, as their data package should pave the way to traditional approval of PRX-102. Part of this package is the Phase III BALANCE trial, specifically the last two years of analysis. This data, alongside the new and evolving regulatory landscape, point to later approval of this Fabry disease treatment.
Additionally, European Fabry disease patients may have access to this treatment soon as well. Protalix and Chiesi met with the EMA’s Rapporteur and Co-Rapporteur last week, both of whom supported the submission of a Marketing Authorization Application (MAA). In response, the two companies plan to submit an MAA in the first quarter of 2022.
About Fabry Disease
Fabry disease is a lysosomal storage disorder that occurs when globotriaosylceramide, a type of fat, builds up in the cells. It typically affects males. This accumulation can affect multiple organs throughout the body, but typically affects the kidneys and heart. As the buildup usually begins in childhood, symptoms will worsen as one ages. These symptoms include pain in the hands and feet, dull and cloudy vision, the inability to sweat, clusters of red spots on the skin, tinnitus, loss of hearing, and gastrointestinal issues. Heart attacks, strokes, and acute kidney damage may be complications. This disease is usually diagnosed after birth, as it is picked up in newborn screening exams. Treatments are symptomatic.