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Pegunigalsidase alfa (PRX-102) awarded Innovation Passport by the UK MHRA for the treatment of Fabry Disease

Manchester (UK), November 2021: Chiesi Limited announces that Pegunigalsidase alfa (PRX-102) was awarded Innovation Passport status by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) on 25th August 2021.

The Innovation Passport is the mandated entry point to the MHRA’s new Innovative Licensing and Access Pathway (ILAP), which aims to accelerate the time to market, facilitating patient access to medicines. The pathway allows early interactions with the MHRA and other stakeholders, including the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC).

To receive an Innovation Passport, PRX-102 met the following criteria: (1) the condition is life-threatening or seriously debilitating; (2) the program is intended for a rare disease or special population; and (3) the medicine has the potential to offer benefits to patients. The Innovation Passport designation is the first step in the ILAP process. It triggers the MHRA and its partner agencies to create a target development profile to chart out a roadmap for regulatory and development milestones with the goal of early patient access in the UK. Other potential benefits of ILAP include a 150-day accelerated assessment, rolling review, and a continuous benefit-risk assessment. PRX-102 is an investigational drug candidate; as such, the safety and efficacy of PRX-102 has not been reviewed or approved by EMA or other regulatory authorities for any patient group.

“We are delighted to be awarded the Innovation Passport for our therapy for patients with Fabry disease” commented Sarah O’Connell, Head of Rare Diseases, Chiesi Limited. “We look forward to working with the MHRA and their partner agencies to develop a roadmap to support our goal of patients gaining early access to PRX-102”.


About Fabry Disease

It has been estimated that 1,350 people in the UK have symptomatic Fabry disease,1,2 a rare, progressive, X-linked inherited lysosomal storage disorder, caused by a genetic mutation which leads to a deficiency of enzyme α-galactosidase A. This deficiency leads to progressive build-up of globotriaosylceramide (Gb3 or GL3) and globotriaosylsphingosine (lyso-Gb3) in the plasma and lysosomes of cells throughout the body. This accumulation causes metabolic dysfunction and cell death, leading to progressive organ failure.3-5 Fabry disease affects multiple organ systems, and may lead to renal dysfunction, cardiac manifestations, neuropathic pain, cerebrovascular disease, gastrointestinal symptoms, angiokeratomas and hypohidrosis/anhidrosis.3 Adults with Fabry disease are generally treated by intravenous infusion with enzyme replacement therapy (ERT) to replace the function of the missing α-galactosidase A enzyme.5


About Pegunigalsidase Alfa (PRX–102)

Pegunigalsidase alfa (PRX–102) is an investigational, plant cell culture-expressed, and chemically modified version of the recombinant a-galactosidase A enzyme. Protein subunits are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with a mean circulatory half-life of approximately 80 hours.6 PRX–102 is designed to potentially address the continued unmet clinical need of patients with Fabry disease.

About Chiesi Group

Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused healthcare group with 85 years of experience in the pharmaceutical industry and a global presence in 29 countries. Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease areas. Its R&D organisation is headquartered in Parma (Italy), and is integrated with R&D groups in France, the USA, the UK, and Sweden to advance Chiesi's pre-clinical, clinical, and registration programmes. Chiesi employs nearly 6,000 people.

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  1. Brennan P, Parkes O. Case-finding in Fabry disease: experience from the North of England. J Inherit Metab Dis. 2014 Jan;37(1):103-7. doi: 10.1007/s10545-013-9629-8. Epub 2013 Jul 5. PMID: 23828401.
  2. ONS (Office for National Statistics), 2021: Population estimates for the UK, England and Wales, Scotland and Northern Ireland: mid-2020
  3. Biegstraaten, M., R. Arngrimsson, F. Barbey, L. Boks, F. Cecchi, P. B. Deegan, et al. (2015). "Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document." Orphanet J Rare Dis 10: 36.
  4. Ortiz, A., D. P. Germain, R. J. Desnick, J. Politei, M. Mauer, A. Burlina, et al. (2018). "Fabry disease revisited: Management and treatment recommendations for adult patients." Mol Genet Metab 123(4): 416-427.
  5. Wanner, C., M. Arad, R. Baron, A. Burlina, P. M. Elliott, U. Feldt-Rasmussen, et al. (2018). "European expert consensus statement on therapeutic goals in Fabry disease." Mol Genet Metab 124(3): 189-203.
  6. Schiffmann, R., O. Goker-Alpan, M. Holida, P. Giraldo, L. Barisoni, R. B. Colvin, C. et al (2019). Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. J Inherit Metab Dis 42(3): 534-544


Media contacts Chiesi Limited

Donna Caslin, Senior PR and Communications Manager 

Phone: (+44) 161 488 5555


Pegunigalsidase alfa (PRX-102) awarded Innovation Passport by the UK MHRA for the treatment of Fabry Disease

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Last Updated: 07-Dec-2021