MHRA Grants Marketing Authorisation for Elfabrio®▼(pegunigalsidase alfa) for the treatment of adult patients with Fabry Disease in Great Britain

MANCHESTER, UK, 15 August 2023 – Chiesi, the international research-focused biopharmaceutical and healthcare group, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for Elfabrio^® (pegunigalsidase alfa) in Great Britain for long-term enzyme replacement therapy (ERT) in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).^[1]

“We are delighted to have received MHRA authorisation for pegunigalsidase alfa, bringing an additional licensed treatment option for Fabry patients across Great Britain,” said Dr Kamran Iqbal, Head of Medical Affairs, Global Rare Diseases, Chiesi UK&I. “As part of our goal to ensure equal access to innovative therapies for people living with rare diseases, we are working closely with health technology appraisal agencies to ensure that all eligible patients can access this new treatment as soon as possible.”

Pegunigalsidase alfa is the first and only PEGylated ERT for Fabry disease.[2] Pegunigalsidase alfa is produced in plant cells using recombinant DNA technology.[3]

The MHRA authorisation is based on an overall positive benefit/risk balance of pegunigalsidase alfa in the claimed indication as stated in the European Medicines Agency’s (EMA) assessment report – which is based on the same evidence dossier submitted to the MHRA.³

The clinical development programme for pegunigalsidase alfa consists of 142 patients with Fabry disease (94 males and 48 females) of which 112 received pegunigalsidase alfa 1 mg/kg every other week.[4] These trials include the Phase 3 BALANCE (77 patients), BRIDGE (22 patients), and BRIGHT (30 patients) clinical trials, a one year Phase 1/2 clinical trial (18 patients), and four related extension studies (69, 29, 18 and 15 patients respectively).³ These studies show that pegunigalsidase alfa is generally well tolerated, with the most common adverse reactions being infusion-related reactions (reported by 6.3% of patients), followed by hypersensitivity and asthenia (reported each by 5.6% of patients).^3,4,[5]

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References

[1] Medicines and Healthcare products Regulatory Agency. Elfabrio^® (pegunigalsidase alfa) Summary of Product Characteristics. Available at https://mhraproducts4853.blob.core.windows.net/docs/97703775fb45191a2ee987d696eca8e011df3d4e. Last accessed August 2023.

[2] Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D. 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. 2019; 42(3): 534-544.

[3] European Medicines Agency. Elfabrio Assessment Report. Available at https://www.ema.europa.eu/en/documents/assessment-report/elfabrio-epar-public-assessment-report_en.pdf. Last accessed August 2023.

[4] European Medicines Agency. Elfabrio Summary of Product Characteristics. Available at https://www.ema.europa.eu/en/documents/product-information/elfabrio-epar-product-information_en.pdf. Last accessed August 2023.

[5] Bernat et al. eP149: Safety and efficacy of pegunigalsidase alfa, every 4 weeks, in Fabry disease: Results from the phase 3, open-label, BRIGHT study (Abstract). Genetics in Medicine. 2022; 27(3): S91-92.