The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has issued Sanofi a Promising Innovative Medicine (PIM) designation for avalglucosidase alfa in the treatment of Pompe disease.
A PIM designation is awarded to treatments that could offer a major advantage for patients and is an early indication that a medicine is a promising candidate for the Early Access to Medicines Scheme (EAMS).
The MHRA’s decision was based on data from the phase III COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa – the standard of care – in patients with late-onset Pompe diseases, as well as the phase II mini-COMET trial =, which evaluated avalglucosidase alfa in patients with infantile-onset Pompe disease previously treated with alglucosidase alfa.
The phase III COMET trial met its primary endpoint, demonstrating non-inferiority of avalglucosidase alfa in improving respiratory muscle strength compared to the standard of care in patients with late-onset Pompe diseases.
In phase II mini-COMET study, the primary objective of assessing the safety of avalglucosidase alfa in patients with infantile-onset Pompe disease was achieved during the primary analysis period.
The MHRA’s PIM designation marks an important milestone in Sanofi’s 20-year commitment to the Pompe community and we are delighted with the decision,” said Nicole Farmer, general manager UK & Ireland, Sanofi Genzyme.
“This not only recognises the life-threatening nature and high unmet need of Pompe disease but indicates that avalglucosidase alfa is an innovative medicine which could offer a potential new therapeutic option for patients with this rare disease,” she added.
Pompe diseases is a rare genetically-inherited disorders which results in the deficiency of the acid alpha-glucosidase enzyme. This causes the accumulation of glycogen in certain organs and tissues, and impairs their ability to function normally.