Patients with late-stage Pompe disease who are failing to respond to enzyme replacement therapy will now have early access to an alternative treatment option, after Amicus Therapeutics'  cipaglucosidase alfa with miglustat was approved for the UK's Early Access to Medicine Scheme (EAMS).

The Medicines and Healthcare regulatory Products Agency (MHRA) has issued a positive scientific opinion for use of the treatment in adults with late-onset Pompe disease previously treated with alglucosidase alfa, based on currently available data.

This means that doctors will be able to prescribe the treatment to eligible patients with the rare, inherited disorder while its licensing is being considered by regulators.

As such, under EAMS the risk and legal responsibility for prescribing remains with the physician, and the opinion and EAMS documentation published by the MHRA are intended only to inform decision making and not to recommend use.

A risk management plan has been developed to ensure that cipaglucosidase alfa in conjunction with miglustat is used as safely as possible.