Avalglucosidase alfa's NICE recommendation is settled, so NHS conversion speed, not cost-effectiveness, now gates the switch from alglucosidase alfa.

An estimated 200 UK patients, infantile- and late-onset combined, receive enzyme replacement therapy for Pompe disease, commissioned by NHS England Highly Specialised Services via clinical commissioning policy at 6-8 specialist metabolic disease centres, including Guy's and St Thomas', Manchester, Birmingham, and Queen Elizabeth Edinburgh. Infantile-onset patients begin alglucosidase alfa (Lumizyme/Myozyme, Sanofi) directly from newborn-screening confirmation, while late-onset patients require documented FVC decline or functional impairment before ERT initiation is authorised. Alglucosidase alfa remains the dominant, NHS-commissioned ERT: roughly 180 of the estimated 200 UK Pompe patients are on it today.

Nexviazyme (avalglucosidase alfa, Sanofi) is NICE-recommended as the first switch candidate (TA821, published August 2022, with a commercial arrangement already in place): the COMET trial showed a 23.5-metre 6-minute-walk-test improvement against a 13.2-metre decline for alglucosidase alfa, alongside better forced vital capacity preservation. With the appraisal and commercial arrangement already settled, the constraint on switching late-onset patients is now the pace of NHS conversion, not a pending cost-effectiveness decision. Pombiliti + Opfolda (Amicus), which showed a 20.8-metre 6MWT improvement in switch patients in the PROPEL trial, sits further back in the NICE appraisal queue and is currently accessible only via named-patient exceptional commissioning.

Cost-effectiveness is settled; the constraint now is how fast NHS centres convert patients.

Five questions this report answers:

Q1 - How quickly will NHS centres convert eligible patients from alglucosidase alfa to avalglucosidase alfa?

Q2 - What inadequate-response criteria will gate NHS switching to avalglucosidase alfa or Pombiliti + Opfolda?

Q3 - Where does Pombiliti + Opfolda sit in the NICE queue behind avalglucosidase alfa's TA821?

Q4 - How did COMET's 23.5-metre versus 13.2-metre 6-minute-walk result favor avalglucosidase alfa?

Q5 - Why does alglucosidase alfa still cover 180 of the UK's 200 Pompe patients?

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