UK – UK’s NICE recommends Roche’s Evrysdi

The first oral therapy for the leading genetic cause of death among babies and young children will be available to 1,500 people in England after Roche signed a price-cutting deal

The National Institute for Health and Care Excellence (NICE) has recommended that Roche’s Evrysdi (risdiplam) be made available to patients in England as part of a managed access agreement.

The deal agreed by Roche and NHS England will see 1,500 people with a rare genetic disorder spinal muscular atrophy (SMA) gain access to the drug while more data is collected to “address the uncertainties” identified by NICE. Once Roche submits this evidence, NICE will make a decision about routine use on the NHS.

NICE’s previous decision to reject Evrysdi for routine NHS use due to cost concerns was criticised by patient organisations including SMA UK, which called the move “disappointing” and urging Roche to reach an agreement with NHS England…