Alnylam and PTC Therapeutics’ rare disease drugs given backing by NICE

pharmafile | January 20, 2023 | News story | Medical Communications  

Alnylam’s Amvuttra and PTC Therapeutics’ Translarna will be available to patients through the NHS after being backed by NICE, despite high list prices.

 

PTC Therapeutics’ Translarna is used to treat nonsense mutation Duchenne muscular dystrophy (DMD). Despite this recent win, Translarna has had a varied past. In 2014 it was initially rejected by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) after failing in a phase 2b trial, however PTC appealed and the verdict was overturned. It has also been rejected three times by the FDA, but CEO Stuart Peltz has expressed hope that data from a June 2022 trial will support another application.

 

It has been available in England under a managed access agreement since 2017, meaning patients could have access to the drug while more data was collected on it. Through the agreement, around 60 children have received the drug.

 

Alnylam’s Amvuttra treats hereditary transthyretin-related amyloidosis (hATTR) in about 150 people in the UK. It’s a recommended option for patients with mild symptoms (can walk unaided), or those with moderate symptoms (need assistance to walk). Due to it costing £95,826.36 per injection, Alnylam has offered a “confidential commercial” discount in order to make it feasible to offer on the NHS.

 

Alnylam was one of the first recommended under NICE’s “proportionate approach to technology appraisals”. This is a simplified process which resulted in a final draft of guidance being produced 20 weeks faster than the standard process.

 

James Spargo


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