Roche and PTC’s SMA treatment shows positive trial results

pharmafile | March 16, 2021 | News story | Manufacturing and Production PTC, PTC Therapeutics, Roche, sma, spinal muscular atrophy 

Roche and PTC Therapeutics’s Evrysdi treatment for children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) has shown positive results two years into PTC’s SUNFISH trial.

The data demonstrated that Evrysdi patients sustained or improved in motor function after 24 months of treatment. The patients and caregivers also reported improvements in their ability to function independently and complete daily tasks.

The double-blind, placebo-controlled trial, in patients aged between 2 and 25 years old, evaluated motor function using total score of Motor Function Measure 32 (MFM-32) – a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA.

The study found the MFM-32 scores showed improvement or maintenance of motor function during the second year of treatment with Evrysdi. It also showed stabilised motor function for patients who began treatment with Evrysdi after 12 months of placebo.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement.

The FDA approved Evrysdi for the treatment of SMA in both adults and children aged two months or older in August last year. The drug is also approved in seven countries and has been filed in more than 50 countries worldwide.

SMA is a rare but life-threatening genetic disease that directly affects approximately 10 people in every 100,000 worldwide.

Stuart W Peltz PhD, CEO of PTC Therapeutics, said: ”We’re encouraged by the long-term results from the SUNFISH trial, which reinforce the sustained clinical benefit that Evrysdi offers to a very broad population of patients with SMA, many of whom experience difficulty performing daily functions.

”These data further support the strong US launch and the need for an at home oral treatment option for the SMA community.”

Jack Goddard

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