pharmaphorum

JANUARY 9, 2023

SMA is a genetic disorder that can cause rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

114

114

European Pharmaceutical Review

MAY 11, 2023

Novartis shared long-term data for its SMA gene therapy in March 2023, which indicated significant long-term motor-milestone achievements in spinal muscular atrophy (SMA). Without this protein, the motor neuron cells shrink and eventually die. 1 This is assuming a $1M price tag for all gene therapies. years post-dosing.

FDA 72

FDA 72

pharmaphorum

AUGUST 10, 2020

Evrysdi is the third treatment approved for SMA , an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene abeparvovec). Evrysdi is designed to treat SMA by increasing production of the survival of the motor neuron (SMN) protein.

FDA 57

FDA Specialty Pharmacies 57