What are the Clinical Trial Results for this Intravenous Treatment?
ZolgensmaTM has been and is still being tested in several different clinical trials (e.g. START, STR1VE EU, STR1VE, STRONG and SPR1NT), which have enrolled a variety of patients with SMA Types (1-2). The gene therapy has been consistently shown to be safe and well tolerated by those treated with the viruses.
The latest decision by the FDA in the USA to approve ZolgensmaTM for SMA patients under 2 years of age comes from the ongoing Phase 3 STR1VE trial and Phase 1 START follow-up trial, both of which include infants with SMA Type 1 and test intravenous injections of the drug.
In the START trial, patients treated with ZolgensmaTM have achieved a range of motor milestones never seen in the natural history of the disease without treatment, including sitting, talking and some patients walking, with no reduction of the effect nearly four years after treatment. In the Phase 3 STR1VE trial, ZolgenmsaTM treatment has been confirmed to result in prolonged event-free survival, improvements in muscle function, and achievement of key milestones.