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SMA
Avexis announced that they have applied for FDA approval for the gene therapy called AVXS-101. This medicine It is a treatment that replaces the missing or damaged SMN1 gene in SMA patients. This medicine is expected to be suitable for SMA Type 1 babies. In addition, Avexis is also testing the intrathecal administration of this drug, that is, through the spinal fluid. Intrathecal application will enable older patients (type 2-3) to access this treatment. FDA will first review this application for completeness, and then FDA will evaluate this application to determine that AVXS-101 is safe and effective when administered intravenously. The final decision is expected to be made in the second half of 2019. This medicine It is a single dose. The healthy gene is injected at once and the patient becomes capable of producing the SMN protein. After Avexis received approval from FDA and EMA, Prices are expected to decrease for Spinraza, the only SMA drug in the world, if it is released to the market. title="To get detailed information about the subject;" href="http://www.curesma.org/news/avexis-statement-fda-filing.html">http://www.curesma.org/news/avexis-statement-fda-filing.html
HUMAN TRIALS OF AVEXIS AVXS-101 GENE THERAPY
|
The Name Of The Study |
Countries of Operation |
Number of Patients |
Manner of Application |
Latest Status |
|
STR1VE |
USA |
- 20 SMA type-1 patient
- less than 6 months |
IV injection |
records complete |
|
STR1VE-EU |
EUROPE |
- 30 SMA type-1 patient
- less than 6 months |
IV injection |
Registration continues. Italy and UK are included |
|
SPRINT < /td> |
WORLD
WIDE |
At at least 27 patients whose symptoms were observed before 6 weeks
- SMN2 copy must be 2 or 3 |
IV injection |
It continues in the USA and Canada.
Australia is newly included. |
PEOPLE OF AVEXIS AVXS-101 GENE THERAPY ÜTESTS ON IT
| ÇName of the Study | Conducted
Countries |
Patient Number | Application Method | Final Status |
| STR1VE | U.S.A. | – 20 SMA type-1 patients
– Under 6 months |
Intravenous injection | Registration completed |
| STR1VE-EU | EUROPE | – 30 SMA type-1 patients
– Under 6 months |
Intravenous injection | Registration continues. Italy and England also included |
| SPRINT | WORLD
OVERALL |
– At least 27 patients whose symptoms were observed before 6 weeks of age
– SMN2 copy must be 2 or 3 |
Intravenous injection | Continued in the USA and Canada.
Australia is new was made. |
| STRONG | U.S.A. | – 27 babies and children with deletion of the SMN1 gene but without gene modification.
– Must be older than 6 months and less than 5 years old – At least 3 copies of SMN2 must be present |
Spinal cord injection | Registration completed |
| REACH | WORLD
GENERAL |
Information from the STRONG study will help decide on the final version of this study | Spinal Cord injection | Scheduled. |
