Developer/Manufacturer
Developed by Avexis, Inc, a company acquired by Novartis in 2018.
Sold under the brand name Zolgensma.
Initial Registration
FDA
Application: BLA 125694
Approval: May 24, 2019
Initial indication: to treat pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Orphan status: Designation for treatment of spinal muscular atrophy on September 30, 2014.
European Medicines Agency
Approval: Pending
Orphan status: Designation for adeno-associated viral vector serotype 9 containing the human SMN gene for the treatment of spinal muscular atrophy , (EU/3/15/1509), June 19, 2015.
Pre-Clinical Development
Zolgensma was developed at Nationwide Children’s Hospital (NCH). NCH is a pediatric hospital and research center in Columbus, Ohio affiliated with the Ohio State University College of Medicine. NCH entered into a collaboration with AveXis to commercialize the gene therapy. AveXis licensed patents from NCH, as well as patent portfolios originally held by three non-profit organizations, the University of Pennsylvania, the University of North Carolina, and Genethon, a French charity.
NIH role
The three U.S. patent portfolio’s licensed the AveXis were funded by the NIH grants. The three principal investigators were:
- James Wilson, University of Pennsylvania.
- Richard Samulski, University of North Carolina.
- Brian Kaspar, Nationwide Children’s Hospital.
The NIH also funded clinical trials.
More on the public sector role here.
Public/Charity Funding of Zolgensma
Several SMA patient charities funded critical research. See more on their role here.
Licensing=
SEC disclosure reports have some details on the AveXis in-licensing of patents, although several of the figures are redacted. We do know that AviXis paid at least $176.1 million in pre-approval licensing fees to RengenXbio, which manages the University of Pennsylvania AAV vector patents, $1.6 million to Ask Bio, for the University of North Carolina patents, and $4.5 million to Geneathon patents portfolio. AveXis paid an additional $75 thousand in pre-approval benchmarks to the NCH, plus consulting fees of at least $29.7 million to Dr. Brian Kaspar, a NCH employee, and provided shares worth $99.4 million (456,043 shares at the $218 per share value paid by Novartis) to NCH and $383 million to Brian Kaspar, on acquire the rights to Zolgensma.
Clinical Trials Undertaken Before Approval
AVXS-101-CL-101; NCT02122952; Phase 1; 2014-05-05; 2017-12-15
AVXS-101-CL-102; STRONG NCT03381729; Phase 1; 2017-12-14; 2021-06-01
AVXS-101-CL-303; STRIVE NCT03306277; Phase 3; 2017-10-24; 2019-11-12
AVXS-101-CL-304|2017-004087-35|JapicCTI-184203; SPRINT NCT03505099; Phase 3; 2018-04-10; 2023-06-01
AVXS-101-CL-302|2017-000266-29; STRIVE-EU; NCT03461289; Phase 3; 2018-08-29; 2020-11-01
AVXS-101-MAP-001; NCT03955679; Expanded Access:Treatment IND/Protocol
AVXS-101-LT-001; START; NCT03421977; OBS
Estimated out-of-pocket-costs for trials, before tax credits: $10,384,066.
Estimate value of US ODTC:
Estimated value of US R&D tax credit:
Estimated cost of failures: before tax credits and deductions
Total Estimated Cost of Clinical Trials:
Total Estimated Cost of Clinical Trials, adjusted for risk and cost of capital: