The FDA has been informed by the therapy's manufacturer that data was manipulated during its animal testing phase.
The US Food and Drug Administration (FDA) is currently assessing the manipulation of data which accompanied applications of spinal muscular atrophy (SMA) gene therapy abeparvovec (Zolgensma)—a drug which the FDA had approved for children aged 2 years or younger with SMA in late May.
According to a statement from the FDA on Tuesday, the agency was informed of a data manipulation issue affecting the accuracy of the product’s performance in animal models as part of its Biologics License Application (BLA) submitted to and reviewed by the FDA prior to the marketing decision.
The manipulation issue was reported by Zolgensma manufacturer AveXis Inc., on June 28, a full month following the gene therapy’s approval to treat the leading genetic cause of infant mortality. AveXis was aware of the manipulation of data prior to the product receiving approval, the FDA reported, yet waited until after the decision to inform them.
“The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties,” Peter Marks, MD, PhD, director for the Center for Biologics Evaluation and Research (CBER), said in a statement.
That said, Marks reiterated the FDA’s interest in retaining Zolgensma on the market as it reviews the incident. Their concerns are currently limited to a concentration of the product testing data that accompanied the BLA.
“These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program,” Marks stated. “The totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favorable benefit-risk profile.”
The FDA’s decision to approve Zolgensma was supported by data from the phase 1 START trial and phase 3 STR1VE trial.
In the START trial, 15 patients were treated with either a low dose (n= 3) or high dose (n= 12) of the therapy. At 24 months post-treatment, 1 patient in the low-dose group required permanent ventilation while none in the high-dose group did. In the high-dose group, 75.0% (9 of 12) were able to sit for ≥30 seconds without support and 2 patients (16.7%) were able to stand and walk without support.
One investigator involved in the clinical trials called the findings “truly remarkable” and said they provide a “ level of unprecedented hope for families battling SMA Type 1.”
Zolgensma is an adeno-associated virus vector that delivers a functional copy of the SMN gene to patients, allowing a single intravenous administration of the therapy to improve muscle function and survival.
The FDA advised parents and health care professionals contact them at (800) 835-4709, or AveXis, for more information.