Positive interim results from a Phase III trial evaluating the effect of Novartis’ Zolgensma in Type 1 SMA patients is expected to make the case stronger for its approval and also its long-term use as a mainstream treatment option, says GlobalData, a leading data and analytics company.

While the company waits in anticipation for a decision on the approval of the drug, the latest results are not surprising. So strong were the results from the pivotal Phase I START trial that it was used as the basis for filing the drug for approval in US, Europe, and Japan. The interim results demonstrated prolonged event-free survival in Type 1 SMA patients who received the therapy as well as achievement of milestone developments compared to the natural history of the disease.

Vinie Varkey, MSc, Senior Neurology & Ophthalmology Analyst at GlobalData, comments: “Besides the strong results, this therapy’s mechanism of action is an attractive treatment as it is proposed to be administered as a one-time infusion compared to currently available Spinraza from Biogen, which needs to be administered more frequently via an intrathecal administration; the latter represents a resource-intensive procedure and one that is considered risky for SMA patients who have undergone spinal fusions in order to address issues related to Scoliosis.”

Due to the therapy being evaluated in a specific type of SMA patient population, in this case Type 1, the results are expected to provide impetus among physicians and patients for its long-term use in that particular patient population and avoid the risk of therapy not being favoured for use due to generalization of safety and efficacy results to other patient populations that may not have been necessarily studied during the clinical trials.

As promising as the results are, there are also concerns and unknowns of such therapies, for example, the long-term safety issues of introducing virus-based delivery platforms to deliver the gene therapy as well as the pricing of these therapies.

Varkey concludes: “Results from ongoing clinical trials that evaluate these innovative therapies as well as collection of real-world evidence of patients who are expected to receive such therapies will help to clarify such issues and hence help in its wider adoption as mainstream option. Sustainable funding solutions are also expected to evolve as discussions between funding agencies and drug developers continue in a concerted effort to make these therapies accessible to more patients.”