Zolgensma’s recent challenge of finding favor with the majority of the insurance companies in the US may just be about to get tougher with the news on manipulation in pre-clinical data.

Vinie Varkey, Senior Analyst of Neurology & Ophthalmology at GlobalData, a leading data and analytics company, offers her view on what this means for Zolgensma going forward:

“The data in question isn’t directly related to the commercial product; however, pre-clinical data does represent a critical component of the FDA’s approval. While Zolgensma’s clinical performance may be unaffected, questions on protocols used during product testing during the development phase could be something that regulators and payers may choose to weigh on when deciding on future approvals for Zolgensma, for example, expanding indications to a wider patient population for Zolgensma or when a payer decides to grant or restrict access to the therapy.

“According to GlobalData, the spinal muscular atrophy (SMA) market is expected to grow at a compound annual growth rate (CAGR) of 16% to reach a global value of $6.6bn in 2028. Furthermore, Zolgensma is expected to be the market leader in 2028 with peak year global sales of $3.1bn.

“It is likely that this development may slow down the progress of ongoing discussions between Novartis and reimbursement agencies in the US, a topic that is critical for the $2.125m therapy to be made available for patients with SMA. Similar regulatory and reimbursement processes in markets outside the US are also likely to be affected.”