7. Zolgensma

From its jaw-dropping price tag to a data falsification scandal, Novartis' spinal muscular gene therapy Zolgensma still raked in $531 million in sales in its first 10 months. (Novartis)

7. Zolgensma
Company: Novartis
Disease: spinal muscular atrophy
Approved: May 24, 2019
First 10 months of sales: $531 million

Novartis’ spinal muscular atrophy gene therapy was a headline hogger even before its official FDA nod. Though not all of those headlines portrayed the med or Novartis in a good way, they haven’t hurt the drug’s uptake—until the pandemic.

The first wave of attention focused on Zolgensma’s price. At one point, Novartis had hinted at a price tag of up to $5 million. Even though the final cost came in way below that, at $2.125 million for the one-time therapy, it still ranks as the world’s most expensive treatment.

Then, the FDA revealed that Novartis’ gene therapy unit AveXis had doctored animal data in Zolgensma’s application package. To make things worse, the company’s delay in reporting its internal finding until after the approval came through drew wide criticism that dragged on for weeks.

Shortly after that fiasco quieted down, the FDA slapped a partial clinical hold on a trial testing the gene therapy’s spinal-injection delivery after belated preclinical data raised a safety red flag.

All the noise aside, Zolgensma is at its core an efficacious drug. It's demonstrated major improvement in terms of motor functions, such as the ability to sit without support, as well as event-free survival in patients with presymptomatic and infantile-onset SMA even beyond five years since treatment. Compared with its rival, Biogen’s Spinraza—which requires chronic dosing— Zolgensma holds the potential to cure the disease with just one injection of an adeno-associated virus vector-based therapy carrying a functional copy of human SMN gene.

RELATED: Roche’s risdiplam delay a relief for SMA rivals Biogen and Novartis—for now: analyst

To help cover the novel therapy, Novartis is offering payers outcomes-based agreements, which spread out the total cost over five years only when the drug continues to work. Payers have welcomed the new models. In July 2019, about 40% of commercial lives and four Medicaid plans had policies in place. The coverage rates quickly grew to 90% of commercial lives and 30% of Medicaid patients by October, according to CEO Vas Narasimhan. Plus, almost all on-label patients won final approvals after Novartis went through certain appeal processes, he said.

Sales-wise, Zolgensma also topped analysts’ estimates a with 2019 haul of $361 million, even though the COVID-19 pandemic slowed things down in the first quarter of 2020 to $170 million.

In the current Type 1 SMA indication, the number of U.S. patients Zolgensma treats each quarter has been steady at around 100, and Novartis now looks to Japan and EU as its next major growth opportunities. What’s more, it aims to tap into older patients with the intrathecal version, though it first needs to resolve the clinical hold on the formulation’s high dose.

7. Zolgensma