Blog September 2, 2019

Zolgensma Launch “On Track,” but Significant Challenges Remain

ZolgensmaOn July 18, Novartis told analysts its Zolgensma launch was “on track.” Despite the gene therapy’s $2.1 million price tag, the company states the launch is “one of the most successful launches from an access standpoint in rare diseases.” Novartis said it had reached coverage deals with more than 20 commercial insurance plans, covering approximately 40% of insured U.S. lives. Insurers have approved coverage for Zolgensma on a case-by-case basis, which Novartis says is usual for any new product until there is more understanding of the treatment’s value.


However, some analysts have commented on payer decisions to date being “surprisingly restricted,” at least initially. Some insurers have been more restrictive in their coverage than the label approved by the U.S. Food and Drug Administration, which indicates Zolgensma’s appropriateness for children with spinal muscular atrophy (SMA) under age 2. For example, Anthem currently cuts off coverage for Zolgensma once an affected child is older than 6 months, criteria the insurer has set based on the clinical trial data published to date. Some other insurers are limiting treatment coverage until an affected child has shown symptoms of SMA, despite the thinking that treating the disease at a pre-symptomatic stage may be more beneficial. Additionally, several firms have placed restrictions on the use of Zolgensma for patients who have also been treated with Spinraza, the other approved therapy for SMA. However, the situation is evolving, with some payers like Aetna reconsidering initial denials of reimbursement based on “emerging information, expert opinions, and the promise of the medication.”


But challenges to Zolgensma’s ultimate market success – and, indeed, the success of other therapies for SMA — go beyond insurance reimbursement issues and relate to how early SMA patients are identified. It is generally believed that patient identification and subsequent treatment initiation in the first weeks of life may offer the greatest benefits. Unfortunately, while approximately 400 infants are born each year with SMA, most are not diagnosed until symptoms are detected, which in children with less severe forms of the disease may take years.  


The U.S. Department of Health and Human Services (HHS) recommends testing newborns for about 60 genetic disorders in its Recommended Uniform Screening Panel (RUSP); SMA was added to this list in 2018, after Spinraza’s approval. However, each state sets its own testing panel based on its deliberations of how common the genetic disorders are among its population, the availability of treatments and support groups for those affected, and other factors. As a result, some states don’t test for all of the 35 conditions that HHS designates as the “core” of the RUSP panel.  Currently, only seven states screen for SMA, with an additional 20 states in the process of adding the condition to their testing panels.


Furthermore, while prenatal testing can screen for SMA, such tests may miss patients. Most SMA tests can only detect whether the SMN1 gene is present or absent, but not its copy number or whether the gene is faulty as a result of point mutations, all important information to the diagnosis of SMA. Additionally, some mothers prefer not to undertake prenatal screening and testing due to the related costs or because they fear that amniocentesis could pose a risk to their pregnancy.