MMIT Reality Check on Spinal Muscular Atrophy (3Q2023)

Payer Coverage:

A review of market access for spinal muscular atrophy treatments shows that under the pharmacy benefit, about 39% of the lives under commercial formularies are covered with utilization management restrictions. Around 24% of the lives under health exchange formularies are not covered for at least one of the drugs.

Under the medical benefit, about 88% of the lives under commercial policies are covered with utilization management restrictions. Around 51% of the lives under Medicare policies have access to at least one of the drugs without utilization management restrictions.

For about 99% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Around 75% of payer-controlled pharmacy benefit covered lives require prior authorization, with 2% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.

Trends:

FDA Expands Evrysdi’s Label

In May 2022, the FDA expanded the label of Roche Group member Genentech USA, Inc.’s Evrysdi (risdiplam) to include the treatment of babies younger than 2 months with spinal muscular atrophy. The agency initially approved the drug for patients at least 2 months old on Aug. 7, 2020; the newest use had priority review. Once-daily dosing for the oral solution is weight based. Website GoodRx lists the price of 160 mL as more than $23,387. The drug’s price is capped at $340,000 per year once someone reaches 44 pounds. For a 15-pound patient, the price would be less than $100,000 per year.

Survey Finds That Payers Are Covering Sequential Use of SMA Agents

A recent FDA approval of a label expansion put the three marketed therapies for spinal muscular atrophy (SMA) on equal footing for the youngest patients. And recent survey found that many payers are covering sequential use of the costly agents, including a gene therapy.

The FDA initially approved Evrysdi (risdiplam) from Roche Group member Genentech USA, Inc. on Aug. 7, 2020, for the treatment of SMA in people at least 2 months old. On May 30, 2022, the agency expanded the drug’s label to include the treatment of infants less than 2 months old. The survival motor neuron 2 (SMN2) splicing modifier is an oral solution administered by mouth or feeding tube and can be administered by a patient or caregiver at home after a recommended consultation with a health care professional prior to the first dose. That label expansion meant that Evrysdi joined two other therapies in being indicated for infants with the most severe type of SMA.

Recently Approved Evrysdi May Have Big Impact on SMA Class

The spinal muscular atrophy (SMA) therapeutic category continues to expand with the Aug. 7, 2020, FDA approval of Evrysdi (risdiplam) from Roche Group member Genentech, Inc. Industry experts maintain that the drug has the potential to significantly impact the class for a variety of reasons, including its route of administration and price.
People with SMA cannot produce enough SMN protein, leading to the loss of motor neurons, which results in problems breathing, swallowing, speaking and walking. Before a therapy was available to treat SMA, the condition was the No. 1 genetic cause of infant death.

Key Findings:

Market Events Drive Changes

In May 2022, the FDA expanded the label of Roche Group member Genentech USA, Inc.’s Evrysdi (risdiplam) to include the treatment of babies younger than 2 months with spinal muscular atrophy (SMA). The FDA initially approved Evrysdi in August 2020 for the treatment of SMA in people at least 2 months old. In May 2019, the FDA approved Novartis Pharmaceuticals Corp. subsidiary AveXis, Inc.’s Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy product intended to treat children less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 gene — the most severe form of SMA.

Competitive Market Landscape

Through the concerted efforts of drug companies, government agencies, academic institutions and advocacy groups, the SMA community has reached an exciting point where new therapeutics have entered clinical trials or are approaching trials in the near future.

Pharmacy, Medical Benefit Implications

Spinraza (nusinersen) and Zolgensma are covered under both the pharmacy and medical benefits, while Evrysdi is covered only under the pharmacy benefit. When there is coverage, prior authorization is almost always required.

Key Players in Market:

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