FDA on Friday approved the first gene therapy to treat a rare type of muscular atrophy in infants, and Novartis, which manufactures the drug, has said it will set the treatment's list price at $2.125 million—making it the highest-priced drug in the world.

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About the drug

FDA approved the drug, Zolgensma, as the first gene therapy to treat children under age two with spinal muscular atrophy (SMA), which is "an inherited neuromuscular disease that causes progressive loss of muscle function," according to STAT News. FDA said the most severe form of SMA, known as Type I, is "a leading genetic cause of infant mortality." According to Reuters, SMA affects approximately one in every 10,000 live births, and 50% to 70% of those affected with SMA have the Type I form of the disease.

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According to FDA, Zolgensma "is an adeno-associated virus vector-based gene therapy that targets the cause of SMA." FDA explained the altered virus "vector delivers a fully functional copy of human [survival motor neuron] gene into the target motor neuron cells."

FDA said it based its approval of Zolgensma on findings from an ongoing clinical trial and one completed clinical trial involving a total of 36 pediatric patients with infantile-onset SMA that were between the ages of approximately 2 weeks and 8 months at the time they entered the trials. The agency said, "A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child's motor neurons, which improves muscle movement and function, and survival of a child with SMA." Specifically, FDA said, "Compared to the natural history of patients with infantile-onset SMA, patients treated with Zolgensma … demonstrated significant improvement in their ability to reach developmental motor milestones (e.g., head control and the ability to sit without support)."

The agency said the most common side effects associated with Zolgensma are elevated liver enzymes and vomiting. FDA mandated that Zolgensma be sold with a boxed warning—the strictest warning the agency can require—that patients taking Zolgensma could experience serious liver injury. FDA said patients with pre-existing liver conditions might be at higher risk of experiencing serious liver injury while being treated with Zolgensma. As such, FDA said health care providers should complete clinical examinations and laboratory tests to assess patients' liver function before they are treated with Zolgensma, and providers should monitor patients' liver function for a minimum of three months after patients receive the treatment.

FDA also noted that certain vaccines are contraindicated for individuals treated with "a substantially immunosuppressive steroid dose," and caregivers therefore should consult with their health care providers to determine whether patients' vaccination schedules should be adjusted.

Acting FDA Commissioner Ned Sharpless said Zolgensma's "approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases." He continued, "With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality. The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future. The FDA will continue to support the progress in this field by helping to expedite the development of products for unmet medical needs through the use of review pathways designed to advance innovative, safe and effective treatment options."

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Price set at $2.1M

Novartis said it will set Zolgensma's price at $2.125 million, making it the highest-priced drug in the world. According to Axios' "Vitals," Novartis has said it will allow health insurers to pay for Zolgensma over the course of five years via annual installments of $425,000.

Zolgensma is administered as a one-time injection, which differs from Spinraza, a drug that was approved in 2016 to treat mild and severe cases of SMA and has been used to treat thousands of patients, STAT News reports. Spinraza is administered via regular spinal infusions over the course of a patient's lifetime. The Spinraza infusions cost $750,000 during the first year and a total of $375,000 annually in subsequent years.

According to STAT News, Zolgensma therefore might be viewed as more convenient than Spinraza. Further, Novartis has noted Zolgensma's annual price of $425,000 is lower than Spinraza's first-year price. David Lennon—head of Novartis' AveXis unit, which developed Zolgensma—said Zolgensma would cost half as much as Spinraza would over a 10-year period.

However, the Institute for Clinical and Economic Review, which evaluates drug pricing and effectiveness, in a statement said a suitable price range for Zolgensma would be between $1.1 million and $1.9 million.

Emmanuelle Tiongson, a pediatric neurologist at Children's Hospital Los Angeles who has treated patients with Zolgensma under an expanded access program, said Zolgensma could be "a new standard of care for babies with the most serious form of SMA," but added, "The job now is trying to negotiate with insurers that this would be a long-term savings."

Novartis announced that, in addition to allowing insurers to pay for Zolgensma in installments, it also will offer refunds if the treatment is not effective and will provide discounts to payers who agree to standardize coverage terms for Zolgensma. Novartis CEO Vas Narasimhan said, "Our goal is to ensure broad patient access to this transformational medicine and to share value with the health care system."

Michael Sherman, CMO of insurer Harvard Pilgrim Health Care, said his company feels Zolgensma's list price is "within the pricing we would deem appropriate," and the insurer is working with Novartis on a value-based coverage agreement for the drug.

Jefferies predicted that Zolgensma sales will reach $2.6 billion, although the drug could face further competition, STAT News reports. According to STAT News, Roche currently is working to develop a daily pill to treat SMA that could reach the U.S. market in 2020 (Feuerstein, STAT News, 5/24; FDA release, 5/24; Owens, "Vitals," Axios, 5/28; Miller/Humer, Reuters, 5/24; Roland, Wall Street Journal, 5/24).

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