A gene therapy to treat children younger than two with spinal muscular atrophy (SMA) has been approved by the FDA, with a price tag of $2.125 million for a five-year course.
The annual price for the drug, Zolgensma, is about $425,000 for five years, according to Novartis, which manufactures the drug. SMA is a rare genetic disease that affects motor neurons in the brain and spinal control muscle movement throughout the body, according to the FDA. Many children with the disease do not survive past early childhood from respiratory failure.
The new treatment minimizes the progression of the disease and is an adeno-associated virus vector-based gene therapy that delivers a fully functional copy of human SMN gene into the target motor neuron cells. A one-time intravenous administration of the product can improve muscle movement and function––as well as survival of a child with the disease. According to Novartis, the price isn’t out of touch with current chronic therapies for young children, which can cost more than $4 million in the first 10 years of life, the company stated. Zolgensma’s price is 50% less than the current 10-year chronic treatment of SMA and 50% below 10-year cost estimates for the treatment of genetic pediatric ultra-rare diseases (estimated between $4.4 million and $5.7 million).
“We have used value-based pricing frameworks to price Zolgensma at around 50% less than multiple established benchmarks including the 10-year current cost of chronic SMA therapy,” Novartis CEO Vas Narasimhan said in a statement. “In addition, the price of Zolgensma is expected to be within the range of traditional cost-effectiveness thresholds used by ICER when updated for its full labeled indications. We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time.”
To get FDA approval, the product underwent a clinical trial that is ongoing and a completed clinical trial with 36 pediatric patients with infantile-onset SMA. Patients treated with Zolgensma demonstrated significant improvement in motor development, such as head control and ability to sit without support. The drug does carry a risk of acute serious liver injury as a side effect.
The drug was approved through the FDA’s fast-track, breakthrough therapy and priority review designations. The agency also gave Novartis a rare pediatric disease priority review voucher to encourage the development of new drugs and biological products for the prevention and treatment of certain rare pediatric diseases.
“We are at the forefront of an exciting time in healthcare when we’ll be able to see major advancements in medical care with potentially curative gene therapies,” Steve Miller, MD, chief clinical officer of Cigna, said in a statement. “While there are many questions that we as a healthcare system need to consider, what does not change is our work to ensure that these life-saving medications are affordable and available to the patients that need them.”