Novartis' SMA Gene Therapy Zolgensma, the World's Most Expensive Drug, Included in UK's NHS

Compiled by Fan Dongdong

According to the latest news, Novartis’s gene therapy for spinal muscular atrophy (SMA), Zolgensma (onasemnogene abeparvovec), has been approved for inclusion in the UK’s National Health Service (NHS).

Novartis’ gene therapy for spinal muscular atrophy, Zolgensma, is considered the world’s most expensive drug, with a list price of up to £1.79 million (US$2.4725 million) per dose. Patients can access this treatment through the UK’s National Health Service (NHS). Regarding pricing, the NHS has reached a confidential discount agreement with Novartis, though the specific figures have not been disclosed. The NHS described this as “a fair price for taxpayers” and hailed the deal with Zolgensma’s manufacturer, Novartis, as “landmark.”

Novartis’ Zolgensma is the first gene therapy for the treatment of spinal muscular atrophy (SMA). This therapy delivers a functional gene capable of expressing the SMN protein, packaged within an AAV9 viral vector. Administered via intravenous infusion, the delivered gene enables sustained expression of the SMN protein in patients. Consequently, Zolgensma is a gene therapy that targets the root cause of the disease, with the potential for a one-time curative effect. Clinical studies have demonstrated that patients with symptomatic and asymptomatic SMA who received a single infusion of Zolgensma achieved clinically meaningful therapeutic benefits. According to available data, Zolgensma has been approved in as many as 37 countries and regions, treating over 800 pediatric patients. In 2020, Zolgensma generated $920 million in sales revenue, representing a 150% increase.

Spinal Muscular Atrophy (SMA) is a rare and refractory disease characterized by decreased muscle strength due to the loss of function of specific genes. The incidence before early childhood is 1 to 2 cases per 100,000 people. Globally, two drugs have been marketed for the treatment of SMA. In addition to Novartis’s Zolgensma, the other drug is Spinraza (nusinersen), developed by Biogen in partnership with Ionis. Approved in 2016, Spinraza was the first therapy worldwide for SMA. It was approved in mainland China in late February 2019 for the treatment of patients with 5q spinal muscular atrophy (5q-SMA), becoming the first SMA therapy available in the Chinese market. Although Zolgensma was not the first approved therapy, it offers the advantage of requiring only a single-dose infusion compared to Spinraza.

External analyses suggest that the successful inclusion of Zolgensma in the UK’s healthcare reimbursement system has paved the way for final approval by the National Institute for Health and Care Excellence (NICE), the UK’s price regulatory authority. Previously, NICE issued a draft guideline recommending Zolgensma for the treatment of infants under 12 months of age with Type 1 spinal muscular atrophy (SMA). The draft guideline is currently open for public consultation until April 6, 2021.

References:

1.Novartis’s SMA gene therapy Zolgensma to be available for NHS use

2.Zolgensma: World's most expensive drug licensed for use on NHS to treat Spinal Muscular Atrophy

3.Novartis' £1.79M gene therapy Zolgensma scores cost watchdog's backing, threatening Biogen's Spinraza