The Most Expensive Drug in The World! Novartis' SMA Gene Therapy Zolgensma Strengthens Its Performance And Is About to Land in China

On April 26, Novartis released its 2022 Q1 financial report. During the reporting period, revenue was US$12.531 billion (+5% cc) and net profit was US$2.219 billion (+26% cc). *cc: Calculated at constant exchange rate.

Among them, Novartis' Spinal Muscular Atrophy (SMA) gene therapy Zolgensma, which has attracted much attention, achieved revenue of US$363 million, an increase of 18% year-on-year, mainly due to the expansion of access to Europe and emerging growth markets.

According to public information, Novartis Zolgensma is the world's first gene therapy for the treatment of spinal muscular atrophy, priced at US$2.125 million (equivalent to about 13.58 million yuan), also known as "the most expensive drug in history".

At present, Zolgensma has been approved in more than 40 countries, and in January 2022, it also obtained an implied license for clinical trials in China.

It is reported that there are currently 3 SMA therapies approved in the world, in addition to Zolgensma, Biogen's Nosinagen Sodium Injection (Spinraza) and Roche's Risprom Oral Solution Powder (Evrysdi).

At present, Spinraza and Evrysdi have both been approved in China. Among them, Biogen’s Spinraza has successfully entered the new list of medical insurance in my country in 2021.

Zolgensm has been approved in many countries and entered into medical insurance, and its performance has grown strongly year by year.

Data show that SMA is a rare inherited neuromuscular disease. Due to the lack of a functional SMN1 gene, SMA causes rapid and irreversible loss of motor neurons, affecting muscle function, including breathing, swallowing and basic movement. SMA is the number one genetic killer of infants and young children under 2 years of age, with type 1 SMA being the most common type, accounting for about 60% of all cases. Without treatment, more than 90% of patients will die or require permanent ventilation by age 2.

Novartis Zolgensma is the first gene therapy for spinal muscular atrophy. The therapy will express the normal gene of SMN protein, loaded in AAV9 viral vector, and the patient will be administered by intravenous infusion, and the injected gene will enable the patient to continuously express SMN protein in the body. Patients only need one injection.

In May 2019, Zolgensma was approved by the FDA for the treatment of spinal muscular atrophy in patients under 2 years of age.

In March 2020, Zolgensma was approved by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of children with spinal muscular atrophy (SMA) under 2 years of age, including those who were pre-symptomatic at the time of diagnosis. It should be noted that patients receiving Zolgensma must be negative for anti-AAV9 antibodies. It is estimated that in Japan, approximately 15-20 patients with SMA are eligible for treatment each year. It is reported that after the approval of Zolgensma Japan, the price was reduced to 1.55 million US dollars.

In May 2020, Zolgensma (onasemnogene abeparvovec) received conditional approval from the European EMA for the treatment of patients with type 1 spinal muscular atrophy (SMA), the most severe form of the disease, and those with SMA with three copies of the SMN2 gene . It is reported that in Europe, about 550-600 babies are born with SMA each year.

Due to the high price of Zolgensma, how to realize its post-listing performance has always been the focus of the industry.

Industry insiders pointed out that Zolgensma's growth potential mainly comes from being approved by more countries and covered by medical insurance in more countries, as well as increasing the SMA screening rate of newborns in developed countries such as the United States and Europe.

It is reported that in the U.S. market, Novartis cooperates with relevant insurance companies, and the family members of patients can pay in installments over five years, with an average of $425,000 per year.

In addition, in Japan and the United Kingdom, Zolgensma is covered by health insurance.

In May 2020, Zolgensma was included in Japan Health Insurance, and patients only need to pay 30% of the cost;

Zolgensma was included in the NHS in March 2021. It is reported that the British NHS and Novartis have reached a certain discount on the price, but the specific amount has not been announced. But the NHS said it was a "fair price to taxpayers" and a "landmark deal" with Novartis.

In terms of performance, Zolgensma has grown strongly year after year.

In 2019, Zolgensma achieved sales of $361 million. (Approved for the first time in the world in May of that year)

In 2020, Zolgensma achieved sales revenue of $920 million, an increase of more than 150% in revenue.

In 2021, Zolgensma will achieve revenue of $1.351 billion, a year-on-year increase of 47%.

In Q1 2022, Zolgensma achieved revenue of $363 million, a year-on-year increase of 18%.

Compared with similar drugs that require long-term treatment of spinal muscular atrophy, Zolgensma only needs to be administered once intravenously to achieve long-term remission or even cure. Therefore, despite the high cost, the industry's prospects for Zolgensma are still relatively optimistic.

Zolgensma's sales are expected to grow to $1.6 billion in 2022 and peak at $2.75 billion in the future, according to estimates by investment firm Jefferies.

Approval status of SMA drugs in China

As mentioned above, there are currently 3 SMA therapies approved worldwide, in addition to Zolgensma, Biogen’s Spinraza Sodium Injection and Roche’s Risprom Oral Solution Powder (Evrysdi).

Nosinagen Sodium Injection and Risprom Oral Solution Powder have been approved in my country, and Nosinagen Sodium Injection will also successfully enter my country's new national medical insurance list in 2021.

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Bojian Nosinagen Sodium Injection

In December 2016, Spinraza was approved in the United States, becoming the world's first SMA precision targeted therapy drug.

In February 2019, Spinraza was launched in China for the treatment of 5q spinal muscular atrophy (SMA) and became the first drug for the treatment of SMA in China.

Previously, Spinraza was priced at close to 700,000 in China, but only sold for $41 in Australia, causing an uproar in Chinese public opinion. (The actual purchase price in Australia is 760,000, but it can be reimbursed.)

In January 2021, Biojian launched a new assistance plan: the cost of the patient's self-payment for Nosinagen Sodium Injection in the first year was reduced from about 1.4 million yuan to 550,000 yuan, a decrease of about 60%; The fee has been reduced from the original average of about 1.05 million yuan per year to 550,000 yuan, a drop of about 50%.

As early as entering the 2021 National New Medical Insurance Catalogue, some local medical insurances such as Chengdu have included Nosinagen sodium into the reimbursement scope, and patients can use Nosinagen sodium for treatment at a minimum of 20,000-30,000 yuan per year.

In addition, inclusive insurance (commercial insurance) such as Guangzhou "Sui Suikang" and Foshan "Ping'an Buddha" also include Nosina Health Sodium. On the whole, patients only need about 100-180 yuan per year, and they can get the highest cumulative amount. More than 2 million yuan to more than 3 million yuan of medical insurance.

In December 2021, Spinraza was included in the latest medical insurance catalogue. According to relevant news, the price dropped to 33,000 per unit, a drop of over 90%.

In terms of sales performance, Spinraza’s global sales in 2020 were US$2.05 billion, and the performance in 2021 was US$1.9 billion, a year-on-year decline of 7.3%.

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Roche risprom powder for oral solution

In August 2020, Evrysdi was approved by the FDA for marketing, becoming the first small molecule targeting RNA to be approved for marketing. According to reports, the price of Evrysdi in the United States depends on the patient's weight, and the annual treatment cost is about 2.37 million yuan.

In June 2021, risprom was approved in my country for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older. This is the first oral disease-modifying drug approved in China for the treatment of SMA.

It is reported that the listing price of Lisplan in China is 63,800 yuan (60mg/bottle), and the patient assistance plan is "buy 3 bottles and get 6 bottles", which is equivalent to the price of a single bottle after charitable assistance is 21,267 yuan, and the annual cost is about 30 RMB 10,000 - RMB 1 million.

In 2021, Lispolan also caught up with the national medical insurance negotiation "bus", but the results showed that Lispilan did not enter the new list of medical insurance that year.

In terms of price, although Sina Pharmaceuticals has not yet been informed of the latest price trends of Lisplan.

However, it is worth noting that although Lisplan has not entered the 2021 National New Medical Insurance Catalogue, it has also entered some local medical insurances before.

According to the Jinan Times, in August 2021, “Qilubao”, Huimin Insurance of Jinan City, Shandong Province, for the first time included both oral risprom and nosinagen sodium for the treatment of spinal muscular atrophy (SMA). 

According to the insurance regulations, the reimbursement amount for the two SMA treatment drugs in "Qilubao" can reach 70%, up to a ceiling of 300,000 yuan, and simultaneous dual-channel reimbursement in hospitals and pharmacies can be achieved.

In terms of performance, it is reported that due to the first global market launch in August 2020, the first full sales year of Lispolan in 2021 shows that its global sales will be 659 million US dollars.

It is worth noting that in the treatment of SMA, both Nosinagen sodium and risprom need to undergo long-term treatment, while the sky-high drug Zolgensma, which was once priced at 13.5 million per needle, only needs to be administered once intravenously, which can achieve long-term treatment. The advantage of relieving or even curing.

At present, the prices of Nosinagen sodium and Risprom have been reduced to varying degrees in my country to meet the needs of more patients.