Text/Yangcheng Evening News all-media reporter Zhang Hua
Recently, the Drug Evaluation Center (CDE) of the State Food and Drug Administration of China announced that novartis' AAV gene therapy drug Zolgensma (OAV101 injection), which treats Spinal Muscular Atrophy (SMA), has obtained tacit permission for clinical drug trials in China. Previously, on October 21, 2021, the clinical drug trial application submitted by the drug was accepted.
Patients with spinal muscular atrophy Photo/Visual China
One injection, long-term relief and even cure of SMA
Speaking of SMA, it is estimated that everyone is not unfamiliar, it is a group of serious genetic neuromuscular diseases caused by muscle weakness and atrophy caused by degeneration of anterior horn cells of the spinal cord. In May 2018, SMA was included in the First Catalogue of Rare Diseases jointly formulated by the National Health Commission and other departments.
According to statistics, in newborns, the incidence of SMA is about 1 in 10000. In the general population, about 1 in every 40 to 50 people is a carrier of the SMA pathogenic gene. According to this incidence rate, the number of neonatal SMA patients in mainland China may be 1200 new every year, and the number of existing patients is about 30,000. With this type of disease, patients may gradually feel physical weakness and the condition will become worse and worse, and it will be difficult to reverse. If left untreated, SMA patients will gradually deteriorate their muscle strength and eventually become infected and die due to problems such as breathing or swallowing.
In February 2019, when Spinraza (Nocinasin Sodium Injection) for SMA treatment entered China, one injection was priced at 699,700 yuan, and patients needed to inject 4 injections in the first 63 days and 1 injection every 4 months thereafter, for lifelong medication. At that time, most SMA patients were unable to afford the expensive drug cost of nearly 700,000 injections, and could only hope for medicine.
It is gratifying that after waiting for three years, on January 1, 2022, the mainland will be this nearly 700,000 yuan a needle of drugs through national negotiations into medical insurance, after medical insurance reimbursement, Guangzhou patients using Northinal sodium injection for the first year about 60,000 yuan out of pocket, if you add Sui Shikang and other commercial insurance, the annual self-payment is estimated at about 20,000 yuan, and finally let the rare disease SMA patients can afford the cost of treatment. As soon as the news came out, it caused a heated discussion in the whole society, and everyone was happy for rare disease patients.
In fact, there is also a cure for treating SMA. On May 24, 2019, the U.S. FDA approved Novartis' first gene therapy for the treatment of SMA patients under 2 years of age, Zolgensma. Patients only need to receive one intravenous administration to express smN proteins in cells for a long time, achieving long-term remission and even cure.
Doctors inject nocinnasine sodium into SMA patients in figures/provided by respondents
Liu Li, chief physician of the Department of Genetics and Endocrinology of Guangzhou Women and Children's Medical Center, chairman of the Rare Disease Branch of the Guangdong Medical Association, and the project leader of the Guangdong Rare Disease Diagnosis and Treatment Collaboration Network, told reporters that a child with SMA who once encountered a child with SMA began to have symptoms after one month of birth, the condition was relatively serious, about four months of age needed ventilator assisted ventilation, completely unable to get rid of the ventilator, nor could he eat by himself, needed gastric tube assisted eating, the child's intelligence and consciousness were normal, and parents actively sought treatment methods After injecting the child with four doses of nocinachy sodium, they began to look for disposable treatment drugs, and finally went abroad to inject Zolgensma after inquiring about it. After returning home, the child's condition has indeed improved, such as being able to wave, and when he is in a good state, he can not need ventilator assisted ventilation for about 10 hours. It can be said that this one-time drug treatment is of great help to the improvement of symptoms in children, but the cost is extremely expensive.
Sky-high prices of drugs are expected to enter the Chinese market
Because it is a one-shot cure, Zolgensma is priced far higher than Nocinasine sodium injection, currently priced at $2.125 million, known as the most expensive drug in pharmaceutical history, and Zolgensma has been approved for marketing in nearly 40 countries and regions around the world.
Today, the drug program of more than 13 million yuan a shot is planned to enter China and conduct clinical drug trials. It's shocking that the price is so expensive. Liu Li said in an interview with reporters that the drug has plans to conduct clinical drug trials in China. Through drug clinical trials, clarifying the safety and efficacy of drugs for patients in China is the preliminary work that needs to be done before the drugs are formally submitted for approval and marketing.
According to the reporter's understanding, at present, 3 SMA therapies have been approved worldwide, in addition to Zolgensma, which also includes Spinraza (Northinasan sodium injection) and Roche's Risdiplam (rispramine). Risperlan was approved for marketing by the State Food and Drug Administration of China in June 2021 for the treatment of patients with spinal muscular atrophy aged 2 months and above, and risperlan is also the first oral treatment drug approved to treat spinal muscular atrophy in Chinese mainland. And Northinasan sodium injection has been included in medical insurance. According to data from meier SMA care center, on January 1 this year alone, more than 20 SMA patients in 11 provinces have used Nocinasin sodium injection.
Director Liu Li said that if Zolgensma enters China, it will have one more choice for SMA patients. However, the current drug also has limitations, that is, it is only for patients under 2 years of age for the time being. Experts believe that if Zolgensma is listed in China, the price needs to be particularly reasonable before there may be patient choices. (For more news, please pay attention to Yangcheng Pie pai.ycwb.com)
Source | Yangcheng Evening News Yangcheng Pie
Editor-in-charge | Wang Moyi