Roche's new drug risporan is approved in China for SMA treatment and enters a new era of oral administration

On June 16, 2021, the National Medical Products Administration of China formally approved Roche's blockbuster neuro-innovative drug Aimanxin® (English trade name: Evrysdi®, Chinese common name: Lisporan oral solution powder, English general) Name: Risdiplam Powder for Oral Solution), used to treat spinal muscular atrophy (SMA) in patients 2 months of age and older
.

This is the first oral disease-modifying drug approved for the treatment of SMA in China, and will provide new treatment options for SMA patients and families in China
.

SMA is a rare neurological disease that endangers multiple organs throughout the body and can cause disability and death.
The main cause of SMA is the lack or mutation of the SMN1 gene in the patient, resulting in insufficient expression of the systemic functional SMN protein [1], which affects the patient’s movement and breathing.
, Swallowing and multiple organs such as the spleen, heart, pancreas, etc.
, even threatening survival
.

SMA is one of the most common genetic diseases that cause infant death [2]
.

If children with severe SMA are not treated effectively, 80% of them will die within one year of age, and rarely can survive beyond two years [3]
.

The incidence of SMA is 1/10,000 of the surviving newborns.
There are about 1,000 new cases of SMA patients in China every year, and about 80% of them start within 18 months after birth [4]
.

In May 2018, the National Health Commission and other five departments jointly formulated the "List of the First Batch of Rare Diseases" to further strengthen rare disease management and improve the level of rare disease diagnosis and treatment
.

SMA was included in the first batch of 121 rare diseases included in the catalog
.

Professor Xiong Hui, deputy head of the Endocrinology, Genetics and Metabolism Group of the Pediatric Branch of the Chinese Medical Association, and chief physician of Peking University First Hospital, said: “The treatment of SMA is most important to two points: First, it is a race against time.
Start effective treatment, the better the prognosis, and even start treatment before symptoms, hopefully reach the state of non-ill children of the same age; the second point is to focus on the overall benefit of the patient, SMA will cause abnormalities in the patient's multiple systems, so pay attention to the patient during treatment Exercise, breathing, swallowing and other systems
.

Therefore, clinically, the treatment of SMA also requires a multidisciplinary team to collaborate and participate
.

"SMA treatment has entered a new stage of oral therapy, bringing new hopes for patients and families with risporan oral solution.
The powder regulates the splicing of SMN2 gene (SMN1 homologous gene) through two-site specificity, promotes the retention of exon 7, and increases the level of functional SMN protein
.

Lisporan can penetrate the blood-brain barrier and is distributed in the center and periphery.
It can increase the level of multi-system SMN protein in the whole body and keep it stable
.

The approval of Lisporan is based on two key multi-center studies FIREFISH and SUNFISH carried out on a global scale
.

The two studies included a broad population of SMA patients, including different types, ages, and exercise status, representing the real-world SMA patient population
.

The results of the study showed that the survival rate of patients with type 1 SMA after risporan treatment was significantly higher than that of natural history, achieving exercise milestones, and improving breathing and swallowing functions [5]
.

For patients with type 2 and type 3 SMA, motor function and life independence improved after medication
.

No drug-related safety incidents led to withdrawal from the study
.

 Based on this result, in April 2020, Roche submitted a listing application for Lisporan to the National Medical Products Administration.
In June, Lisporan was recognized as a priority review qualification.

.

As of today, Lisporan has been approved in more than 40 countries and regions, including China, and more than 3000 SMA patients have been treated with Lisporan worldwide
.

"The approval of Lisporan is an important milestone in the history of SMA treatment in China.
It means that the treatment of SMA has entered a new stage of oral therapy.
Chinese SMA patients and families usher in new hope
.

" The Pediatric Branch of the Chinese Medical Association is rare.
Professor Wang Yi, head of the disease group and chief physician of the Pediatric Hospital of Fudan University, said: “We are very pleased to see clinical research data confirming the effectiveness and safety of risporan in the treatment of various types of SMA patients
.

By improving the whole body functional SMN protein levels, profit Secretary flapping blue reversed the natural course of the disease, bringing multiple benefits for patients, including improved motor function, event-free survival, such as breathing and swallowing
.

At the same time, oral administration also rushed Langley Division Patients provided the convenience of medication
.

"Professor Jiang Yuwu, deputy chairman of the Pediatric Branch of the Chinese Medical Association, head of the neurology group, and director of the Pediatrics Department of Peking University First Hospital, said: "The approval of Lisporan is for domestic SMA patients and families.
This brings new options and will also further promote the clinical standard diagnosis and treatment of SMA in China
.
In
recent years, the clinical diagnosis and treatment of SMA in China has developed rapidly.
At present, a number of SMA diagnosis and treatment centers with multidisciplinary diagnosis and treatment capabilities and experience have been established in China
.

With the availability of more effective treatment drugs, the standardized diagnosis and treatment of SMA and the long-term management of the disease will gradually be improved.
The experience of SMA treatment in China will be further enriched, which will bring comprehensive benefits to SMA patients and families
.

" Zhou Zhou, President of Roche Pharmaceuticals China Ms.
Hong said: "Adhering to the concept of'Patients First', Roche has long been committed to the research and development of innovative drugs in the field of rare diseases and neurological diseases to meet the unfulfilled needs of patients
.

The approval of Aimanxin® in China demonstrates the determination and efforts of the Chinese government to accelerate the approval of new drugs and promote the development of rare disease diagnosis and treatment
.

In the future, we will continue to work closely with the government and various partners to bring more Roche neurological disease pipelines into China as soon as possible.
At the same time, we will also actively promote the development of SMA diagnosis and treatment
.

"Reference: [1]Kolb SJ and Kissel JT.
Spinal muscular atrophy.
Neurol Clin.
2015;33:831-46.
[2]Cure SMA.
About SMA.
2018.
Available from: http:// org/sma/about-sma/.
Accessed March 2019.
[3] Guidelines for the diagnosis and treatment of rare diseases (2019 edition) [4] Expert consensus on multidisciplinary management of spinal muscular atrophy.
Chinese Medical Journal.
2019.
99(19): 1460 -1467.
[5]Baranello G, et al.
N Engl J Med.
2021 Mar 11;384(10):915-923.
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