Pfizer, Novartis, Sanofi, Takeda, Teva and many other rare disease new drugs have been listed in China and have been included in medical insurance

3.
On December 19, 2020, Takeda China announced that the management tool myPKFiT for hemophilia A (congenital factor VIII deficiency) under the guidance of its pharmacokinetics (PK) was officially launched for familiarization with the treatment of hemophilia A Used by licensed medical professionals (HCP) to assist patients with hemophilia A who are 16 years of age and older (weight 45 kg and above) to receive perinezhi (recombinant human coagulation factor VIII for injection)
.
myPKFiT is China's first and currently the only approved NMPA support hemophilia A pharmacokinetic (PK) under the guidance of management tools
.

| Teva

On January 17, 2021, Teva Medical Information Consulting (Shanghai) Co.
, Ltd.
announced that its innovative drug Antaitan (deuterium tetrabenazine tablets) was launched in China for the treatment of chorea and adults related to Huntington's disease (HD) Tardive dyskinesia (TD)
.
In addition, Antaitan was officially included in the "National Basic Medical Insurance, Work Injury Insurance and Maternity Insurance Drug List (2020)" on December 28, 2020, which will greatly improve the treatment burden of patients and increase the availability of innovative therapies
.
Huntington's disease is a rare and fatal neurodegenerative disease that affects approximately 0.
4 people per 100,000 people in Asia
.

| Italy Topei Pharmaceutical

On February 3, 2021, the Zhongshan Ophthalmology Center of Sun Yat-sen University, Guangzhou, China realized the first patient prescription of Senejimin eye drops (Ou Shiwei)
.
Ou Shiwei has been approved for marketing by the National Medical Products Administration of China on August 12, 2020.
It is used for adult patients with moderate or severe neurotrophic keratitis.
As a monotherapy, it brings avoidable invasiveness to patients.
A new treatment option for surgery
.
Neurotrophic keratitis is a rare progressive ophthalmological disease with complex and insidious causes, easy to be missed and misdiagnosed, and easy to recur
.

Cooperative introduction to meet the needs of patients

| Hong Kong Weijian Pharmaceutical and Taiwan Ancheng Biological

On January 7, 2021, Hong Kong Weijian Pharmaceutical Group and Taiwan Ancheng Biotechnology Co.
, Ltd.
announced a strategic cooperation to authorize Hong Kong Weijian Pharmaceutical Group to develop rare disease product AC-203 in mainland China, Hong Kong and Macau.
This product The main indication is hereditary epidermolysis bullosa (EB), and other indications include bullous pemphigoid and other skin diseases
.
Hereditary epidermolysis bullosa (EB) is a group of rare inherited skin diseases.
Due to genetic mutations, the skin is very fragile, just like the wings of a butterfly.
A slight collision or friction will produce blisters or blood bubbles, and then trauma.
Fester
.

| Hong Kong Weijian Pharmaceutical and CLINUVEL

On April 24, 2020, Hong Kong Weijian Pharmaceutical Group and CLINUVEL signed a cooperation agreement for the erythropoietic porphyria (EPP) therapeutic drug SCENESSE.
The two companies have reached cooperation for the first time in the field of rare diseases
.
CLINUVEL (headquartered in Australia) will launch SCENESSE (Alphanopeptide 16mg) in China through Weijian Pharmaceutical and its subsidiaries for the treatment of rare genetic metabolic disorders-erythropoietic porphyria (EPP)
.
EPP is a genetic disease.
Once a patient is exposed to visible light, especially sunlight, exhaustive burns of the skin and internal tissue damage will occur, causing severe skin pain and disease
.

Agency takes the lead to explore new solutions for rare diseases

In September 2020, the rare disease registration and diagnosis and treatment standard training of the National Rare Disease Diagnosis and Treatment Network was successfully held in Shanghai
.
During the meeting, Weisheng Pharmaceutical and China Rare Disease Alliance formally signed a strategic cooperation agreement.
In the next 5 years, the two parties will carry out a series of measures in the field of achondroplasia (ACH) to improve the diagnosis and treatment status of China's ACH and improve the treatment of rare diseases in China.
In particular, the cognition of ACH has promoted the improvement of the overall level of diagnosis and treatment of rare diseases in China, and ultimately improved the diagnosis and treatment plan and quality of life of patients with rare diseases in China
.

In October 2020, the Porphyria MDT of the Second Hospital of Hebei Medical University was established
.
The team was initiated by Professor Zhang Songyun from the Department of Endocrinology.
Gastroenterology, Neurology, Emergency Department, Critical Care Medicine, Dermatology, Pain, Hemodialysis, Gynecology, Reproductive Medicine, Immunorheumatology, Urology, Pharmacy, Specialty 15 majors including examination department and imaging department participated in the formation
.