$124 million!

Rare disease is a disease with a low incidence.
At present, there are about 20 million patients with various rare diseases in China, and more than 200,000 new patients are diagnosed every year
.
With the growing number of patients, there is a huge unmet medical need in this field of medicines
.
However, it should be noted that due to the low investment in scientific research and the low diagnosis rate, rare disease drugs have long been characterized by low market demand, high research and development costs, and high costs
.
In order to solve the dilemma of "no medicine available" and "can't use medicine" for rare disease patients, in recent years, relevant national departments have attached great importance to the diagnosis, treatment and research and development of rare diseases, and have continued to issue rare disease catalogues, rare disease diagnosis and treatment guidelines, and priority review.
Approval, the inclusion of rare disease drugs into the scope of medical insurance and other policy measures to continuously accelerate the research and development of rare disease drugs, as well as the review and approval procedures for drug listing and import
.
At present, with a series of favorable policies, domestic pharmaceutical companies are accelerating the development of drugs for rare diseases, and the speed of importing drugs for rare diseases into China is also accelerating
.
For example, some time ago, the State Drug Administration approved the import registration application of siltuximab for injection (English name: Siltuximab for Injection), a rare disease drug urgently needed in clinical practice, through the priority review and approval process, for human immunodeficiency virus ( HIV)-negative and human herpesvirus-8 (HHV-8)-negative adult patients with multicentric Castleman disease (MCD)
.
It is worth mentioning that in recent days, there has been good news from domestic pharmaceutical companies on new drugs for rare diseases
.
It is understood that on January 4, Shufang Pharmaceutical and Santhera Pharmaceuticals announced that they had reached an exclusive licensing agreement for the new rare disease drug Vamorolone
.
Specifically, the agreement shows that Shufang Pharmaceutical has obtained the exclusive right to develop and commercialize Vamorolone in Greater China for Duchenne muscular dystrophy and other rare disease indications
.
The former will pay an upfront payment in the tens of millions of dollars and milestone payments related to U.
S.
registration filings, totaling $20 million
.
Also, double-digit percentage royalties will be paid
.
Duchenne muscular dystrophy (DMD), which Vamorolone targets, is a rare X-chromosome recessive disorder that affects almost exclusively men
.
The major milestones of the disease are loss of walking ability, loss of self-feeding ability, initiation of assisted ventilation, and development of cardiomyopathy
.
Due to respiratory and/or heart failure, people with DMD generally have a life expectancy of no more than 40 years
.
As early as 2018, Duchenne muscular dystrophy has been included in the national "First Batch of Rare Disease List", and there is a huge clinical demand in this disease field
.
Since there is currently no approved DMD treatment drug in China, the industry believes that in the future, Shufang Medicine will usher in huge development opportunities after Vamorolone is approved
.
In fact, for this cooperation, the relevant person in charge of Shufang Pharmaceutical also made it clear that he is very optimistic about the therapeutic potential of Vamorolone in the field of Duchenne muscular dystrophy and other rare diseases
.
He believes that the rich experience of the Shufang Pharmaceutical team in the registration, clinical and commercialization of rare disease products will help patients in Greater China to benefit from this differentiated product candidate as soon as possible
.