There is a large unmet clinical demand for rare disease drugs, and multinational pharmaceutical companies continue to enter the market

 There is a large unmet clinical need in the rare disease market, both in China and overseas
.
Data shows that the global rare disease market has continued to heat up in recent years.
In 2020, the market size of rare disease drugs will reach 135.
1 billion US dollars.
It is estimated that in 2030, this market size may reach 383.
3 billion US dollars, with a compound annual growth rate of 11%
.
Facing the huge market potential, more and more multinational pharmaceutical companies are now entering the market
.
Take Merck as an example.
In September 2021, Merck acquired Acceleron, a pharmaceutical company, for $11 billion.
According to data, Acceleron is a clinical-stage biopharmaceutical company focusing on the research and development of anticancer drugs and orphan drugs for rare diseases
.
This acquisition also marks an important layout of Merck in the field of rare diseases
.
In addition to Merck, multinational pharmaceutical companies are increasingly taking measures in the field of rare diseases
.
For example, in August 2021, Roche Pharma spent US$3 billion to reach a multi-target strategic cooperation and licensing agreement with Shape Therapeutics to jointly develop gene therapy drugs targeting specific targets in Alzheimer's disease, Parkinson's disease and rare diseases
.
In December 2020, AstraZeneca acquired rare disease company Alexion for US$39 billion; in February 2019, Roche Pharmaceuticals acquired gene therapy pioneer Spark for US$4.
3 billion; in April 2018, Takeda Pharmaceutical acquired rare disease company Xia for US$65 billion Er et al
.
It is reported that Pfizer is also relatively early in the treatment of rare diseases, with the main orphan drug Vyndaqel (tafamidis) for the treatment of transthyretin-related familial amyloid polyneuropathy (ATTR)
.
At present, Pfizer's listed rare disease products mainly cover three areas: hemophilia, transthyretin amyloidosis polyneuropathy and adult wild-type or genetic transthyretin amyloidosis cardiomyopathy
.
Novartis is a very large pharmaceutical company in the field of rare diseases.
Data shows that in 2018, Novartis accounted for 11.
3% of the market share in the orphan drug market.

.
Its main orphan drug, Tafinlar (dabrefenib), is a B-Raf inhibitor used as a single agent or in combination with trametinib for the treatment of advanced melanoma patients with BRAFV600E or V600K mutations; in combination with trametinib, it can also treat patients with BRAFV600E Patients with mutated metastatic non-small cell lung cancer
.
Bristol-Myers Squibb, as an early multinational pharmaceutical company in the field of rare diseases, has also made continuous efforts in the research and development of rare diseases in recent years
.
For example, in December 2021, the FDA approved Bristol-Myers Squibb's Orencia (abatacept) for the prevention of acute graft-versus-host disease (aGVHD); in February 2021, Bristol-Myers Squibb and Sutro Biopharma (STRO.
US) jointly developed The product CC-99712 therapy has been granted orphan drug designation by the FDA for the treatment of multiple myeloma,
etc.
While multinational pharmaceutical companies are scrambling to enter rare disease drugs, the domestic market is also increasing their support for rare disease drugs
.
For example, in May 2018, the "First Batch of Rare Disease Catalog" was published in China, including 121 rare diseases; in September 2021, the "Research Report on the Definition of Rare Diseases in China 2021" was released, which clarified the definition of rare diseases
.
According to the industry, although there is no rare disease certification for drug development in China at present, relevant policies have been introduced to encourage the development of drugs for rare diseases.
The review time limit for overseas marketed rare disease drugs is 70 days; you can apply for the "Clinical Urgently Needed Overseas New Drug Review and Approval Work Procedures", overseas marketed rare disease drugs; accelerated access to medical insurance,
etc.
Driven by favorable policies, the domestic rare disease drug market has also begun to develop continuously
.
Data show that in 2016 and 2020, the Chinese rare disease drug market accounted for 0.
4% and 1.
0% of the global rare disease market, respectively.
It is estimated that the domestic orphan drug market will reach US$6.
4 billion in 2025
.
Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice to anyone
.