Domestic and foreign pharmaceutical companies are rushing in, and the $100 billion market has become a "sweet pastry"

Rare disease drugs, also known as orphan drugs, have huge drug gaps due to difficulties in research and development, small audiences, high prices, and difficulties in promotion
.
However, in recent years, with the increasing attention of rare diseases in the industry and the continuous introduction of a series of favorable policies at home and abroad to encourage pharmaceutical companies to make drugs for rare diseases, the market is expanding
.
According to Frost & Sullivan, the global rare disease drug market will increase from US$135.
1 billion in 2020 to US$383.
3 billion in 2030, with a compound annual growth rate of 11%
.
Faced with such a huge market potential, more and more pharmaceutical companies have entered the rare disease market one after another, speeding up the "race to the ground" by acquiring and expanding the R&D pipeline of rare disease drugs
.
Among them, multinational pharmaceutical companies performed very well
.
It is reported that in December 2020, AstraZeneca acquired rare disease giant Alexion for US$39 billion, officially entering the rare disease market
.
Prior to this, in February 2019, oncology giant Roche acquired gene therapy pioneer Spark Therapeutic for US$4.
3 billion; in the same year, Pfizer also successfully completed the acquisition of rare disease company Therachon Holding AG and obtained the rare disease drug TA-46
.
In April 2018, Takeda Pharmaceuticals acquired rare disease giant Shire for $65 billion
.
In addition, AbbVie's layout in the field of rare diseases is relatively early
.
Between 1983 and 2019, AbbVie has approved 24 orphan drugs
.
In 2020, AbbVie once again received FGA Orphan Drug Designation and Fast Track designation for elezanumab (ABT-555), an investigational therapy for patients with spinal cord injury
.
At present, with the promotion and upgrading of drug administration reform and medical insurance payment, the domestic pharmaceutical industry has also begun to see the business opportunities of rare diseases.
are involved
.
It is worth mentioning that in addition to accelerating the deployment of the rare disease drug market, related drugs from Chinese pharmaceutical companies are also frequently going overseas
.
For example, Junshi Bio’s Toripalimab has successively obtained multiple orphan drug qualifications.
On November 16 last year, Junshi Bio released an announcement saying that Toripalimab for the treatment of esophageal cancer has been granted an orphan drug by the FDA Qualification
.
This is also the fourth FDA orphan drug designation for Toripalimab.
The previous indications for orphan drug designation are mucosal melanoma, nasopharyngeal carcinoma and soft tissue sarcoma
.
Incomplete statistics show that in 2020, 26 products of 19 Chinese companies have obtained 35 FDA orphan drug qualifications, accounting for 8% of the number of orphan drug qualifications granted by the FDA throughout the year
.
At present, in order to encourage the research and development and marketing of drugs for rare diseases, China is still issuing policies to support it, attracting overseas products to the domestic market, and increasing the importance of local pharmaceutical companies on the research of drugs for rare diseases
.
In this context, combined with the support of a series of powerful policies such as the acceleration of new drug approval and review, the industry expects that different rare disease drugs may be launched in the domestic market in the next five years
.
While further meeting the drug needs of patients with rare diseases, competition with multinational companies in this field will also become increasingly fierce
.
Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice to anyone
.