In October, a number of multinational pharmaceutical companies launched large-scale acquisitions to increase the layout of rare diseases

According to the definition of the World Health Organization (WHO), rare diseases are diseases
in which the number of patients accounts for 0.
65‰ to 1‰ of the total population.
At present, there are more than 7,000 known rare diseases in the world, and the number of rare diseases is increasing
at a rate of 250 to 280 per year.
According to research data from Europe and the United States, there are currently about 270 million patients with rare diseases in the world, and these patients still have huge unmet clinical needs, and pharmaceutical companies at home and abroad are continuing to make efforts
to challenge this disease field.
Since October this year, a number of multinational pharmaceutical companies have launched large-scale acquisitions to increase the layout of
rare diseases.
For example, on October 3, Eastern time, Alexion Pharmaceuticals, a subsidiary of AstraZeneca's rare disease business, announced the acquisition of clinical-stage genetic company LogicBio Therapeutics
for $68 million 。 According to the data, LogicBio was founded in 2014 as a gene therapy development company spun off from the laboratory of Dr.
Mark Kay, professor of genetics at Stanford University and AAV vector expert, to treat various genetic diseases including rare diseases, and is currently mainly focusing on the development of novel gene therapies
for children with rare diseases.
LogicBio has two technology platforms: gene editing platform GeneRide and gene delivery capsid platform sAAVy
.
LogicBio has now laid out 10 product pipelines around these two technology platforms, which also shows that the company has strong potential
in developing genomic drugs.
Through this acquisition, AstraZeneca will further accelerate Alexion's rapid development in the field of genomic drugs through the accumulation of technology accumulation in genomics technology platform developed by LogicBio, rare disease R&D team and preclinical development
。 It is worth mentioning that for the rare disease business, AstraZeneca continues to acquire, after in December 2020, AstraZeneca acquired Alexion, a leading company in the development of rare disease therapies, for US$39 billion, making it a subsidiary of AstraZeneca focusing on rare disease business; In September 2021, AstraZeneca acquired Caelum BioSciences, a rare disease company, for $500 million and acquired an entire controlling stake in
the company.
Now AstraZeneca's acquisition of LogicBio may help the company take a further step
in the rare disease business.
In October, Pfizer's acquisitions in the field of rare diseases also continued, and it launched two acquisitions on October 3 and 5 local time, including: Alexion Pharmaceuticals, a subsidiary of its rare disease business, announced the acquisition of clinical-stage genomics company LogicBio Therapeutics for $68 million, and acquired Biohaven for $11.
6 billion on the same day; and the $5.
4 billion acquisition of GBT, which the industry sees as a signal
that Pfizer is looking for new assets in the biopharmaceutical field to expand its pipeline and seize the rare hematology field.
Previously, Pfizer also acquired rare disease company Therachon Holding AG and listed rare diseases as a separate division
in the December 2021 restructuring.
In recent years, rare diseases have attracted more and more attention from multinational pharmaceutical companies, and there have been frequent
acquisition news.
In addition to AstraZeneca and Pfizer, in 2011, Sanofi spent $20.
1 billion to acquire Genzyme; In 2019, Takeda announced the completion of the acquisition of Shire for USD 62 billion, entering the field of
rare diseases.
According to the "Rare Disease Drug Report" (2018) released by EvaluatePharma, the global rare disease drug market totaled $70.
3 billion in 2017 and is expected to increase to $262 billion
by 2024.
In China, the rare disease drug market has reached $1.
3 billion in 2020 and may increase to $25.
9 billion
in 2030.
In the face of a considerable blue ocean, not only multinational pharmaceutical companies, but also local pharmaceutical companies are also actively deploying the rare disease track, including traditional pharmaceutical companies and emerging innovative pharmaceutical companies such as Hengrui Pharmaceutical, Shanghai Pharmaceutical, Hansen Pharmaceutical, Sunflower Pharmaceutical, BeiGene, Junshi Biologics, and CANbridge, all of which are actively deploying
in the field of rare disease drugs 。 Among them, CANbridge has 13 innovative product pipelines for rare diseases with a large number of patients and rare tumor indications, and one of the three products on the market is the first rare disease drug approved for the treatment of MPSII (mucopolysaccharidosis type II) in China, meeting the treatment needs
of patients.
Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice
to anyone.