The rare disease drug market is "attractive", and the pharmaceutical company has spent tens of millions of dollars to re-layout

【Pharmaceutical Network Enterprise News】Rare disease drugs, also known as orphan drugs, have always had huge unmet clinical needs
in the market due to research and development difficulties, small audiences, high prices, and difficulties in promotion.
At present, in the face of huge market potential, more and more pharmaceutical companies are accelerating their layout
.
Recently, AstraZeneca and its subsidiary, Allemic Pharmaceuticals, agreed to acquire LogicBio Therapeu
for approximately US$68 million (approximately RMB484 million) per share.
Founded in 2014, LogicBio is a clinical-stage genomic drug company based in Lexington, Massachusetts, treating a variety of genetic diseases, including rare diseases, and is currently focusing on developing new gene therapies
for pediatric patients with rare diseases.
Analysts believe that this acquisition is another layout
of AstraZeneca in the field of rare diseases.
It is understood that the field of rare diseases is one of the important sectors of AstraZeneca's development, and it has been frequently
acting in the field of rare diseases in recent years.
On September 7, 2021, AstraZeneca China confirmed the establishment of the Rare Diseases Division to Cyberblue
.
On December 7, 2021, AstraZeneca announced a global development and commercialization agreement
with Ionis Pharmaceuticals for the latter's investigational antisense oligonucleotide (ASO) eplontersen (IONIS-TTR-LRX), in excess of $3.
5 billion 。 Following this, AstraZeneca also announced that its company Alexion (hereinafter referred to as "Alexion Brother"), which focuses on rare diseases, has reached an exclusive global cooperation and licensing agreement with Neurimmune (Swiss biotechnology) for NI006.
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It is worth mentioning that in China, the field of rare diseases has been mentioned by AstraZeneca as an important position
alongside tumors and the two major chronic disease plates.
The industry believes that as AstraZeneca continues to use its own technical advantages and market advantages to optimize the product structure and expand the product line, its sales and commercialization capabilities may bring new vitality and opportunities
to the further expansion of the rare drug market in the future.
In fact, at present, in China, more and more pharmaceutical companies have begun to enter the market
.
Pharmaceutical companies such as Beihai Kangcheng, Cantini Pharmaceutical, Deyi Sunshine Biological, Shufang Pharmaceutical, and Faith Pharmaceutical Technology have invested huge funds and energy
in the field of rare disease drugs.
It is worth mentioning that under the continuous investment in research and development, domestic pharmaceutical companies have also begun to usher in the field
of rare diseases.
It is reported that in September this year, the State Food and Drug Administration issued an announcement to approve the registration and listing of chlorobachan tablets independently developed by Yichang Renfu Pharmaceutical, and simultaneously issued the registration number of chlorbachan's APIs
.
Data show that clobachan tablets are rare disease drugs in children and are suitable for the combination treatment
of seizures in patients with Lennox-Gastaut syndrome (LGS) aged 2 years and older.
On July 21, Junshi Bio announced that its self-developed anti-PD-1 monoclonal antibody drug terreprimumab for the treatment of nasopharyngeal cancer has been granted orphan drug qualification by the European Commission
.
So far, the indications for nasopharyngeal carcinoma of terreprimab have been recognized as "orphan drugs" in the pharmaceutical markets of the two major developed countries in the
world.
On June 14, Hengrui Pharmaceutical announced that the company's Methopopopolamine tablets (thrombopoietin agonist TPO-RA) for the indications for thrombocytopenia caused by chemotherapy for malignant tumors were granted orphan drug qualification by the US Food and Drug Administration (FDA).
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In general, with the continuous expansion of the market size and the accelerated layout of large international pharmaceutical companies like AstraZeneca in this field, the research and development efforts and investment scale of domestic pharmaceutical companies in the field of rare disease drugs will not stop here, and there may soon be a larger-scale jump
.
At the same time, the domestic rare disease drug market may also accelerate in the
future.
It is estimated that the domestic orphan drug market will reach $6.
4 billion in 2025.

Disclaimer: Under no circumstances does the information herein or the opinions expressed in this article constitute investment advice
to any person.