China Business Times | Frost & Sullivan: Capital Continues to Pour In, Driving the Development of the Rare Disease Industry, and More Patients with Rare Diseases Will Benefit from It

China Business Times | Frost & Sullivan: Capital Continues to Pour In, Driving the Development of the Rare Disease Industry, and More Patients with Rare Diseases Will Benefit from It

2022/10/19

AstraZeneca's Rare Diseases Business UnitAlexion Pharmaceuticals(Later referred to as 'Ali Brothers Pharmaceutical') announced that6,80010,000 US dollars (about4.84RMB 10 billion) to acquire a clinical-stage gene companyLogicBio TherapeuticsIn recent years, with policy support and market demand, many pharmaceutical companies including Novartis and Takeda are accelerating their layout in the field of rare diseases.


Why do large companies choose to layout in the rare disease field through mergers and acquisitions? In recent years, the research and development of rare disease drugs has become a hot area for pharmaceutical companies to compete in. With continuous capital inflows, what impact will this have on the entire industrial chain?  Frost & SullivanFrost & SullivanLi Qian, Senior Consulting Director for Healthcare in Greater China at Frost & Sullivan (referred to as 'Frost & Sullivan'), was interviewed by Huaxia Times to discuss the reasons and impacts of large enterprises deploying in the rare disease space.


Huaxia Times Net

Multinational pharmaceutical giants are increasing their investment in the field of rare diseases.


Eastern US Time10month3AstraZeneca's Rare Diseases Business UnitAlexion Pharmaceuticals(Later referred to as 'Ali Brothers Pharmaceutical') announced that680010,000 US dollars (about4.84RMB 10 billion) to acquire a clinical-stage gene companyLogicBio TherapeuticsOn the same day, Pfizer announced that it will116US$100 million acquisitionBiohavenThe company, just two days later, Pfizer announced again with54US$100 million acquisitionGBTThe company has been interpreted by the outside world as Pfizer beginning to seek new assets in the biopharmaceutical field to expand its pipeline and capture the rare hematology market.



In fact, Merck Sharp & Dohme, Novartis Pharmaceuticals, and GlaxoSmithKline have been investing heavily in rare disease areas over the past year through acquisitions. Domestic pharmaceutical companies such as Beijing Kangcheng Biotechnology Co., Ltd., Boya Yijing Biotech Co., Ltd., Conti Biotech Co., Ltd., Shanghai Zhongqiang Pharmaceutical Co., Ltd., Shufang Medicine Co., Ltd., and Deyi Sunshine are also actively deploying rare disease R&D pipelines.

"In recent years, rare diseases have become a popular investment area, with mergers and acquisitions worth hundreds of billions frequently occurring." Li Qian, senior consulting director for healthcare at Frost & Sullivan, said in an interview with the China Times that pharmaceutical companies will face the crisis of product patent expiration and need to expand their product pipeline to achieve sustainable growth, with mergers and acquisitions being one solution. The continuous influx of capital will drive the development of the rare disease industry, benefiting more patients with rare diseases.

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Increase investment

Recently, AstraZeneca and its subsidiary AstraZeneca Oncology announced the acquisitionLogicBio TherapeuIt is reported that this acquisition,LogicBioIt will bring unique technologies, an experienced rare disease team, and expertise in preclinical development to support the development of Genomics Medicine at Alisheng Pharmaceutical.


Public information shows,LogicBioEstablished2014Genome Therapeutics, a clinical-stage gene therapy company located in Lexington, Massachusetts, treats a variety of genetic diseases including rare disorders. At present, it is mainly focused on developing novel gene therapies for children with rare diseases.

LogicBioA technology platform for delivering and inserting genes to treat genetic diseases, as well as a platform for improving the manufacturing process of viral vectors, have been developed. These platforms, together withLogicBioAn experienced team, along with the progress of Brother Alistair and AstraZeneca, will drive future scientific possibilities and the development of next-generation drugs for treating rare genetic diseases.


Alexandre Dunoire, CEO of Alistair Pharma, said: 'The proposed acquisitionLogicBioIt represents a significant development for our growing genomic medicine research.LogicBioThe personnel, experience, and platform have provided new scientific capabilities by integrating first-class technology and expertise into our genomics strategy. Since the acquisition last year, scientific collaboration between Alexion and AstraZeneca has been an important area of focus.LogicBioThe addition will expand this foundational work.


Coincidentally, another multinational pharmaceutical giant, Pfizer, has also10month3Riyo5Today, it was announced that116US$100 million acquisitionBiohavencompany, with54US$100 million acquisitionGBTcompany.

It is understood thatBiohavenA neurology drug development company, specializing in migraine medicationsNurtec ODTat2022Net revenue in the first quarter of the year reached1.24billion US dollars, Pfizer's acquisition is mainly to secure this drug. After the acquisition is completed,BiohavenThe company's medical business for amyotrophic lateral sclerosis (ALS) and obsessive-compulsive disorder (OCD) will be spun off into a new entity.BiohavenThis acquisition is2022One of the largest pharmaceutical mergers and acquisitions globally.


GBTThe company was established in2011In [year], research and development of drugs for the treatment of sickle cell disease were initiated.2019year,GBTSickle cell disease treatment drugsOxbrytaApproved in the United States, with two other drugs in3 - 4The clinical phase is ongoing. This acquisition by Pfizer will strengthen its business in the rare disease space.


In fact, in recent years, with policy support and market demand, major pharmaceutical companies are accelerating their layout in the field of rare diseases.

Earlier this year, GlaxoSmithKline took the lead.2022year4Month, GlaxoSmithKline with19$100 million acquisition of rare cancer targeted therapy companySierra.Sierra OncologyFocus on the development of targeted therapies for rare tumors, with its main candidate productsMomelotinibAlready this year6Towards the United StatesFDAThe submission of a new drug application for the treatment of myelofibrosis is expected to be2023went public in the United States last year. At the same time, GlaxoSmithKline expects to2022Submit a new drug application for the product in Europe in the second half of the year.

2021year11Month, Merck Sharp & Dohme announced that115Billion dollars to complete the acquisition of a rare disease new drug R&D companyAcceleron PharmaThe acquisition, it is reported,AcceleronIt is a clinical-stage biopharmaceutical company focusing on the research and development of anti-cancer drugs and rare medicines for rare diseases.


2021year12In January, Merck Sharp & Dohme acquired a drug research and development company focused on rare neuro-inflammatory diseasesChord TherapeuticsWith this acquisition, Merck Sharp & Dohme will gain access to the treatment for neuromyelitis opticaobstacleNMOSD) and myasthenia gravisMGpipeline drugsCRD1Further strengthen the layout in the field of rare diseases.

2021year12In January, Novartis Pharmaceuticals announced the acquisition of a neuroscience drug developerCadent TherapeuticsThe maximum acquisition amount will reach7.7billion US dollars. As part of the acquisition, Novartis has receivedCadentRare disease productsCAD-9303andCAD-1883, whereinCAD-9303For schizophrenia,CAD-1883For movement disorders (e.g., cerebellar ataxia).

Earlier,2019year1month8Today, Takeda Pharmaceutical invested640Acquired US-based Therapeutics Moderna for $1000 million, becoming the largest company in rare diseases.

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M&A in the Blue Ocean

Rare diseases, also known as 'orphan diseases', refer to those with extremely low incidence rates, typically chronic and serious conditions that often endanger the lives of patients. According to the American Rare Diseases Organization(NORD)Official website. Currently, there are more than rare diseases known globally70,000species, and the number of rare disease patients has exceeded3billion, of which50%The patient is a child.


Due to the limited number of applicable populations and high R&D costs, pharmaceutical companies rarely invest funds in research and development production. Therefore, rare disease drugs are also known as 'orphan drugs'.


 Frost & Sullivan's 'Frost & Sullivan'2022The 'China Rare Disease Industry Trend Observation Report' points out that in recent years, capital has continuously flowed into the research and development of rare disease drugs, innovative biotechnology platforms, internet technology, and innovative medical services, promoting the construction of a rare disease ecosystem. Resources are collaborating with each other, injecting vitality into the rare disease industry chain.


Meng Lilian, the chief expert at Sichuan Tianfu Health Industry Research Institute, pointed out in an interview with a reporter from China Times that although rare diseases seem to have a large population base, they are a general term for all rare conditions, and the number of people affected by each rare disease is not very large. People suffering from rare diseases need effective medications, which is their right as well as the obligation of the state, enterprises, and relevant parties. With the gradual improvement of various conditions, the development of rare disease drugs has received attention, and capital from all sectors is gradually entering the field of rare diseases. This is the result of joint attention and anticipation from all parties.


Deng Zhidong, General Manager of Hainan Boao Medical Technology Co., Ltd., said in an interview with a reporter from China Times that globally, the total number of rare disease patients has exceeded3hundreds of millions, and the number of rare disease patients in our country is about2000Ten thousand, and the number of new patients each year exceeds20Ten thousand, expected to arrive2024In [Year], the global market size for rare disease medications will reach2420billion dollars.


He further pointed out that in recent years, the research and development of drugs for rare diseases has become an important area in international new drug research and development. With social attention and policy support, the market for rare disease drugs is promising for the future.


 *This article is reprinted from China Times.  Reporters: Sun Mengyuan, Yu Na  Original title:   In the rare disease sector, three mergers and acquisitions were announced intensively in a week. Behind the pharmaceutical giants' increased investment in orphan drugs   ">


Recommended Reading

 👉International Rare Diseases Day|Frost & Sullivan, in collaboration with the Pledge to End Violence Against Women, has released2022China Rare Diseases Industry Trend Observation Report


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