The Rare Disease Market Continues to Heat Up. How Will The Global Top 10 Pharmaceutical Companies by Revenue in 2021 be Deployed?

In recent years, the rare disease market has continued to heat up. 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 multinational pharmaceutical companies have entered the rare disease market one after another, and accelerated the "race to the ground" by acquiring and expanding the R&D pipeline of rare disease drugs.

Among the 2021 global revenue TOP10 pharmaceutical companies announced this year, almost all companies are actively deploying in the field of rare diseases.

01 Johnson & Johnson

Johnson & Johnson has been in the field of rare diseases for many years, and the main orphan drug Darzalex (daratumumab) is used to treat multiple types of multiple myeloma (MM) patients, including first-line treatment of MM patients who cannot receive hematopoietic stem cell transplantation.

In 2019, Johnson & Johnson entered the field of gene therapy, researching and developing gene therapy for rare genetic diseases. Janssen Pharmaceuticals, a Johnson & Johnson company, announced on January 31, 2019 that it has entered into a global collaboration and license agreement with clinical-stage gene therapy company Meiragtx Holdings plc (MGTX) to develop gene therapies for the treatment of rare inherited retinal diseases (IRDs).

02 Pfizer

Pfizer has also been relatively early in the treatment of rare diseases, with the main orphan drug Vyndaqel (tafamidis) for the treatment of transthyretin-associated familial amyloid polyneuropathy (ATTR).

In 2019, Pfizer successfully completed the acquisition of the rare disease company Therachon Holding AG and obtained the rare disease drug TA-46.

In September 2021, Pfizer announced that it will adjust its organizational structure from December 1, 2021, and establish 6 independent business units. Rare disease is one of the independent departments and has become one of the important sectors of its development. At present, Pfizer's marketed rare disease products mainly cover three areas: hemophilia, transthyretin amyloidosis polyneuropathy, and adult wild-type or hereditary transthyretin amyloidosis cardiomyopathy.

03 Roche

Roche is one of the leading pharmaceutical companies in the field of rare diseases. The company's main orphan drug products are Hemlibra (emicizumab) and Rituxan (rituximab), which treat hemophilia A with FVIII inhibition deficiency and moderate to severe pemphigus vulgaris, respectively. Sores (PV).

In recent years, Roche has invested more and more in the research and development of rare disease drugs, and the research and development of rare disease drugs in its clinical pipeline has increased from 12.2% in 2017 to 16.3% in 2020.

In February 2019, Roche acquired Spark Therapeutics, a pioneer in gene therapy, for US$4.3 billion, and the gene drug Luxturna, which received FDA orphan drug designation, expanded its presence in the field of rare diseases by deploying gene therapy.

In 2022, two of Roche's anticancer drugs received orphan drug designation: glofitamab (formerly CD20-TCB) for the treatment of mantle cell lymphoma (MCL); pralsetinib for the treatment of metastatic RET fusion-positive solid tumors.

04 AbbVie

AbbVie’s layout in the field of rare diseases is also relatively early. At present, the main orphan drug is Imbruvica (billion, ibrutinib), which is used to treat a variety of B-cell blood cancers, including mantle cell lymphoma (MCL), chronic lymphocytic Leukemia (CLL), and Waldenstrom's macroglobulinemia.

Between 1983 and 2019, AbbVie received 24 orphan drug approvals. In 2020, AbbVie again received FGA Orphan Drug Designation and Fast Track designation for elezanumab (ABT-555), an investigational therapy for patients with spinal cord injury.

05 Novartis

Novartis is a very leading pharmaceutical company in the field of rare diseases. According to the data of pharmaceutical consulting company IgeaHub, in 2018, Novartis was relatively leading in the orphan drug market, accounting for 11.3% of the market share.

Novartis’ flagship orphan drug Tafinlar (dabrefenib) is a B-Raf inhibitor for the treatment of advanced melanoma patients with BRAFV600E or V600K mutations as a single agent or in combination with trametinib; in combination with trametinib, it can also treat patients with BRAFV600E Patients with mutated metastatic non-small cell lung cancer.

In December 2020, Novartis announced the acquisition of neuroscience drug developer Cadent Therapeutics for $770 million, acquiring Cadent's rare disease product CAD-9303 and product CAD-1883, CAD-9303 for schizophrenia; CAD-1883 for exercise Disorders (eg, cerebellar ataxia).

06 Merck & Co.

Merck's layout in the field of rare diseases is relatively late. In August 2021, the FDA approved the launch of Merck's innovative oncology drug MK-6482, and MK-6482 was granted orphan drug status by the FDA in 2020.

In September 2021, Merck announced the acquisition of Accelron for US$11.5 billion, starting to deploy in the field of rare diseases and enriching the drug pipeline in the field of rare diseases.

In December 2021, Merck announced the acquisition of Chord Therapeutics, a drug development company focusing on rare neuroinflammatory diseases. With this acquisition, Merck will acquire CRD1, a pipeline drug for the treatment of neuromyelitis optica spectrum disorder (NMOSD) and myasthenia gravis (MG). , to further strengthen the field of rare diseases.

07 Bristol-Myers Squibb

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. Its flagship orphan drug Yervoy (ipilimumab) is a monoclonal antibody targeting the immune checkpoint protein CTLA-4 for the treatment of unresectable or metastatic melanoma.

In January 2020, the red blood cell maturation agent Reblozyl (luspatercept) developed by Bristol-Myers Squibb and Acceleron Pharmaceuticals was granted orphan drug designation by the FDA for the treatment of myelofibrosis.

In February 2021, Bristol-Myers Squibb and Sutro Biopharma (STRO.US) jointly developed the CC-99712 therapy with the FDA orphan drug designation for the treatment of multiple myeloma.

In December 2021, the FDA approved Bristol-Myers Squibb's Orencia (abatacept) for the prevention of acute graft-versus-host disease (aGVHD), which was previously granted orphan drug designation by the FDA.

08 GlaxoSmithKline

GlaxoSmithKline entered the rare disease field earlier, but because of its poor performance in this field, it said in 2017 that it might split the rare disease business; in 2018, GSK and Orchard Therapeutics gradually transitioned the rare disease business to Orchard Therapeutics. However, from the back performance, GSK has not given up on the rare disease business.

In November 2020, Eidos Therapeutics received a takeover offer from GlaxoSmithKline, although it was ultimately unsuccessful. In April 2022, GlaxoSmithKline acquired Sierra, a rare cancer targeted therapy company, for $1.9 billion.

09 Sanofi

In early 2011, Sanofi acquired Genzyme, which is known as the "King of Orphan Drugs", at a high price of US$20 billion, which enabled Sanofi to quickly enter the orphan drug market. In recent years, Sanofi has gradually focused its business on the field of rare diseases. Its main orphan drug, Myozyme (alglucosidasealfa), is an enzyme replacement therapy used to replace acid alpha-glucosidase (GAA) for the treatment of rare diseases. Bay (Pompedisease).

In 2018, Sanofi acquired Bioverativ, a U.S.-specialized blood rare disease company, for $11.6 billion. Such a big deal plays an important role in strengthening its capabilities in rare disease treatment.

In June 2021, Sanofi and Magnesium Health held a strategic cooperation signing ceremony in Shanghai. The two parties will strengthen cooperation in the field of "Internet + Medicine + Medicine + Insurance" on innovative drugs in the fields of rare diseases, chronic diseases and tumors.

In April 2022, Sanofi launched an international multicenter Phase 3 clinical study of venglustat in patients with Fabry disease in China. Venglustat is an oral GCS inhibitor, and Sanofi is currently conducting Phase 2/3 clinical studies of the drug for a variety of rare disease indications.

10 AstraZeneca

As a "rookie" in the field of rare diseases, AstraZeneca has made frequent moves in the field of rare diseases in recent years.

In December 2020, AstraZeneca acquired Alexion, a company specializing in rare diseases, for US$39 billion, acquiring the complement technology R&D platform and R&D capabilities of rare diseases of Alexion Pharmaceuticals. Now the company is exploring various ways including ATTR amyloidosis. new treatments for rare diseases.

In September 2021, AstraZeneca China announced the establishment of the AstraZeneca China Rare Disease Business Unit.

In December 2021, AstraZeneca announced a global development and commercialization agreement with Ionis Pharmaceuticals for an investigational orphan drug eplontersen of over US$3.5 billion.

In March 2022, AstraZeneca announced that its rare disease company, Alexion, and Neurimmune have entered into a partnership to obtain a treatment for the rare disease transthyretin amyloid cardiomyopathy (ATTR) with an initial payment of US$30 million + milestone payments of up to US$730 million. -CM)'s global exclusive rights to the monoclonal antibody drug NI006, which is currently in Phase Ib clinical stage.

At present, AstraZeneca's flagship orphan drug is Lynparza (olaparib), which is jointly developed by AstraZeneca and Merck for the treatment of ovarian, breast and prostate cancer patients with BRCA1 and BRCA2 gene mutations.

In addition to the above-mentioned pharmaceutical companies actively deploying in the field of rare diseases, Takeda and Bayer are also leading pharmaceutical companies in the field of rare diseases.

In particular, Takeda has become the number one giant in rare diseases since April 2018, when Takeda acquired the rare disease giant Shire in the UK. In recent years, Takeda has accelerated the introduction of 6 innovative treatments in the field of rare diseases into China, of which 2 innovative drugs for rare diseases have been successfully included in the 2021 National Medical Insurance List.

Rare diseases represent a highly developed disease field with serious unmet medical needs and huge commercial value. Therefore, in the future, rare diseases will become a key area for pharmaceutical companies to seek innovative breakthroughs and rapid rise.

With the increasing emphasis on rare diseases in my country, local pharmaceutical companies have also joined this field one after another to promote the research and development and innovation of domestic rare disease drugs.