2022 global new drug inventory: 'First-in-class' therapy accounted for more than half, cell and gene therapy explosion!

In 2022, the global macro economy faced challenges, coupled with the new crown epidemic, the pharmaceutical industry experienced a difficult year.

However, looking back over the past year, we find that a number of innovative therapies have been approved by regulators, and 2022 is still a bumper year for innovative medicines.

The US FDA approved 37 new drugs, and "first-in-class" therapy accounted for more than half

In 2022, the FDA's Center for Drug Evaluation and Research (CDER) approved 37 new drugs, including 22 small molecule drugs and 15 biologic drugs.

Compared with the nearly 50 new drugs approved by the FDA each year in previous years, the number of new drugs approved this year has decreased, but the pace of innovation has not slowed down. According to statistics, in 2022, CDER approved 21 "first-in-class" therapies, more than half of the total number of approved new drugs.

The number of new drugs approved by the US FDA over the years

IN TERMS OF COMPANIES, BRISTOL-MYERS SQUIBB (BMS) WAS THE BIGGEST WINNER, WITH 3 NEW DRUGS APPROVED THROUGHOUT THE YEAR, INCLUDING SOTYKTU, CAMZYOS AND OPDUALAG. In addition, Roche and Sanofi each have 2 new drugs approved for marketing.

From the perspective of disease fields, among the new drugs approved by the FDA in 2022, oncology drugs accounted for the highest proportion (30%), with 11 models, involving a variety of common tumors and rare tumors; This is followed by rare diseases, autoimmunity, infections, etc.

From the perspective of drug types, the new drugs approved by the FDA in 2022 are:

1. 17 small molecule drugs: including pyruvate kinase allosteric activator, myomyosin allosteric inhibitor, TYK2 allosteric inhibitor and HIV-1 capsid inhibitor;

2. 2 contrast agents: gadolinium complex contrast agent, superpolarized Xe 129 contrast agent;

3. 6 monoclonal antibodies: including the first C1s antibody, the first CD3 antibody to delay the occurrence of type 1 diabetes, the first LAG-3 antibody, the first IL-36R antibody, etc.

4. VEGF-A/Ang-2、CD3/BCMA、CD20xCD3

5. 3 other proteins, 1 ADC, 1 polypeptide, 1 cyclic peptide, 1 RNAi therapy, 1 phytopharmaceutical product.

Note: The list includes a number of new drugs and selected biologics approved by CDER, excluding vaccines, allergy products, blood and blood products, plasma derivatives, cell and gene therapy products, and other products approved by the Center for Biologics Evaluation and Research in 2022.

Data source: US FDA official website, PharmaSmart US Listed Drug Database

In addition to CDER, the US FDA's Center for Biologics Evaluation and Research (CBER) has also approved 15 Biologics Licensing Applications (BLAs) involving a number of blockbuster new drugs, including Legend Biologics' BCMA CAR-T and a variety of gene therapies.

A number of innovative therapies were the first to be launched in the European Union and Japan

In 2022, a number of new drugs were also approved outside the United States (mainly the European Union and Japan), of which 8 of the world's first new drugs were recommended by the EU CHMP, and Japan's PMDA also approved more than ten of the world's first new drugs.

In 2022, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) completed the review of 92 new drug marketing applications, of which 89 new drugs were positively opinionated, including 51 new drugs, including 8 of the world's first batch of new drugs.

Among the first 8 new drugs, 7 are biological drugs, including 2 gene therapy drugs, 1 cell therapy drug, 3 antibody drugs and 1 protein drug, and only 1 chemical small molecule drug, Sunlenca, a new HIV drug from Gilead.

In 2022, CHMP recommended the launch of 3 bispecific antibody drugs, two of which are the world's first first-in-class products, namely the CD20 x CD3-targeted bispecific antibody Lunsumio (mosunetuzumab) developed by Roche and the first fully humanized BCMA x CD3-targeted bispecific antibody Tecvayli (Teclistamab) developed by Johnson & Johnson, all indications for hematological tumors.

In addition, in 2022, Japan's Pharmaceuticals and Medical Devices Agency (PMDA) approved the marketing authorization of more than 100 new drugs (including chemicals, biological products, excluding cell therapy and gene therapy products), of which Japan's PMDA approved more than 10 drugs containing new and effective ingredients for the first time in the world.

Data source: PMDA official website, PharmaSmart Japan Listed Drug Database

The Pharmaceutical and Food Administration of the Ministry of Health, Labor and Welfare of Japan stipulates in the documents related to the drug marketing application that drugs containing new active ingredients refer to active ingredients contained in drugs that contain different from drugs that have obtained marketing authorization in Japan (including drugs in the Japanese Pharmacopoeia). Drugs that contain esters or salts of active ingredients that have already been marketed in Japan also contain new active ingredients, such as otaltampan phosphate sodium phosphate for injection (sodium phosphate salt of tolvaptan that has been approved in Japan).

Unlike the European Union, most of the drugs containing new active ingredients approved by Japan's PMDA in 2022 are small molecule drugs, and there are two antibodies and two new protein drugs.

Cell and gene therapy has exploded

2022 will not be easy for the global biomedical industry, and it can be seen from the above that the number of new drugs approved last year has decreased compared with previous years. However, it is worth noting that the number of cell and gene therapies approved in 2022 reached a record high, with a total of 8 models.

Data source: EMA official website, FDA official website

Innovative therapies represented by cell and gene therapy have changed the traditional medical model and given many patients suffering from fatal diseases new hope. After several years of development, the development of this field has begun to take shape.

In 2022, the US FDA approved 4 gene therapies. From the point of view of indications, the treatment of rare diseases is the mainstay. For example, Bluebird Bio's Zyntegro is priced at $2.8 million in the United States, Skysona is priced at $3 million, and CSL Behring's Hemgenix is priced at $3.5 million, making it the world's most expensive drug.

It is worth mentioning that a gene therapy for the treatment of bladder cancer was also approved in 2022, indicating that this type of therapy is expected to break through the limitations of rare diseases and benefit more patients.

In 2022, the US FDA also approved 1 cell therapy, from the Chinese local company Legend Biotechnology, which is also the first domestic CAR-T therapy approved by the FDA.

In the EU, cell and gene therapy are included in the management of advanced therapeutic medical products (ATMP), and in 2021, the EU updated and improved the guidelines for the development of gene and cell therapy drugs to actively guide and support the development of ATMP.

In 2022, the EU approved 3 gene therapy drugs and 3 cell therapy drugs, of which 2 gene therapy drugs and 1 cell therapy drug are among the first new drugs in the world. They are: Upstaza (eladocagene exuparvovec) for the intracerebral injection gene therapy developed by PTC Therapeutics for the treatment of rare disease aromatic L-amino acid decarboxylase (AADC) deficiency, and Roctavian (valoctocogene roxaparvovec) for severe hemophilia A gene therapy developed by BioMarin Pharmaceutical. and Atara Biotherapeutics and Pierre Fabre, the allogeneic T cell therapy Ebevallo (tabelecleucel).

It is worth mentioning that unlike the highly customized autologous CAR-T therapy on the market, Ebvallo uses EBV T cells from donors, which can be quickly delivered to patients within three days, and if it is finally approved, it will be a milestone in the commercialization of universal cell therapy.

Conclusion

Innovation is the eternal theme of drug development. In the past few years, the form of drugs has ushered in rapid development, and new therapies such as cell therapy and gene therapy have begun to revolutionize the understanding of previous treatments, bringing the dawn of treatment to patients with rare and refractory diseases.

Back to China, what new drugs will be approved for marketing in China in 2022? Welcome to pay attention to Boyao, and continue to interpret it for you in the next issue~