2020 is coming to an end. As of press time, the Center for Drug Evaluation and Research (CDER) of the US FDA has approved 53 innovative drugs. In 2020, when the epidemic has become the global focus, FDA's work in approving COVID-19 innovative therapies, tests and vaccines may be more interesting. In this regard, as of December 22, the FDA has issued emergency use authorization (EUA) for 306 COVID-19-related sampling and testing methods, and has also approved the first innovative drug Remdesivir for the treatment of new coronavirus infections. . This month, two new coronavirus vaccines based on mRNA technology have also received EUAs issued by the FDA, bringing hope to the progress of COVID-19 control.
As the review work related to the epidemic occupies a lot of FDA's time and energy, whether the routine review of innovative drugs can be completed in time is one of the focuses of the industry. The data at press time indicate that the FDA has given satisfactory answers in reviewing the marketing of innovative drugs.
So far, the number of innovative drugs approved by CDER this year only lags behind the record number of 2018 (59 models), ranking second in history.
Orphan drug approval rate hits a 10-year high
In the past 10 years, the proportion of orphan drugs approved by the FDA in the approved innovative therapies has gradually increased. So far, 32 innovative therapies that have obtained orphan drug qualifications have been approved this year, accounting for 60.4% of new drugs approved throughout the year. In terms of proportion, it has reached a new high in the past 10 years, which is higher than the peak in 2018 (57.6%).
The development of orphan drugs for rare diseases not only benefits from the incentives of various regulatory measures issued by the FDA, but also reflects the importance of the pharmaceutical industry on the development of rare disease treatments. Many of the orphan drugs approved this year are for rare diseases with few patients. For example, Koselugo (selumetinib), the first drug therapy for type 1 neurofibromatosis, Viltepso (viltolarsen), an oligonucleotide therapy for Duchenne muscular dystrophy (DMD), and Evrysdi, the first oral innovative drug for the treatment of spinal muscular atrophy (Risdiplam) Wait.
On the other hand, with a better understanding of the molecular biology of cancer, researchers can develop targeted and precise therapies based on specific mutations carried by tumors. The cancer patients are subdivided according to molecular biological characteristics, so that the number of patients treated has also reached the criteria for obtaining orphan drug qualifications, which has also increased the number of orphan drugs approved. This year the FDA has approved a number of anti-cancer drugs targeting specific molecular biological characteristics, including Ayvakit (avapritinib) and RET inhibitor Gavreto (pralsetinib) developed by Blueprint Medicines, and Tabrecta (capmatinib), a MET inhibitor developed by Novartis. RET inhibitor Retevmo (selpercatinib) developed by Loxo Oncology, a subsidiary of Lilly, etc. This type of precision therapy for specific gene mutations often achieves better results while reducing adverse reactions caused by drugs.
The rise of innovative treatment models
In addition to traditional small molecule drugs and monoclonal antibody therapies, this year the FDA also approved a number of new therapies with innovative treatment models. Oxlumo (lumasiran) developed by Alnylam Pharmaceuticals became the third RNAi therapy approved by the FDA in three years. The CAR-T therapy Tecartus (brexucabtagene autoleucel) developed by Kite Pharma, a subsidiary of Gilead Sciences, has also become the third CAR-T therapy approved by the FDA.
Looking back on 2020, the FDA approved a total of 6 drugs with new treatment models (defined as antibody-conjugated drugs + gene therapy + cell therapy and oligonucleotide therapy), which is almost the same as the number of approvals in 2019 (7 models). Considering that the new crown epidemic may have affected Novartis' RNAi therapy inclisiran and Bristol-Myers Squibb's CAR-T therapy liso-cel this year, the FDA still maintains a good momentum in approving innovative model drugs.
The increase in the number of approved therapies with innovative treatment models also reflects the importance placed on these treatment models by the biomedical industry. According to the 2019 R&D Achievements report released by IQVIA, next generation Biotherapeutics (NGB) represented by cell therapy, gene therapy and oligonucleotides have developed rapidly in the past five years. In 2019, 99 products under development entered the late-stage clinical research and development pipeline, raising the number of products under development to 369, which is a three-fold increase compared with 2014.
Moreover, these NGB therapies are more likely to be approved by the FDA without undergoing phase 3 clinical trials than other traditional therapies under investigation. Because the FDA believes that these treatments under investigation are designed to treat patients with serious unmet medical needs. This means that although the number of NGB therapies entering phase 3 clinical trials is relatively small, they may be closer to being approved for marketing than non-NGB therapies, which will benefit patients sooner.
In terms of antibody-conjugated drugs (ADC), recent improvements in ADC construction technology have effectively increased the benefit/risk ratio of drugs. It has made considerable progress in the design of cytotoxic molecules and the stability of linkers connecting antibodies and loads. improvement. This field has also become a hot field for the development of innovative therapies in recent years. Statistics on the ADC R&D pipeline show that there are currently hundreds of ADC therapies in the clinical development stage, treating about 400 indications.
Compared with traditional small molecule and antibody therapies, some of these innovative treatment models can integrate the advantages of traditional therapies (for example, ADC can combine the specificity of antibody therapy and the killing effect of chemotherapeutics), and some can target the difficulties of traditional therapies. Targeting the target, and providing one-time treatment, the possibility of a lifetime benefit of cure. At the BIO 2020 conference held this year, Dr. James Sabry, head of Roche's global medical cooperation, said that in 30 years, cell and gene therapy will become the mainstream of future therapy!
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