Source: Prism (ID: lengjing_qqfinance), Wall Street Journalist
Edit | Yang Wei
Recently treatment has been approved for types of rare tumors by the United States Food and Drug Administration (FDA) and has been interpreted to have a 75% improvement rate for multiple cancers. It has caused great concern among the people. However, this is not the case.
The approved drug, known as Larotrectinib (Vitkravi trade name) was developed jointly by the "medical specialist" Loxo Tumor and the "Bayer" traditional pharmaceutical maker. Bayer said the drug is the first anti-tumor chemical approved by the US Food and Drug Administration for "no difference between tumor types."
The US Food and Drug Administration has said that the approved drugs will prevent the unusual ban of the NTRK gene with other genes – a combination seen as a sign of growth that supports the development of a tumor. The combination of NTRK is not common, and usually only in body cancers, including lung cancer and thyroid cancer.
75% of the rate of improvement is chastised
According to Bayer, the overall response rate (ORR, translation response rate) was 75% amongst adults and children who were part of the experiment, including 22% of total relief and 53% Partial relief. The rate of leakage known refers to the proportion of people who have reduced or disappeared after the patient receives treatment, and does not refer to the proportion of the treatment.
According to the experimental data, a total of 55 patients participated in the three experiments. The US Food and Drug Administration said that the experimental response was sustainable: from an analysis of the experimental results, 73% of reliefs lasted for at least six months, and 39% of & # 39; The relief lasts for at least one year. The approved drugs are effective for soft tissue sarcoma, saline gland cancer, baby fibrosarcoma, thyroid cancer, and lung cancer.
Although new drugs that are approved can treat a variety of cancers, known as "broad spectrum anti-cancer drugs", they are only effective for some patients with specific characteristics at a genetic level. Therefore, the US Food and Drug Administration also provides her "orphanage qualification" – according to the relevant legislation, orphans can have convenient conditions such as fast approval, tax reduction, exclusion from reporting fees , research and development subsidies, etc., which will provide incentives for pharmaceutical companies To help and encourage the development of drugs to treat rare diseases.
In particular, only those patients with "NTRK born in tumors" are eligible for treatment with the new drug that has been approved. According to Bayer, the relevant diagnosis can be diagnosed using next generation sequence (NGS) technology and fluorescence in hybridization at the location (PYSG).
In a statement to the press, Bayer, Robert LaCaze, quoted his oncology strategy business unit, as he said, "The combination of NTRK is scarce, but it exists in many different types of tumor … for those with NTRK gene flu. It is very meaningful to provide a specialist treatment plan for patients with firm higher tumors. "
According to Bayer, the United States Food and Drug Administration "approves the general response rate and the length of non-drug-based drug treatment" and commends it for "adult solid tumor patients and children "by fast procedure. He also stated that "subsequent approval could rely on verifying and describing clinical benefits in a trial trial".
Commissioner of Food and Drug Administration of the United States Scott Gottlieb said the approval of the new drug reflects the increase of biomarkers in leading drug development and the use of more detailed drugs.
High price, hard to sell
Lowm Tumors and Bayer reached a unique cooperation agreement in November 2017 to develop two drugs: Larotrectinib and the next generation TRK cancer LOXO-195.
With regard to the distribution of labels, Loxo tutors will lead clinical research and subsequent regulatory issues in the US, while Bayer dominates regulatory issues outside the US and global business activities. In the United States, both parties will promote joint market promotional activities.
This is not a cheap business partnership: if the co-operation goes well, Bayer will pay Loxy Cancer a total of $ 1.55 billion.
Under the agreement, Bayer will pay an advance payment of $ 400 million to the Loxo tumor; When Larotrectinib receives regulatory approvals in a very important market and completes the first commercial sales, the latter will receive another $ 450 million. When the LOXO-195 completes the same steps, it will receive another $ 200 million.
In terms of expenditure, both companies will share the R & D costs; In the United States, both will share business spending and balance the profits. In the US market, Bayer will pay $ 25 million when a net sales reach a specific threshold; in the United States outside the US, both companies will use an innovative tier-to-tier approach to sales, and Loxo tumors will receive up to $ 475 million. .
Reuters quoted BTIG's financial firm BTIG Dane Leone saying that the annual sales of Larotrectinib will reach a peak in 2026, at $ 1.5 billion. The Financial Times quoted a Citigroup analysis saying that the annual sales of the drug will reach the maximum in 2030, or around $ 1.9 billion.
The newly approved drug is expensive, and Forbes Bayer quoted saying that capsules 100 mg per day for 30 days were $ 32,800 for adult capsules. The class form for adults or children is $ 11,000 per month.
Earlier, some analysts believe that both companies will face marketing difficulties. On the one hand, the proportion of patients with the disease is limited in cancer patients. Dr Yu, a botanist doctor at the University of Peking, mentioned in a popular cancer science article that accounts for only 0.21% of about 11,000 cancer patients that belong to TRK fungus cancer. On the other hand, associated resolving techniques are not currently popular, and it is not difficult to screen the corresponding patients.
Introduce Chinese market approval process or accelerate
When Larotrectinib was launched in the United States, the United States Food and Drug Administration's preferred procedures such as Priority Review, Cutting and Fast Approval Therapy. In fact, the agency has also widely publicized the drug approval process as a model for improving its efficiency.
Earlier, the US government promised to further streamline the Ministry of Food and Drug supervision. The Director of the new Gottlieb Food and Drug Administration stated publicly that there was a need to accelerate drug approval, support the promotion of over-the-counter drugs, and relax the generic drug supervision. Here is the Larotrectinib approved test.
The wise procedure of the United States Food and Drug Administration on drug approval has been criticized for a long time. Critics say that the long review time delayed the patient's treatment and on the other hand affects the commercial arrangement of the drug factory and costs increases. In response, the agency sought simplification and relaxation of regulation.
Drugs that receive priority approval often "have improved significantly in terms of the safety, effectiveness and effectiveness of treatment, diagnosis, or severe conditions" compared to drugs following the Standard Review process . Approval time was shortened from 10 months to 6 months.
An overweight therapy qualification may be relevant when the drug being developed is used to treat a severe condition and the initial clinical result indicates that it has a "significant improvement" in the long run " clinically meaningful ".
In the rapid approval process, the food and drug supervision can use the replacement point or an intermediate final point to approve the drug. In other words, when these indicators can predict that the drug is likely to have clinical effectiveness or benefit, approve the drug to the market subject.
According to Bayer, it will give verbal capsules and answers to patients in the US market; In addition, the company has submitted a listing request to the European Medicines Agency in August. Regarding the arrangements for listing in China, Bayer said that he will only continue to fulfill his commitment to delivering more innovative and leading products.
The industry's people told Tencent "Pixel", if Bayer intends to introduce related products in China, it is expected "the time of approval will not be very long." The reason is that Chinese drug regulatory agencies are also trying to improve the efficiency of drug approval.
This is demonstrated by the approval process for the monoclonal antibodies "Pabolym Anticancer Dwarf Drugs". The anti-cancer drug produced by Merck has been listed in China in July 2018, and the approval process took less than half a year. In a press release, Merck quoted the words of his Chinese president Luo Wanli, who strongly praised the practice of Chinese regulators actively introducing innovative drugs and increasing the speed of approving new drugs.
Improving workflow is one of the measures. Earlier, the Deloitte accounting company said in a report for innovative drugs, the Chinese "strict access" attitude allowed approval of clinical trials to be longer than other markets such as the United States.
In addition, local governments are also working hard. The Shanghai Municipal Government announced on July 10 that the "100-platform expansion plan" action plan had mentioned "to fight for the urgent need for clinically available anti-tumor drugs that have been listed overseas and have not & # 39; u approve to register in China, and it will be used in Shanghai. " .
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The original drug "original" spectrum antagonist "Deficit": a 75% improvement rate is misinterpreted, which is relevant to be less expensive and difficult to sell. Prism
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