Source: Prism (ID: lengjing_qqfinance), Wall Street Journalist
Edit | Yang Wei
The treatment of rare types of tumors has recently been approved by the US Food and Drug Administration (FDA), and it has been determined that it has a 75% cure rate for several types of cancer. This caused great concern among people. However, it is not.
An approved drug called Larotrectinib (trade name Vitkravi) was jointly developed by the high-precision medical company Loxo Tumor and the traditional pharmaceutical manufacturer Bayer. Bayer said the drug is the first anticancer chemical approved by the US Food and Drug Administration "without distinguishing between types of tumors."
The US Food and Drug Administration said the latest approved drugs would prevent the abnormal fusion of the NTRK gene with other genes — a fusion that was observed to result in a growth signal that supports tumor development. NTRK fusion is not a common occurrence, and it usually happens only in cancer parts of the body, including lung cancer and thyroid cancer.
75% cure rate misunderstood
According to Bayer, the overall response rate (ORR, response rate to translation) was 75% among adults and children participating in the experiment, including 22% complete remission and 53% partial relief. The so-called remission rate refers to the proportion of people who have decreased or disappeared after the patient received treatment and does not relate to the proportion of treatment.
According to experimental data, a total of 55 patients participated in these three experiments. The US Food and Drug Administration said that the experimental response was stable: according to the analysis of the experimental results, 73% of the relief lasted at least six months, and 39% of the relief lasted at least one year. Approved drugs are effective for soft tissue sarcoma, salivary gland cancer, pediatric fibrosarcoma, thyroid cancer, and lung cancer.
Although approved new drugs can treat various types of cancer, known as “broad-spectrum anticancer drugs,” they are effective only for a few patients with certain characteristics at the genetic level. Thus, the US Food and Drug Administration also gives it a "qualification for orphans" – according to the relevant legislation, orphan drugs can receive convenient conditions, such as quick approval, tax cuts, exemption from reporting fees, scientific subsidies research and development, etc., that will encourage pharmaceutical companies to assist and encourage the development of medicines for the treatment of rare diseases.
In particular, only those patients with "merging NTRK genes in tumors" are eligible for treatment with this newly approved drug. According to Bayer, an appropriate diagnosis can be diagnosed using next-generation sequencing technology (NGS) and in situ hybridization fluorescence (FISH).
In a press release, Bayer quotes Robert Lacaze, the head of his business unit for the oncology strategy, saying: “NTRK fusion is rare, but it exists in many different types of tumors … for those who have NTRK gene fusion. It is very important to provide a specialized treatment plan for patients with advanced solid tumors. ”
According to Bayer, the US Food and Drug Administration "approves the overall response rate and duration of remission based on a drug for treating a tumor" and approves it for "adults and children with solid tumors" in accordance with the accelerated procedure. He also stated that "the subsequent statement may depend on the verification and description of the clinical benefits in the confirmatory test."
US Food and Drug Administration Commissioner Scott Gottlieb said that the approval of a new drug reflects the progress of biomarkers in managing drug development and more accurate drug use.
High price, hard to sell
Loxo Tumors and Bayer signed an exclusive cooperation agreement in November 2017 on the development of two drugs: Larotrectinib and the new-generation TRX cancer inhibitor LOXO-195.
With regard to the division of labor, Loxo tumors will conduct subsequent clinical research and regulatory issues in the United States, while Bayer dominates regulatory problems outside the United States and global business activities. In the United States, both parties will jointly promote market promotion activities.
This is not a cheap business partnership: if the cooperation goes well, Bayer will pay a total of $ 1.55 billion. Loxo Cancer.
Under the agreement, Bayer will pay an advance of $ 400 million for a Loxo tumor, when Larotrectinib receives regulatory approval from a specific large market and completes the first commercial sale, the latter will receive another $ 450 million. When LOXO-195 performs the same steps, it will receive another $ 200 million.
As for costs, the two companies will share the costs of research and development; in the United States, they will share business expenses and equalize profits. In the US market, Bayer will pay $ 25 million when net sales reach a certain threshold, and in the United States outside the US, both companies will use a phased progressive approach to sales, and Loxo tumors will receive up to $ 475 million. ,
Reuters quotes BTIG financial company analyst Daina Leone, saying that the annual sales of Larotrectinib will reach a peak in 2026, reaching $ 1.5 billion. The Financial Times cites Citigroup analysis, which says that the annual sales of the drug will reach a peak in 2030, or about $ 1.9 billion.
The recently approved drug is expensive, and Forbes quotes Bayer that for adult capsules the cost of a package of 100 mg capsules per day for 30 days is $ 32,800. Dosage form for adults or children is $ 11,000 per month.
Previously, some analysts believed that both companies would face marketing difficulties. On the one hand, the proportion of patients with this disease is limited in cancer patients. Dr. Y., a doctor of botany at Peking University, mentioned in a popular science article on cancer that only 0.11% of about 11,000 cancer patients are cancer of the TRK diet. On the other hand, the associated detection methods are not currently popular, and it is not difficult to identify the relevant patients.
Introduce the Chinese market approval process or speed up
When Larotrectinib was launched in the United States of America, the US Food and Drug Administration selected procedures such as Priority Review, Breakthrough Therapy and Accelerated Approval. In fact, the agency also widely covered the drug approval process as a model to increase its effectiveness.
Earlier, the new US government promised to further simplify the supervision of the Office of the Food and Drug Administration. The new director of food and drug control, Gottlieb, has publicly stated that it is necessary to speed up the approval of drugs, support the promotion of over-the-counter drugs, and stop the supervision of generic drugs. This is proof that Larorectinib has been approved.
The US Food and Drug Administration’s reasonable procedure for drug approval has been criticized for a long time. Critics say that a long review time delays the treatment of patients, and on the other hand affects the commercial location of the pharmaceutical factory and increases costs. In response, the agency tried to simplify and weaken the regulation.
Drugs that receive priority approval are often “significantly improved in terms of safety, efficacy and effectiveness of treatment, diagnosis or prevention of serious conditions” compared with drugs that follow the standard review process. Approval time reduced from 10 months to 6 months.
Claiming pulmonary therapy can be used when the drug being developed is used to treat a serious condition, and the initial clinical result indicates that it has a “significant improvement” in the “clinically significant” endpoint.
In an accelerated approval process, food and drug surveillance may use an end point of substitution or an intermediate end point for drug approval. In other words, when these indicators can predict that a drug is likely to have efficacy or clinical benefit, conditionally approve the drug on the market.
According to Bayer, he will provide patients with oral capsules and solutions in the US market, in addition, the company applied for participation in the European Medicines Agency in August. With regard to listing arrangements in China, Bayer only said that he would continue to fulfill his obligations to introduce more innovative and leading products.
Industrial people told Tencent "Pixel" that if Bayer really intends to introduce related products in China, it is expected that "the approval time will not be very long." The reason is that Chinese drug control agencies are also trying to improve drug approval.
This is illustrated by the approval procedure for the “Broad Specific Anticancer Drug” monoclonal antibody, Sabolim. The anti-cancer drug produced by Merck was registered in China in July 2018, and the approval process took less than six months. In a press release, Merck quoted the words of his Chinese President, Luo Wanli, and praised the practice of Chinese regulators who are actively introducing innovative drugs and increasing the speed of approval of new drugs.
One of the measures is to improve the workflow. Earlier, the accounting firm Deloitte stated in its report that, for innovative drugs, the “strict entry” attitude of the Chinese government allowed us to approve applications for clinical trials longer than in other markets such as the United States.
In addition, efforts Defangzhengfu. On July 10, the Shanghai government announced that the “Expansion plan for 100 people” action plan mentions “to fight for the urgent need for clinically available anticancer drugs that were listed abroad and were not approved for registration in China and are being used in Shanghai”. ,
End of text
Please leave a message in the comments area below.of
Messages, as the first place to get 30Q coins, as of December 3 (next Monday).
The original name “Decipher” a broad-spectrum anticancer drug: 75% treatment rate is incorrect, applicable to less expensive and difficult to sell | Prism
If you have a quality, intimate, and original article, please feel free to contribute to the famous column on Wall Street.
Submission method : Please send your personal profile, and a representative works at email@example.com, and attach a phone call and WeChat for further communication. In the subject specify: Submit an application to enter the column + Member Name